HALF-YEAR REPORT 2021 - Investors
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VIFOR
PHARMA
HALF-YEAR
REPORT
2021
TA B L E O F
CONTENTS
ON THE COVER:
Faizan is a kidney transplant patient
Learn more about his story at Viforpharma.com/patient-stories
2 Vifor Pharma Ltd. | Half-year Report 2021
HIGHLIGHTS
HIGHLIGHTS OUTLOOK
4 Letter to Shareholders 42 2021 Outlook and financial guidance
6 Financial Highlights
8 Our Company
12 Performance Overview C O N S O L I DAT E D I N T E R I M
F I N A N C I A L S TAT E M E N T S
PORTFOLIO
PORTFOLIO 46 Consolidated statement of income
47 Consolidated statement of
IRON THERAPIES comprehensive income
17 Ferinject®/Injectafer® 48 Consolidated statement of financial
21 Maltofer® position
22 Vamifeport (VIT-2763) 49 Consolidated statement of changes
N E P H R O LO GY in equity
29 Mircera® 50 Consolidated statement of cash flows
29 Retacrit® 51 Notes to the consolidated financial
30 Venofer® statements
30 Vadadustat
30 Velphoro®
32 Rayaldee®
32 Difelikefalin 56 Upcoming dates
34 Avacopan 57 Contact information
35 ANG-3777
CARDIO-RENAL
OUTLOOK
37 Veltassa®
FINANCE
Vifor Pharma Ltd. | Half-year Report 2021 3
HIGHLIGHTS
LETTER TO SHAREHOLDERS
DEAR SHAREHOLDERS, ing clinical practice for patients in cardiac surgery.
Among the most important updates to the practice
In the first half of 2021, Vifor Pharma Group guideline is the change from blood preservation to
reported growth in both revenues and profitability. PBM, focusing on a multidisciplinary management
Strong recovery was reported in key markets approach for patients.
during the second quarter, as patient access
improved, and COVID-19 measures were lifted. We Recognized by the World Health Organization as
further strengthened our leadership position in playing a key role in optimizing clinical practice, PBM
iron deficiency and made significant progress addresses a significant and underserved patient
towards becoming a global leader in nephrology. population. A real-world evidence study is underway
We are proud of our employees and partners, in 2021, to analyze the clinical and economic impact
whose ongoing diligence has ensured patients and of implementing PBM measures with Ferinject®.
healthcare providers receive the treatments on We look forward to preliminary results expected at
which they depend despite challenging circum- year-end, and publication in 2022.
stances.
Within our nephrology product portfolio,
In H1 2021, net sales increased by 5.0%, at constant Velphoro® maintained a leadership position by
exchange rates (CER), to CHF 859.3 million. This sales value in the phosphate binder market, with
encouraging performance was driven by a a 31.7% market share as of April 2021. Venofer®
significant rebound in Ferinject®/ Injectafer® sales, also maintained its position as a leading i.v. iron
up 22.8% at CER. EBITDA in H1 2021 increased to brand in terms of volume, with sales mainly driven
CHF 281.0 million, or by 5.3% at CER. Based on by continued stronger demand in hospitals in the
a solid first half and continued positive momentum US.
we are confident to achieve full-year guidance.
We are confident that the innovative pipeline of
Our Ferinject®/ Injectafer® business realized kidney disease therapies, including avacopan,
strong growth recovery in key markets from March difelikefalin, vadadustat and ANG-3777, have the
onwards. We are confident that the rebound in potential to generate significant growth and to
growth will continue in parallel to the easing of further establish Vifor Pharma as a global leader in
COVID-19 restrictions and the resulting improve- nephrology. In the first half of 2021, we made major
ment in patient access to i.v. iron treatment. progress in our nephrology pipeline with several
regulatory submissions, and significant progress in
We continue to invest in clinical trials to demon- a number of clinical trials.
strate the benefits of Ferinject® in therapy areas
with high unmet medical need. In H1 2021, addi- Positive top-line data from the pivotal phase-III
tional publications have followed results from the ADVOCATE trial for avacopan were published in
AFFIRM-AHF trial, including a sub-analysis on the “New England Journal of Medicine” in Febru-
quality of life published in June by the “European ary, further demonstrating important clinical
Heart Journal.” Results showed a statistically benefits in patients with C3G kidney disease, for
significant and clinically meaningful improvement in which there is currently no approved therapy.
the quality of life for patients treated with Ferinject®
and help highlight the need for chronic heart failure We were also pleased to announce the US Food
patients to be more frequently screened for iron and Drug Administration (FDA) acceptance and
deficiency and treated accordingly. priority review of Cara Therapeutics’ New Drug
Application (NDA) for difelikefalin, with a PDUFA
The pandemic has highlighted the importance of target date of 23 August 2021. In addition, the
Patient Blood Management (PBM), as a decline in European Medical Agency accepted the Marketing
blood donations around the world put lives at risk. Authorisation Application for review. Vifor Frese-
An update to the “Clinical Practice Guidelines for nius Medical Care Renal Pharma and Otsuka
Blood Conservation” was published in June by Canada signed an agreement to commercialize
several global leading medical societies, further both avacopan and difelikefalin in Canada.
strengthening the importance of PBM in optimiz-
4 Vifor Pharma Ltd. | Half-year Report 2021
HIGHLIGHTS
Our partner Akebia Therapeutics announced that In June, we were delighted to announce that Abbas
the US FDA accepted the NDA for vadadustat for Hussain will take on the position as Chief Executive
the treatment of anemia due to chronic kidney Officer of Vifor Pharma. Abbas is an experienced
disease (CKD) in patients on dialysis and not on and entrepreneurial leader. With a track record of
dialysis. The FDA assigned standard review, with over 30 years at an international level, he has held
a PDUFA date set on 29 March 2022. At the time of various leadership positions at major pharmaceu-
filing, the FDA also indicated that they were not tical companies including GlaxoSmithKline and Eli
currently planning to hold an Advisory Commit- Lilly. He has impressive experience across the
tee meeting to discuss the application for pharmaceutical value chain in both mature and
vadadustat. emerging markets, and is ideally suited to further
drive Vifor Pharma’s growth.
Another important milestone was the completion
of patient enrolment in a comprehensive clinical Stefan Schulze has decided to step down as CEO
PORTFOLIO
program to advance ANG-3777 as a potential and leave our company after seven years of
treatment option to prevent acute kidney injury distinguished service. Prior to taking on the role of
following cardiac surgery, an indication with high CEO in May 2020, he was President of the Executive
unmet medical need. We are looking forward to Committee and Chief Operating Officer of Vifor
the results of the phase-II trial, as well as a phase-III Pharma from 2017 to 2020. He joined the company
study in transplant-associated acute kidney injury, in 2014 as CEO of VFMCRP. The Board of Directors
expected later in the year. sincerely thank Stefan Schulze for his contribution
and commitment to Vifor Pharma’s success.
While the potassium binder market hasn’t grown in
line with our expectations recently, we believe in Dr Alexandre LeBeaut and Åsa Riisberg were both
the growth of Veltassa® and are working to drive elected to the Board of Directors at the Annual
evidence-based care in CKD and heart failure General Meeting in May. Their broad experience
through several studies to further differentiate the will optimally complement the Board to ensure
product. A significant step forward was the Vifor Pharma’s ongoing success. After 25 years
enrolment of the first patient into the CARE-HK with Vifor Pharma and four years of service on the
global registry platform. This registry aims to Board of Directors, Dr. Gianni Zampieri did not
improve understanding of treatment decisions stand for reelection. He has been instrumental in
with RAAS (renin-angiotensin-aldosterone system) the strategic development of the company in
OUTLOOK
inhibitors in clinical practice, barriers to optimal various roles. On behalf of the Board of Directors,
guideline-directed care in heart failure patients we extend our appreciation to him for his excellent
with or at high risk of hyperkalemia, and to assess and successful work for Vifor Pharma.
use of Veltassa® in management of this patient
population. Our achievements in the first half of the year were
made possible through the continued confidence
In June, we announced that the phase-IIIb and support of our shareholders, and the dedica-
DIAMOND study was amended with new and tion and hard work of our employees. We can look
clinically relevant endpoints, following the recom- forward with confidence and optimism to an
mendation of the independent study Executive exciting future ahead as we deliver on our strategy
Committee, and the significant impact of to become global leaders in iron deficiency and
COVID-19 on recruitment as well as lower inci- nephrology therapies.
dence of cardiovascular events. We are looking
forward to publishing the data during spring 2022 Yours sincerely,
at a major conference.
Our ferroportin inhibitor, vamifeport (VIT-2763),
FINANCE
was granted orphan drug designation by the FDA
and EMA for sickle cell disease (SCD) in Q1 2021. Stefan Schulze Jacques Theurillat
A phase-IIa trial in SCD has been initiated. Chief Executive Officer Chairman
Vifor Pharma Ltd. | Half-year Report 2021 5
HIGHLIGHTS
EBITDA NET SALES
859.3
MILLION
CHF
+1.5%
(+5.0% AT CER)
281.0
MILLION
CHF
+0.2%
(+5.3% AT CER)
CORE EARNINGS CASH FLOW FROM EQUITY
PER SHARE¹ FROM- OPERATING RATIO
CONTINUING ACTIVITIES
OPERATIONS
2.92CHF
189.8
MILLION
76.5
%
+22.7% CHF -0.6 P.P.
+17.1 MILLION CHF
1
Core earnings are defined as net
profit attributable to shareholders of
Vifor Pharma Ltd. adjusted for
proportionate amortization and
impairment of intangible assets.
6 Vifor Pharma Ltd. | Half-year Report 2021
HIGHLIGHTS
FERINJECT®/ INJECTAFER®
NET SALES
320.5
MILLION
CHF
+22.4%
(+22.8 % AT CER)
PORTFOLIO
MIRCERA®/ RETACRIT®
NET SALES
246.4
MILLION
CHF
–15.8%
(–9.7% AT CER)
OUTLOOK
VELPHORO®
NET SALES
79.9
MILLION
CHF
+8.1%
(+13.6% AT CER)
FINANCE
Vifor Pharma Ltd. | Half-year Report 2021 7
HIGHLIGHTS
O U R C O M PA N Y
OUR MISSION
We strive to help patients around the world
with severe and chronic diseases lead better,
healthier lives.
OUR VISION
To be global leader in iron deficiency,
nephrology and cardio-renal therapies.
O U R C O M PA N Y
At Vifor Pharma, patients are at the center of market and aim for global leadership in the
everything we do, and we go where only few segment of cardio-renal therapies. Our
go to find the right treatment solutions for continued success is underpinned by a strong,
them. Over the last three decades, we have diversified portfolio of commercial products
proven our ability to identify and serve key and an exciting pipeline.
therapeutic areas with significant unmet
medical need, successfully building on our We are proud to be a partner of choice for
experience and ability to create new markets. pharmaceuticals and innovative patient-
focused solutions worldwide. By leveraging
We are one of the largest Swiss pharmaceuti- our unique competitive strengths to in-license
cal companies and a global leader in the promising new products and compounds,
treatment of iron deficiency. We have estab- we continue to build strong partnerships and
lished a strong presence in the nephrology alliances to drive our vision forward.
8 Vifor Pharma Ltd. | Half-year Report 2021
HIGHLIGHTS
PORTFOLIO
OUTLOOK
FINANCE
Vifor Pharma Ltd. | Half-year Report 2021 9
HIGHLIGHTS
O U R C O M PA N Y
O U R S T R AT E GY Pharma (VFMCRP), which for over ten years,
has combined Vifor Pharma’s extensive and
Partnering and in-licensing is the cornerstone growing portfolio of nephrology medicines
of Vifor Pharma’s growth strategy. Our with Fresenius Medical Care’s nephrological
therapies are sold in more than 100 countries competency, strong dialysis expertise and
through our own commercial expertise in key infrastructure.
markets and best-in-class partnerships
designed to increase global reach. Our ability The cardio-renal patient population is a
to work successfully with partners across natural extension of our existing therapy area
the world has enabled us to strengthen ties focus in both nephrology and cardiology.
with patients, healthcare professionals and Vifor Pharma is well positioned to unlock
payers – to drive innovation, build awareness significant growth potential, by addressing
and allow patients access to the treatments underserved co-morbidities such as iron
they need and deserve. Working with others deficiency and hyperkalemia in cardio-renal
enables us to continually expand our innova- patients.
tive portfolio of iron deficiency, nephrology
and cardio-renal therapies, acquiring and At Vifor Pharma, our people are key to our
in-licensing novel late-stage assets and success. We strive to cultivate a unique
creating mutually beneficial joint ventures. culture, and resilient and agile business
operating model by bringing our values of
As pioneers in iron-based therapies, we have Entrepreneurship, Respect and Teamwork to
and continue to demonstrate strong scientific, life. We believe we can accomplish our vision
regulatory and commercial expertise to identi- of becoming global leader in iron deficiency,
fy opportunities that help create, develop and nephrology and cardio-renal therapies by
mature markets. Around one in three people listening to the patients we serve, and en-
worldwide are affected by iron deficiency1 and abling our employees to achieve their full
the market for therapies has grown substan- potential. By engaging with patients, valuing
tially over the past 25 years. We aim to drive our employees, caring for the environment
further growth by raising awareness of the and supporting communities, we demonstrate
symptoms and serious health consequences our determination to lead by example and to
of iron deficiency, to ensure patients receive remain at the forefront of our industry. As
appropriate treatment. a global pharmaceutical company, we recog-
nize our wider responsibilities and are com-
Nephrology is also a large and expanding mitted to conducting business with integrity.
market, with chronic kidney disease (CKD)
affecting more than one in ten of the global The global COVID-19 pandemic has under-
population1. Vifor Pharma is establishing itself lined our commitment to putting patient
as the partner of choice in nephrology, health and quality of life at the heart of
bridging the gap between high unmet everything we do. Over the last year, we have
medical need and patient reach. Through implemented measures to protect patients by
an expanding, innovative nephrology portfo- securing continuity of product supplies, at the
lio and individualized solutions, our goal is to same time as ensuring the health and well-
preserve renal function, manage complica- being of our employees and partners, and the
tions and improve quality of life for chronic communities in which we operate. We are
kidney disease patients. Our strong presence proud of the dedication of our colleagues in
in nephrology is built on our unique joint manufacturing, quality control, supply chain
company, Vifor Fresenius Medical Care Renal and many other functions, whose hard work
ensured our medicines continued to reach
1 Hill NR et al PLOS ONE, DOI:10.1371/journal. pone.0158765 patients and healthcare providers in such
10 Vifor Pharma Ltd. | Half-year Report 2021OUR THERAPY AREAS
HIGHLIGHTS
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OUTLOOK
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NEPHROLOGY
challenging conditions. We also took steps to other partners to help meet the health
protect patients and caregivers involved in challenges of COVID-19. Our Patient Advocacy
clinical trials, fully complying with regulatory team is offering support and guidance to
guidance. patient organizations, and a global fund to
FINANCE
support patient groups has been launched
Vifor Pharma Group continues to work closely with the aim to improve patients’ health and
with the European Federation of Pharmaceuti- quality of life while social and economic
cal Industries and Associations (EFPIA) and restrictions are in place.
Vifor Pharma Ltd. | Half-year Report 2021 11HIGHLIGHTS
PERFORMANCE OVERVIEW
KEY PROFIT AND LOSS FIGURES1 Investments in R&D amounted to CHF 108.1
million compared to CHF 146.6 million in
Vifor Pharma Group net sales increased by H1 2020. The decrease was mainly attributable
1.5% to CHF 859.3 million compared to to the impairment of the CCX140 intangible
H1 2020, or 5.0% at constant exchange rates asset of CHF 56.2 million in H1 2020, partially
(CER), as patient access recovered in line with offset by higher DIAMOND, CARE-HK and
easing COVID-19 restrictions in key markets. vamifeport (VIT-2763) study costs in H1 2021
The growth was mainly driven by our iron compared to H1 2020.
portfolio with better patient access to i.v. iron
treatment. General and administration expenses
amounted to CHF 71.0 million compared to
EBITDA increased to CHF 281.0 million CHF 90.1 million in H1 2020. The decrease
compared to CHF 280.4 million in H1 2020, was mainly driven by a reduction of person-
an increase of 0.2% or 5.3% at CER despite nel-related costs across support areas.
a significant decline in other income. Excluding
other income, EBITDA increased by 9.4% The average number of permanent full-time
compared to H1 2020, or 15.0% at CER. This is equivalents (FTE) amounted to 2,384 in
a result of profitable growth in net sales H1 2021, compared to 2,446 in H1 2020. This
combined with diligent cost containment. represents a decrease of 62 FTEs or 2.5%. This
reduction was achieved as a result of optimi-
Other income was CHF 20.4 million compared zation in operational, commercial and support
with CHF 42.2 million in H1 2020. This was functions and despite investments being
primarily due to lower income from partnering made in preparation for the launch of our
and other activities compared to H1 2020. The pipeline products.
higher other income in the prior period was
driven by one-off payments for partnering of Depreciation, amortization, and impairment
Ferinject® in China as well as Velphoro® and amounted to CHF 103.7 million compared to
Veltassa® in Canada, a gain on disposal of CHF 166.9 million in H1 2020. Of these
non-core products in Spain and CellCept® amounts, CHF 82.9 million and CHF 90.2
royalties for which the royalty period ended in million, respectively, are recorded under cost
2020. of sales. The decrease of CHF 63.2 million
compared to H1 2020 is mainly due to the
Cost of sales amounted to CHF 329.7 million aforementioned impairment of the CCX140
compared to CHF 345.9 million in H1 2020, intangible asset in H1 2020.
resulting in a gross profit margin of 62.5%
compared to 61.1% in H1 2020. The gross profit The net financial result amounted to
margin increase is primarily due to the higher an income of CHF 36.6 million compared to
share of sales from injectable iron products. an expense of CHF 14.1 million in H1 2020.
The increase in the net financial result com-
Marketing and distribution expenses pared to H1 2020 is driven by revaluation gains
amounted to CHF 193.5 million, up 0.6% from on financial investments of CHF 22.4 million
H1 2020. The additional investments in and unrealized foreign currency gains of
pre-launch activities of our pipeline products CHF 13.3 million, mainly on USD cash positions.
were largely offset by cost containment.
Tax expense amounted to CHF 30.2 million in
H1 2021, corresponding to an effective tax
rate (ETR) of 14.2%. The ETR in H1 2020 of 8.6%
1 On 30 September 2020, the Group completed the sale was unusually low due to a one-off tax gain of
of OM Pharma. Therefore OM pharma is presented as a
discontinued operation and the prior-period profit or
CHF 6.1 million related to prior periods.
loss figures are restated accordingly.
12 Vifor Pharma Ltd. | Half-year Report 2021HIGHLIGHTS
Net profit attributable to non-controlling Cash flow from financing activities
interests increased to CHF 57.8 million from amounted to CHF –208.9 million and was
CHF 38.8 million, due to the proportionate mainly influenced by dividend distributions of
impact of impairing the CCX140 intangible CHF –189.8 million, whereof CHF –60.0 million
asset in H1 2020. was paid to Fresenius Medical Care and
CHF –129.8 million was distributed to share-
Net profit attributable to shareholders of holders of Vifor Pharma.
Vifor Pharma Ltd. for H1 2021 increased to
CHF 124.4 million compared to CHF 67.9
million in H1 2020. The increase was mainly FINANCIAL POSITION
driven by the aforementioned lower deprecia-
Goodwill and intangible assets amounted to
PORTFOLIO
tion, amortization and impairment, as well as
the strong net financial result in H1 2021. CHF 2,447.5 million at the end of H1 2021
compared to CHF 2,454.5 million at the end of
Core earnings per share from continuing 2020, representing 49.8% of total assets (end of
operations amounted to CHF 2.92 in H1 2021, 2020: 47.1%).
an increase of 22.7% compared to CHF 2.38
in H1 2020. The increase reflects the afore Financial assets amounted to CHF 386.2
mentioned revaluation and foreign currency million at the end of H1 2021 compared to
gains in the net financial result of H1 2021. CHF 725.7 million at the end of 2020. The
Core earnings are defined as net profit attribut- significant decrease is due to the fair value loss
able to shareholders of Vifor Pharma Ltd. of CHF 386.5 million on our equity investment
adjusted for proportionate amortization and in ChemoCentryx, Inc. Revaluation adjustments
impairment of intangible assets of CHF 65.2 on our equity investments are recorded in
million in H1 2021 (H1 2020: CHF 104.6 million). other comprehensive income.
As a result of the revaluation of financial assets,
CASH FLOWS total assets declined significantly from
CHF 5,211.7 million at the end of 2020 to
OUTLOOK
Cash flow from operating activities CHF 4,916.3 million at the end of H1 2021.
amounted to CHF +189.8 million compared to
CHF +172.6 million in H1 2020. The increase is Vifor Pharma Group achieved a net cash
due to lower investments in net working capital position of CHF 86.6 million at the end of
in H1 2021 compared to H1 2020. H1 2021 compared to a net cash position of
CHF 190.6 million at the end of 2020. The
Cash flow from investing activities decrease in net cash is mainly due to the
amounted to CHF –98.6 million. The key dividend payment of CHF 189.8 million and
drivers are capital expenditures of CHF –26.0 investments made in H1 2021 which exceed
million in production-related assets, milestone the cash generated from operating activities.
payments for in-licensing agreements of
CHF –27.5 million, investments in IT projects of With CHF 3,763.0 million of shareholders’
CHF –15.0 million and the equity investment in equity at the end of H1 2021, Vifor Pharma
Angion Biomedica Corp. of CHF –22.3 million Group continues to have a strong equity ratio
concurrent with Angion’s initial public offering of 76.5%.
in February 2021.
FINANCE
Vifor Pharma Ltd. | Half-year Report 2021 13PORTFOLIO 14 Vifor Pharma Ltd. | Half-year Report 2021
HIGHLIGHTS
IRON THERAPIES
17 Ferinject®/Injectafer®
21 Maltofer®
22 Vamifeport (VIT-2763)
N E P H R O LO GY
29 Mircera®
29 Retacrit®
PORTFOLIO
30 Venofer®
30 Vadadustat
30 Velphoro®
32 Rayaldee®
32 Difelikefalin
34 Avacopan
35 ANG-3777
CARDIO-RENAL
37 Veltassa®
OUTLOOK
FINANCE
Vifor Pharma Ltd. | Half-year Report 2021 15OUR PORTFOLIO
IRON THERAPIES
OWN PRODUCTS AND *PIPELINE
Vifor Pharma has been
a pioneer in the
development of iron-based
products and has
Vamifeport* (VIT-2763) established itself as
a global leader in the
treatment of iron
deficiency. Our leadership
is built on our scientific,
regulatory and commercial
expertise, resulting in the
creation of globally trusted
brands.
16 Vifor Pharma Ltd. | Half-year Report 2021HIGHLIGHTS
Y E A R -T O - D AT E G R O W T H
22.8%
TOTAL FERINJECT®/INJECTAFER®
NET SALES INCREASE (AT CER)
I M PAC T O F C O V I D - 1 9
The COVID-19 pandemic continued to
negatively influence the number of patients
PORTFOLIO
actively seeking treatment for iron deficiency
and iron deficiency anemia during the first
quarter of 2021. From March onwards, Vifor
Pharma was able to increase engagement with
customers as COVID-19 measures were lifted,
F E R I N J E C T ® / I N J E C TA F E R ® and patient access to healthcare providers
improved in key markets. Over the same
Ferinject®/Injectafer® (ferric carboxymaltose) period, global net sales performance signifi-
is a leading intravenous (i.v.) iron therapy with cantly improved with year-to-date growth up
market authorization in 84 countries by the 22.8% at CER by the end of June compared
end of June 2021. Ferric carboxymaltose is with prior year. While all markets experienced
commercialized under the brand name a return to growth from March onwards, the
Injectafer® in the US. More than 16 million rate and magnitude varied across different
patient years of experience have helped to markets. Overall utilization of i.v. iron has been
establish Ferinject®/ Injectafer® as a trusted highly correlated with the intensity of lock-
brand, with clinical benefits demonstrated by down measures, and a return to pre-pandemic
its efficacy and safety data1. growth rates is anticipated.
OUTLOOK
Affecting up to one third of the global popula- Overall, key therapy areas continue to be
tion, iron deficiency and iron deficiency affected to varying degrees, with treatment
anemia are serious conditions with a wide for patients requiring emergency procedures
range of debilitating symptoms which can prioritized over those with non-life-threaten-
have a significant impact on quality of life for ing conditions. Elective surgeries for instance,
patients. Ferinject® continues to address the which have been significantly impacted during
high unmet medical need and growth poten- the pandemic, are postponed rather than
tial for treatment of patients with iron deficien- cancelled. With fewer people donating blood
cy and iron deficiency anemia2. Key therapy during the pandemic, global associations have
areas include chronic heart failure, patient recognized the importance of implementing
blood management (PBM), nephrology, PBM to overcome blood shortages and
gastroenterology, oncology and women’s maintain optimal patient care while conserv-
health. ing blood supplies for patients who need it
most. In situations where healthcare could not
be postponed, e.g., caring for patients with
iron deficiency during pregnancy and patients
with iron deficiency anemia requiring dialysis,
FINANCE
we have seen that i.v. iron treatment continued
steadily throughout the pandemic. In addition,
1 Scott Drugs. 2018 Mar;78(4):479–493. doi: 10.1007/
s40265-018-0885-7.
physicians had the option to use a high dose
2 Consolidation of DA patient’s drop-off GBP 22-24. of Ferinject® in pregnant iron deficient
Vifor Pharma Ltd. | Half-year Report 2021 17IRON THERAPIES
patients for a proper and sustained iron which is associated with reduced quality of
repletion, potentially reducing the number of life, reduced exercise capacity and increased
hospital appointments for repeated infusions. risk of hospitalization. In the EU5, more than
80% of eligible iron deficient heart failure
patients remain undiagnosed, untreated or
REPORTED NET SALES IN H1 2021 inadequately treated3. Increasing awareness
around the importance of diagnosis and
Ferinject® / Injectafer® net sales increased treatment of iron deficiency in heart failure
22.4% (22.8% at CER) to CHF 320.5 million in patients remains a key priority for Vifor
H1 2021 from CHF 261.9 million a year earlier. Pharma, as is our focus on Ferinject® as
Net sales performance recovered strongly a guideline recommended treatment option.
and in line with easing of COVID-19 restric-
tions, and improved patient access to infu- The publication of the AFFIRM-AHF study in
sions in Q2. Overall strong market recovery The Lancet in November 2020, was a major
was led by major European markets as well as milestone for Vifor Pharma. The study findings
Australia and New Zealand. Continued growth showed that, compared with placebo, treat-
is expected in the second half of the year. ment with Ferinject® initiated at hospital
discharge in stabilized patients with acute
heart failure and concomitant iron deficiency,
I N J E C TA F E R ® U S resulted in a risk reduction4 for the combined
endpoint of total heart failure hospitalizations
In the US, our partner American Regent, Inc., and cardiovascular death, falling just short of
recorded net sales of USD 239.2 million in H1 statistical significance. The total number of
2021, up from USD 205.7 million a year earlier. heart failure hospitalizations was significantly
During the first half, sales began to rebound lower in the ferric carboxy-maltose group
from the COVID-19 pandemic as federal and compared with the placebo group. Statistically
state lockdown restrictions eased, and access significant treatment benefits of ferric
to physicians and infusion sites recovered. carboxymaltose compared with placebo were
seen for the time to first heart failure
hospitalization or cardiovascular death, and
THERAPY AREAS WITH HIGH for days lost due to heart failure hospitalization
UNMET MEDICAL NEED and cardiovascular death5.
Vifor Pharma is committed to further building The study demonstrated that administration
market awareness, improving diagnosis and of Ferinject® in stabilized AHF patients with
ensuring treatment in therapy areas with high iron deficiency significantly reduces the risk of
unmet medical need. In 2021, the company subsequent heart failure hospitalizations6, and
continues to build global market awareness of highlights the need for AHF patients to be
iron deficiency and iron deficiency anemia more frequently screened for iron deficiency.
through a number of lifecycle management
activities to further demonstrate the strong
3 Drop-off consolidated from CBP 2020.
safety and tolerability profile of the product in 4 Risk reduction rate of of 0·79 (95% CI 0·62–1·01,
key therapy areas, including chronic heart p=0·059)
5 Ponikowski P. et al (2020) Ferric carboxymaltose for iron
failure, PBM, nephrology, women’s health and deficiency at discharge after acute heart failure: a
gastroenterology. multicentre, double-blind, randomised, controlled trial.
The Lancet, 396, 1895-1904 DOI:https://doi.
org/10.1016/S0140-6736(20)32339-4
Chronic Heart Failure 6 Anker, S.,D. (2018). Effects of ferric carboxymaltose on
hospitalisations and mortality rates in iron-deficient
One in every two chronic heart failure patients heart failure patients: an individual patient meta-analy-
has iron deficiency or iron deficiency anemia, sis. Eur J Heart fail. 20(1):125-133. doi: 1002/ejhf.823.
18 Vifor Pharma Ltd. | Half-year Report 2021HIGHLIGHTS
PAT I E N T S
16 MILLION
PATIENT YEARS OF EXPERIENCE
(APPROX.)
PORTFOLIO
OUTLOOK
84
FINANCE
FERINJECT®/INJECTAFER® IS APPROVED
IN 84 COUNTRIES WORLDWIDE
Vifor Pharma Ltd. | Half-year Report 2021 19IRON THERAPIES
The results of the trial may lead to an updated serving patients’ blood. In the EU’s five largest
reference to ferric carboxymaltose in the countries, an estimated 1.2 million patients8
ESC-HFA guidelines on heart failure. The final are not treated and could benefit from PBM.
2021 ESC HF guidelines are expected to be Proactive identification and treatment of iron
published during the ESC 2021 congress at deficiency anemia in patients scheduled for
the end of August. elective surgery has been associated with
a reduced need for blood transfusion9,
In the first half of 2021, additional related reduced length of hospital stay10, improved
publications followed. A sub-analysis of the patient’s outcomes11, and overall healthcare
AFFIRM-AHF study on the health-related expenditure reduction12.
quality of life was published in June 2021,
which showed a statistically significant and A cost-effectiveness analysis at the Fiona
clinically meaningful improvement in quality Stanley Hospital in Western Australia showed
of life for patients treated with ferric carboxy- potential cost savings of AUD 3,776 per
maltose in the AFFIRM-AHF trial, from weeks patient if they underwent pre-operative iron
4 to 24, corresponding with the treatment deficiency screening and treatment.13
period in the trial.
The World Health Organization has recog-
A second publication in June, investigated the nized that PBM plays a key role in optimizing
cost-effectiveness of ferric carboxymaltose clinical practice and included it as one of the
compared to placebo from the UK, US, Italian six strategic objectives in its 2020 action
and Swiss payer perspectives, based on the framework to advance universal access to safe
AFFIRM-AHF study. The results demonstrated blood. In order to implement this objective in
that ferric carboxymaltose was dominant in 2021, a group of experts are working on
both cost saving and in generating incremen- guidance for the implementation of PBM into
tal quality adjusted life years (QALY) outcomes healthcare systems. This is planned to be com-
in the UK, US and Switzerland, and well below pleted by the end of 2021 or early 2022. In
the acknowledged willingness to pay thresh- addition, the European Commission has
old in Italy. announced plans to revise the EU Blood
Directive in 2021 and has issued a consulta-
Vifor Pharma’s US partner American Regent, tion where a number of stakeholders have
Inc., is conducting one of the largest studies of highlighted the need to introduce the concept
i.v. iron in heart failure, the HEART-FID study7. of PBM within the directive.
HEART-FID is a double-blind, multi-center,
prospective, randomized, placebo-controlled In June 2021, an update to the "Clinical
study to assess the efficacy and safety of Practice Guidelines for Blood Conservation"
Injectafer® in the treatment of patients with was published by several global leading
heart failure with reduced ejection fraction in medical societies14, further strengthening the
iron deficiency patients. The study remains on importance of PBM in optimizing clinical
track for completion in 2022. practice for patients in cardiac surgery.
Patient Blood Management 8 Vifor Pharma Market research data 2020.
Patient blood management (PBM) is designed 9 Froessler B et al, Ann Surg 2016.
10 Froessler B et al, Ann Surg 2016.
to improve surgical and medical patient 11 Kotzé A et al, British Journal of Anaesthesia 2012.
outcomes by optimally managing and pre- 12 Basora M et al, Blood Transfus 2018.
13 Trentino et al, Anaesthesia 2020.
14 Society of Thoracic Surgeons (STS), Society of
7 Randomized Placebo-controlled Trial of FCM as Cardiovascular Anesthesiologists (SCA), American
Treatment for Heart Failure with Iron Deficiency Society of ExtraCorporeal Technology (AmSECT),
(HEART-FID). Duke Clinical Research Institute, Society for the Advancement of Blood Management
ClinicalTrials.gov (SABM)1.
20 Vifor Pharma Ltd. | Half-year Report 2021HIGHLIGHTS
PORTFOLIO
Among the most important updates to the M A LT O F E R ®
practice guideline is the change from blood
preservation to PBM, focusing on a multi-dis- Maltofer® is the originator oral iron polymalt-
ciplinary management approach for patients. ose complex (IPC) and plays an important role
in the management of patients with iron
In 2021, a Vifor Pharma real-world evidence deficiency. On the market since for over 50
study, analyzing the clinical and economic years, it has become a widely accepted and
impact of implementing PBM measures with well-tolerated therapy for infants, children,
OUTLOOK
Ferinject®, is ongoing. Approximately 31,000 adolescents and pregnant women. Registered
patients are enrolled from four European in 76 countries, Maltofer® is a leading oral iron
countries: UK, Germany, Italy and Spain. Vifor product in a fragmented market segment,
Pharma plans to generate substantial evidence with over 3,000 global suppliers.
to show the impact of Ferinject® on several
clinical and health economics and outcomes In the first half of 2021, net sales of Maltofer®
research (HEOR) indicators. Preliminary results increased by 15.0% compared to the prior
are expected at the end of 2021. year to CHF 36.1 million. This increase was
primarily driven by growth in Brazil and
G E O G R A P H I C E X PA N S I O N Mexico.
In line with its commitment to make life-im- In March 2021, Maltofer® became the number
pacting treatments available to patients one oral iron brand in Australia by value,
worldwide, Vifor Pharma is expanding its having been launched six years prior, in 2015.
geographical footprint in major pharmaceuti-
cal markets. In 2020, the company announced
a strategic partnership with Fresenius Kabi in
FINANCE
China, which has the world’s largest iron
deficiency population with estimated preva-
lence of 20%. Ferinject® approval in China is
expected in Q4 2021.
Vifor Pharma Ltd. | Half-year Report 2021 21IRON THERAPIES
OTHER IRON THERAPIES
V A M I F E P O R T ( V I T- 2 7 6 3 ) A phase-IIa trial in non-transfusion dependent
IN DEVELOPMENT beta-thalassemia (NTDT) is ongoing, with
expected completion of enrolment in H2 2021.
Vifor Pharma is using its expertise in under- The main objective of this randomized,
standing the chemistry and biology of iron to controlled, multinational trial is to investigate
develop vamifeport (VIT-2763), the first the safety, tolerability and efficacy of
ferroportin inhibitor with the potential to treat vamifeport in NTDT patients. Trial activities for
diseases characterized by ineffective erythro- a phase-IIb trial in transfusion dependent
poiesis and iron overload, such as beta-thalas- beta-thalassemia (TDT) have started, with the
semia or other hemoglobinopathies like sickle aim to begin recruitment in H2 2021. The main
cell disease (SCD). In these conditions, by objective of this randomized, controlled,
decreasing iron overload and correcting multinational trial is to identify the most
ineffective erythropoiesis in beta-thalassemia, efficacious and safe dose for future confirma-
or by decreasing hemolysis and vascular tory trials.
occlusions in SCD, iron control via ferroportin
inhibition may have a beneficial effect on the Vamifeport has also shown promising results
overall clinical outcome. in a preclinical model of SCD, a group of
inherited red blood cell disorders and genetic
Beta-thalassemia is an inherited rare blood condition present at birth. In this disease, red
disorder that reduces the production of blood cells carry abnormal hemoglobin, which
functional hemoglobin in red blood cells, makes them prone to deform (sickle) and
which can lead to a lack of oxygen in many rupture. This promotes obstruction of the
parts of the body and potentially cause blood vessels which can prevent oxygen
anemia. Beta-thalassemia is often treated with supply to tissues and cause injury and/or pain
blood transfusions, which may lead to excess crisis. There are an estimated 150,000 patients
levels of iron in the body, known as iron in both Europe and the US living with the
overload. The condition is estimated to affect condition.
around 25,000 patients in both the US and
Europe. Both the FDA and the EMA granted Both the FDA and the EMA granted orphan
orphan drug designations for vamifeport in drug designation for vamifeport in SCD in
beta-thalassemia in 2019. Q1 2021. A phase-IIa trial in SCD has been
initiated.
22 Vifor Pharma Ltd. | Half-year Report 2021HIGHLIGHTS
PORTFOLIO
OUTLOOK
FINANCE
Vifor Pharma Ltd. | Half-year Report 2021 23OUR PORTFOLIO
N E P H R O LO GY
OWN PRODUCTS
Chronic kidney disease
(CKD) is relatively
common among adults,
with prevalence of up to
13%15 and rising as the
population ages. Vifor
Pharma strives to improve
lives of people suffering
from kidney diseases,
IN-LICENSED PRODUCTS AND *PIPELINE
through our broad,
innovative nephrology
portfolio and
individualized solutions.
Rayaldee®
Vadadustat*
Difelikefalin*
Avacopan*
ANG-3777*
24 Vifor Pharma Ltd. | Half-year Report 2021HIGHLIGHTS
Our diversified nephrology
portfolio is built around five
primary disease areas, focusing
on distinct comorbidities and
PORTFOLIO
complications in CKD patients.
This includes managing
complications for CKD patients
and CKD patients on dialysis,
preserving renal function, acute
kidney injury and transplantation.
OUTLOOK
N E P H R O LO GY Our expanding presence in the global
nephrology market is primarily built around
People living with kidney disease face a long Vifor Fresenius Medical Care Renal Pharma
and difficult journey associated with multiple (VFMCRP), a unique and powerful joint
and varying conditions and complications that company providing innovative therapies in
severely impact quality of life and life expec- combination with patient management
tancy. Through an innovative nephrology systems to ensure best outcomes for patients.
portfolio and individualized renal care For the last ten years, the joint company has
solutions, Vifor Pharma aims to preserve renal combined Vifor Pharma’s expertise in pharma-
function, manage complications and improve ceuticals with the skills and infrastructure of
quality of life for CKD patients. We partner Fresenius Medical Care – a global leader in
with specialists to improve the lives of patients dialysis clinics and services. Fresenius Medical
with high unmet medical need across all Care has access to around 350,000 patients in
stages of kidney disease, from early stage to its global network of over 4,000 dialysis clinics
post transplantation. 15 and is the world’s leading provider of prod-
ucts and services for people with chronic
FINANCE
kidney failure. A distinctive model makes
VFMCRP a highly attractive commercial
15 1. Hill NR et al PLOS ONE, DOI:10.1371/journal.
partner for companies with innovative ne-
pone.0158765. phrology therapies.
Vifor Pharma Ltd. | Half-year Report 2021 25N E P H R O LO GY
Through our innovative nephrology
portfolio and individualized solutions we
aim to preserve renal function, manage
complications and improve quality of life.
We partner with specialists to improve lives of
patients with high unmet medical needs across
all stages of kidney disease, from early stage
to post transplantation.
PRESERVE RENAL FUNCTION
* *
ACUTE KID NEY
K I DN EY P R OTECTI O N ANCA-A S S OCIATED VA S CULITIS INJURY
M A N AG E C O M P L I C AT I O N S - C K D M A N A G E C O M P L I C AT I O N S - D I A LY S I S
* *
H Y P ER KAL EM I A C K D-MBD I RO N DEFI C I ENCY IRON D EFICIENCY HYPERKA LEMIA CKD - A S S OC IAT E D
/ ANEMI A / A NEMIA P R UR IT US
MANAGE ACUTE KIDNEY INJURY I M P R O V E T R A N S P L A N TAT I O N
* *
AC UTE K I DNEY D ELAYED G RA FT
I NJ URY FUNCTION
* P I PE LI N E
26 Vifor Pharma Ltd. | Half-year Report 2021S TAG E 3
S TAG E 4
Vifor Pharma Ltd. | Half-year Report 2021
S TA G E 5 N O N - D I A LY S I S
S TA G E 5 D I A LY S I S
T R A N S P L A N TAT I O N
27
FINANCE OUTLOOK PORTFOLIO HIGHLIGHTSN E P H R O LO GY 28 Vifor Pharma Ltd. | Half-year Report 2021
HIGHLIGHTS
MIRCERA® SUPPLIED TO
3,800
DIALYSIS CLINICS IN THE US
PORTFOLIO
Our diversified nephrology portfolio is built Mircera® net sales were CHF 218.9 million,
around five primary disease areas, focusing on down –18.5% compared to the prior year
distinct comorbidities and complications in period (or –12.6% at CER). In addition to
chronic kidney disease (CKD) patients. This negative foreign exchange effects, Mircera®
includes managing complications for CKD performance was mainly driven by phasing
patients and CKD patients on dialysis, pre- impacts, lower volumes resulting from
serving renal function, acute kidney injury and an excess mortality rate in the dialysis seg-
transplantation. ment due to COVID-19, and pricing adjust-
ments in the mid-size and independent
The portfolio of products, which encompasses dialysis market in the US.
the five primary disease areas, is detailed
below: R E TAC R I T ®
Retacrit® (epoetin alfa-epbx) is a short-acting
C O M P L I C AT I O N S I N C K D PAT I E N T S ESA approved by the US FDA in May 2018 for
A N D C K D PAT I E N T S O N D I A LY S I S all indications of its reference drug, epoetin
RENAL ANEMIA MANAGEMENT alfa. It is the first and only biosimilar ESA
approved for use in the US. Vifor Pharma
OUTLOOK
E R Y T H R O P O I E S I S - S T I M U L AT I N G licensed rights from Pfizer Inc. in 2015 to
AGENT (ESA) PORTFOLIO commercialize Retacrit® in certain channels,
primarily comprising the US non-hospital
Vifor Fresenius Medical Care Renal Pharma dialysis market and nephrology office prac
(VFMCRP) commercializes products for the tices.
treatment of anemia in patients with CKD. The
joint company’s ESA portfolio includes both Vifor Pharma’s net sales of Retacrit® totaled
short-acting and long-acting ESAs to offer CHF 27.5 million, up 14.1% compared to the
customers a full range of treatment options to prior year period (or 22.1% at CER). This
best support patient needs. growth was due to increased adoption of the
biosimilar across the existing market, the
addition of new customer accounts, and
M I R C E R A® incremental growth in existing accounts due
to increased demand for a multiple-dose
Mircera® (methoxy polyethylene glycol-epoe- presentation, first launched in late Q4 2020.
tin beta) is a long-acting ESA licensed from
F. Hoffmann-La Roche AG since 2015 to treat
FINANCE
symptomatic anemia associated with chronic
kidney disease. Mircera® is currently supplied
to over 3,800 dialysis clinics in the US and its
territories.
Vifor Pharma Ltd. | Half-year Report 2021 29N E P H R O LO GY
VENOFER®
Venofer® is the originator intravenous (i.v.) iron
sucrose product. A wealth of clinical data and
clinical experience make Venofer® the trusted
and preferred iron sucrose treatment in iron
#
1 VENOFER® IS A
LEADING I.V. IRON
BRAND IN TERMS OF
VOLUME WORLDWIDE
therapy for anemic dialysis patients. In the first
half of 2021, Venofer® maintained its position
as a leading i.v. iron brand in terms of volume
worldwide, with over 30 million patient years PAT I E N T S
30
of experience by the end of June 2021.
Reported net sales of Venofer® decreased
–1.5% (up 2.7% at CER) to CHF 67.3 million in
H1 2021. Sales were primarily driven by contin-
ued stronger demand in hospitals in the US,
MILLION
where it is a leading i.v. iron therapy in PATIENT YEARS OF EXPERIENCE
hemodialysis. (APPROX.)
VA DA D U S TAT
IN DEVELOPMENT
Vadadustat is an investigational oral hypox- patients on dialysis and adult patients not on
ia-inducible factor (HIF) prolyl hydroxylase dialysis. The FDA has assigned a Prescription
inhibitor developed by Akebia Therapeutics, Drug User Fee Act (PDUFA) target action date
Inc., a NASDAQ-listed, US bio-pharmaceutical of March 29, 2022. Pursuant to the License
company. The New Drug Application (NDA) Agreement with Akebia Therapeutics, Vifor
for vadadustat for the treatment of anemia Pharma does not have rights to sell vadadu-
due to chronic kidney disease (CKD) is under stat, subject to FDA approval, outside of the
review by the US Food and Drug Administra- US dialysis settings referenced above.
tion (FDA).
Vifor Pharma has been granted an exclusive MINERAL AND BONE DISEASE
license to sell vadadustat to Fresenius Kidney MANAGEMENT
Care dialysis centers in the US and to certain
third-party dialysis organizations that together VELPHORO®
account for up to 60% of the dialysis patients
in the US. The license is subject to vadadu- Velphoro® is a non-calcium, iron-based,
stat’s approval by the US FDA, the earlier of chewable phosphate binder approved for the
a determination by the Centers for Medicare & control of phosphate levels in the blood in
Medicaid Services (CMS) that vadadustat will adults with chronic kidney disease (CKD) on
be included in the End-Stage Renal Disease dialysis. Velphoro® is launched in 31 countries,
Prospective Payment System or that vadadu- and maintains a leading position by sales
stat will be reimbursed using the Transitional value16 in the phosphate binder market with
Drug Add-on Payment Adjustment (TDAPA), a 31.7% market share as of April 2021.
and a milestone payment from Vifor Pharma.
In June 2021, Akebia announced that the FDA
16 Unlike volume data, value data are unrepresentative of
accepted NDA filing for vadadustat for the competitive dynamics because they sum different
treatment of anemia due to CKD in both adult types of prices for different medicines
30 Vifor Pharma Ltd. | Half-year Report 2021HIGHLIGHTS
In H1 2021, net sales of Velphoro® increased
by 8.1% (or 13.6% at CER) to CHF 79.9 million,
from CHF 73.9 million in the same prior year
period. Growth was mainly driven by the US
and Japan.
#
1 VELPHORO® HAS A
LEADING POSITION IN THE
CALCIUM-FREE PHOSPHATE
BINDER MARKET
Growing real-world evidence continues to
demonstrate the benefits of Velphoro® for
patients. In February 2021, the results of the
31.7%
European Post Authorization Safety Study
VERIFIE were published in the “Clinical Kidney
Journal”17, confirming the safety and effective-
PORTFOLIO
ness of Velphoro® in a real-life setting by
doubling the patients with controlled phos-
phate levels 12 months after initiation.
VELPHORO® MARKET SHARE AS OF
In April 2021, Vifor Fresenius Medical Care APRIL 2021
Renal Pharma (VFMCRP) announced positive
results from a phase-III study in China (PA-CL-
CHINA-01). The study evaluated the efficacy
of Velphoro® (PA21) compared to sevelamer
carbonate in lowering and maintaining serum
phosphorus in adult Chinese patients with
CKD on dialysis after 12 weeks of treatment.
The study met its primary endpoint, demon-
strating non-inferiority versus sevelamer
REAL-WORLD
carbonate in the change from baseline in
serum phosphorus levels at week 12.
EVIDENCE
This study reinforces the potential of DEMONSTRATES THE BENEFITS
OUTLOOK
OF VELPHORO® FOR PATIENTS
Velphoro® as an important new treatment
option for dialysis patients with hyperphos-
phatemia in China. VFMCRP intends to submit
the Chinese New Drug Application to the
National Medical Products Administration’s
Center for Drug Evaluation at the end of 2021.
In the US, an additional 6-month pediatric
exclusivity has been granted on all Orange
Book listed patents. So far, no FDA approval
has been issued for any generic copy.
In the European Union, Velphoro® received
an extension of the indication for use in
pediatric patients.
FINANCE
17 Clinical Kidney Journal, sfaa211,
https://doi.org/10.1093/ckj/sfaa211
Vifor Pharma Ltd. | Half-year Report 2021 31N E P H R O LO GY
R AYA L D E E ® It is developed as a treatment for chronic
kidney disease-associated pruritus (CKD-aP)
Rayaldee® is the first and only oral extended and if approved, would be the first treatment
release18 formulation of calcifediol (ERC), specifically indicated for this condition in the
a pro-hormone of the active form of vitamin US and Europe.
D3, for the treatment of secondary hyperpara-
thyroidism (SHPT) in patients with chronic Moderate-to-severe CKD-aP is an intractable
kidney disease (CKD) with vitamin D insuffi- systemic itch condition that impacts up to 40%
ciency. SHPT is a chronic progressive disease of dialysis patients around the world.20
which increases in severity as CKD worsens. It Moderate-to-severe chronic pruritus has
is estimated to affect between 40% and 82% repeatedly been shown to be associated with
of patients with stage three or four CKD19, and decreased quality of life, poor sleep quality,
requires early and effective control to avoid and depression.21 CKD-aP is also associated
severe bone and cardiovascular complica- with an increased risk of hospitalization, and is
tions. an independent predictor of mortality among
hemodialysis patients22. There are no ap-
There is currently no established standard proved therapies in Europe or the US for the
of care for the treatment of SHPT in non-dia treatment of CKD-aP.
lysis (ND-CKD) patients across Europe.
Vifor Fresenius Medical Care Renal Pharma Vifor Fresenius Medical Care Renal Pharma
( VFMCRP) has obtained the rights from OPKO (VFMCRP) and Cara Therapeutics have
Health Inc. for this indication in Europe and a license agreement to develop and commer-
selected markets outside the US. cialize difelikefalin injection for the treatment
of CKD-aP in hemodialysis and peritoneal
Rayaldee® has received marketing authoriza- dialysis patients worldwide, excluding the US,
tion in all European countries applied for, Japan and South Korea. In the US, the compa-
including Switzerland. In preparation for nies agreed to promote difelikefalin to
supply readiness, VFMCRP is working closely Fresenius Medical Care North America
with the supplier to produce Rayaldee® for (FMCNA) dialysis clinics – which account for
European markets. First launch of Rayaldee® is about 34% of all dialysis patients in the US,
expected in Germany and Switzerland in 2022. under a profit-sharing arrangement.
Vifor Pharma and Cara Therapeutics signed
CHRONIC KIDNEY DISEASE a license agreement for the commercialization
A S S O C I A T E D P R U R I T U S of difelikefalin injection for the treatment of
CKD-aP in the US dialysis market for non-Fre-
D I F E L I K E FA L I N senius Medical Care clinics under a prof-
IN DEVELOPMENT it-sharing arrangement in H2 2020. This will
allow Vifor Pharma to expand its nephrology
Difelikefalin, developed by Cara Therapeutics presence in the US, to reach more dialysis
Inc., is a peripherally restricted kappa opioid patients in need of treatment.
receptor (KOR) agonist, targeting KORs on
peripheral sensory neurons and immune cells.
20 Rayner HC, et al. Clin J Am Soc Nephrol 2017;12:2000–7.
18 Rayaldee® is approved with the terminology “extended- 21 Ramakrishnan K, et al. Clinical characteristics and
release” in the US and as “prolonged-release” in the EU. outcomes of end-stage renal disease patients with
19 Levin, A., Bakris, G. L., Molitch, M., Smulders, M., Tian, self-reported pruritus symptoms. International Journal
J., Williams, L. A.& Andress, D. L. (2007). Prevalence of of Nephrology and Renovascular Disease. 2014; 7: 1-12.
abnormal serum vitamin D, PTH, calcium, and 22 Sukul N. et al. Self-reported Pruritus and Clinical,
phosphorus in patients with chronic kidney disease: Dialysis-Related, and Patient-Reported Outcomes in
Results of the study to evaluate early kidney disease. Hemodialysis Patients. Kidney Med. 2020 Nov
Kidney International, 71(1), 31–38. 21;3(1):42-53.
32 Vifor Pharma Ltd. | Half-year Report 2021HIGHLIGHTS
PORTFOLIO
In March 2021, Vifor Pharma and Cara Thera- The submission for registration in Switzerland,
peutics announced that the US Food and Drug Australia and Canada through Access Consor-
Administration (FDA) had accepted and tium filing is planned for H2 2021, with
granted Priority Review for the New Drug approval expected in H2 2022.
Application (NDA) of difelikefalin solution for
injection, for the treatment of moderate-to-se- The filings are based on positive data from
vere pruritus in hemodialysis patients. The two pivotal phase-III trials, KALM-1 – which
PDUFA target action date for difelikefalin is was conducted in the US and published in the
23 August 2021, with the FDA stating that it New England Journal of Medicine in 2020,
does not plan to hold an advisory committee and the global KALM-2 study. The registration
meeting to discuss the application. The FDA dossier also includes supportive data from
OUTLOOK
has conditionally accepted KORSUVA™ as the an additional 32 clinical studies. If approved,
trade name for difelikefalin injection. difelikefalin would be the first medicine
specifically indicated for the treatment of
VFMCRP and Cara Therapeutics announced in CKD-aP in the US and Europe.
March 2021, that the European Medicines
Agency (EMA) had accepted to review the Launch preparations in the US and Europe
Marketing Authorization Application (MAA) have been initiated, with a focus on disease
for difelikefalin injection for the treatment of education and stakeholder engagement to
pruritus associated with chronic kidney ensure awareness of the unmet medical need
disease in hemodialysis patients. The EMA will and burden of disease. VFMCRP is also
review the application under the centralized investigating how the quality of life of dialysis
marketing authorization procedure. If ap- patients with CKD-aP can be improved,
proved, difelikefalin would receive marketing including diagnosis, treatment and evaluation
authorization in all member states of the of outcome measures.
European Union (EU), as well as in Iceland,
Liechtenstein and Norway. The EMA’s decision In April 2021, VFMCRP signed an out-licensing
on the EU MAA is expected in H1 2022. agreement with Otsuka Canada regarding the
FINANCE
Subject to approval, first launches in Europe promotion and distribution of difelikefalin
are expected in 2022. injection in Canada. Additional partnerships
in further key markets, including China, are
under discussion with third parties.
Vifor Pharma Ltd. | Half-year Report 2021 33You can also read
