Zolgensma Approval and Access June 2019 - Cure SMA
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Zolgensma Approval and Access
June 2019After Approval - Access 1. Key Issues: A. Sites and capacity B. Insurance coverage C. Data 2. Broader Approval: A. Label vs Trials B. FDA did more than usual again (extrapolated for SMA) 3. Rules and Regulations: A. Who can say what B. US Only C. Combinations and sequential (more at conferences)
Public Treatment Sites
AL - Children’s of Alabama
AR
CA
- Arkansas Children’s Hospital
- Rady Children’s Hospital
- UCLA
27 sites consented
CT
FL
IA
- Yale Pediatric Neuromuscular Clinic
- Nemours Childrens Hospital
- University of Iowa Stead Childrnens Hospital
19 states
IL
MA
MD
- Ann and Robert Luri Children’s Hospital of Chicago
- Boston Children’s Hospital
- Johns Hopkins Hospital
- Updated daily
MN - University of MN
MO - Washington University/St. Louis Children’s Hospital
- Children’s Mercy Hospital
NC - Duke University Medical Center
NY
OH
- University of Rochester
- SUNY Downstate Medical Center
- Nationwide Children’s Hospital
Goal is 50 to 100 sites
- Cincinnati Children’s Hospital
- Rainbow Babies and Children’s Hospital/University Hospitals of Cleveland
OR - OHSU Doernbecher Children’s Hospital
PA - Children’s Hospital of Philadelphia, Buerger Center for Advanced Pediatric Care
- Clinic for Special Children
TX - University of Texas Southwestern/Children’s Health
- Texas Children’s Hospital
UT - University of Utah/Primary Children’s Hospital
VA - Children’s Hospital of the King’s Daughters.
http://www.curesma.org/zolgensma-administration-sites.html
treatments@curesma.orgFDA-Approved Label:
INDICATIONS AND USAGE
ZOLGENSMA:
-IV delivery AAV9 vector-based gene therapy
Approved for:
• patientsFDA-Approved Label:
INDICATIONS AND USAGE
Limitations of ZOLGENSMA use:
1. Repeat dosing of ZOLGENSMA has not been
evaluated
2. The use of ZOLGENSMA in patients with advanced
SMA (e.g., complete paralysis of limbs, permanent
ventilator dependence) has not been evaluated
3. ZOLGENSMA should not be used in premature
infants due to neurologic developmental concerns
related to using corticosteroids in premature infantsTrial vs Label vs Policy
-FDA label includes:
• < 2 years of age at dosing
• All copy numbers of SMN2
• All types
-Insurance
Coverage
Policies
?
-IV Clinical trials included:
• < 9 months of age at dosing
• Two copies of SMN2Insurance Coverage
Publicly Available Policies
Covers if prior
Coverage up to Coverage for Spinraza Percentage of
Payer Type 0 Type I Type II Type III Type IV 1 SMN2 2 SMN2 3 SMN2 4 SMN2 2 years advanced SMA treatment Coverage
Commercial 0%
Aetna 0%
Anthem 0%
Cigna 0%
HCSC (BCBS) 0%
BCBS MI 0%
Humana 0%
Molina 0%
UnitedHealth No Yes No No No Yes Yes Yes No No No yes 38%
Wellcare 0%
Medicaid 0%
AL 0%
AK 0%
AZ 0%
AR 0%
CA 0%
CO 0%
CT 0%
DE 0%
FL 0%
GA 0%
HI 0%
ID 0%
IL 0%
IN 0%
IA 0%
KS 0%
KY 0%
LA 0%
ME 0%
MD 0%
MA 0%
MI 0%
MN 0%
MS 0%
MO 0%Future Steps for Gene Therapy
1. Intrathecal (IT) delivery for older and bigger patients:
A. Differences to IV
B. Current Clinical Trial: Study of Intrathecal Administration of
AVXS-101 for Spinal Muscular Atrophy:
1) Clinicaltrials.gov Identifier: NCT03381729
2) Also known as STRONG
3) Phase1: 27 subjects at 11 sites in the US
4) 6 months to 60 months of age and three copies of SMN2 and symptoms < 12
months of age
5) Sit independently and not standing or walking independently
6) Three different dose cohorts
a. Currently enrolling for highest dose cohort C
7) Timing and FDA.
C. Potentially additional trials
D. Data for FDA and Community and Coverage
E. Potential timing and scopeTiming and Waiting
1. Evaluate and discuss the following with your neurologist:
A. All available safety and efficacy data
B. Method of administration
C. Practical and process timing issues
2. Cure SMA recommends not waiting for a future treatment or
trial option if other options are available now:
A. Earliest possible administration of SMN enhancing drugs yields
best results
B. Timing is one of the biggest factors in degree of response to SMN
upregulation
3. Additional testing prior to dosing and insurance process will also
factor into timingFDA-Approved Label:
INDICATIONS AND USAGE
ACUTE SERIOUS LIVER INJURY
Acute serious liver injury and elevated aminotransferases can
occur with ZOLGENSMA.
• Every child considered for ZOLGENSMA must have the
following blood tests prior to treatment:
• AAV antibodies
• Liver function tests
• Platelet count
• Troponin-I
• ZOLGENSMA may be delayed or may not be given if any
of these results are abnormal.SMA and Care and Monitoring • Continue close follow up by an experienced healthcare provider: • Monitor for symptoms of SMA • Monitor for side effects of treatments • Optimize the benefits of treatment • Discuss new information about SMA
FDA-Approved Label:
INDICATIONS AND USAGE
• After treatment, blood tests are repeated as follows:
• Liver function tests – weekly x 1 month, then every 2
weeks x 2 months until results normalize
• Platelet count – weekly x 1 month, then every 2
weeks x 2 months
• Troponin-I - weekly x 1 month, then monthly x 2
months
• At 30 days, if liver function tests are abnormal the
prednisolone course is extended. See package
insert. Prednisolone dose must be tapered.Options and Choices 1. A or B (A not B) 2. A then B (B then A) 3. A and B Considerations: • Data whether benefits and what risks: • Trials, Anecdotes, Real World Evidence • Important to • Community, and • Coverage • More complicated when a treatment is permanent • Combination risks
Resources
• General Information:
– www.cureSMA.org/Zolgensma
• Administration Site List:
– www.cureSMA.org/ZolgensmaSites
• Choice and Connection to Care (insurance information*):
– http://www.curesma.org/support-care/care-
publications/
• Questions on Zolgensma or other treatment-related
inquiries:
– Email treatments@curesma.org
*Choice and Connection to Care provides general information on choosing and navigating
insurance coverage.You can also read
