Zolgensma Approval and Access June 2019
After 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 27 sites consented AR - Arkansas Children’s Hospital CA - Rady Children’s Hospital - UCLA 19 states CT - Yale Pediatric Neuromuscular Clinic FL - Nemours Childrens Hospital IA - University of Iowa Stead Childrnens Hospital IL - Ann and Robert Luri Children’s Hospital of Chicago - Updated daily MA - Boston Children’s Hospital MD - Johns Hopkins Hospital MN - University of MN MO - Washington University/St. Louis Children’s Hospital - Children’s Mercy Hospital NC - Duke University Medical Center Goal is 50 to 100 sites NY - University of Rochester - SUNY Downstate Medical Center OH - Nationwide Children’s Hospital - 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.org
FDA-Approved Label: INDICATIONS AND USAGE ZOLGENSMA: -IV delivery AAV9 vector-based gene therapy Approved for: • patients <2 years of age at dosing with SMA verified by gene mutations or deletion in both survival motor neuron 1 ( SMN1) genes • All types and all SMN2 copy numbers
FDA-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 infants
Trial 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 SMN2
Insurance 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 scope
Timing 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 timing
FDA-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.
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