What does the Approval of Sarepta’s Treatment for Duchenne Muscular Dystrophy Foreshadow?

Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle degeneration and weakness, starting in early childhood – most individuals diagnosed with DMD don’t live beyond their teenage years without intervention. An FDA ad comm meeting, held to review and evaluate data concerning the safety and effectiveness of investigational and marketed therapies, met earlier this year to evaluate Sarepta’s SRP-9001 DMD drug candidate. For many–including those outside of the DMD community–the meeting was much anticipated. This ad comm is the first for a DMD therapy since 2016. Around the time of the original ad comm, Syneos Health® provided a perspective on the FDA ad comm's narrow positive vote in favor of Sarepta’s gene therapy candidate. On June 22, 2023, the moment many have been eagerly awaiting occurred–the FDA approved Sarepta's Elevidys for DMD¹.   

Response in the Media  

The prevailing media soundbite on the approval was "first gene therapy for Duchenne Muscular Dystrophy⁴” with a reoccurring theme surrounding the $3.2 million price tag for the treatment, making it the second-most expensive drug in the world.³ Of note, top trade outlets almost always paired the mention of price with an acknowledgment of the high unmet need for this incurable disease, paired with a heart-warming vignette of a patient who had received the treatment during clinical trials or quote from a passionate patient advocacy organization.  

Outside of pricing and unmet need, articles expressed a number of concerns, including: 

• Does Elevidys really work? Elevidys is the first gene therapy approved via the FDAs accelerated approval pathway.  The accelerated approval was based on a surrogate marker. The drug will need to prove in an ongoing clinical trial that it actually improves physical function and mobility in patients in order to stay on the market 
• Elevidys’ narrow indication - limited to boys ages 4 to 5 years old (representing about 6% of the population diagnosed with DMD). “Patient advocacy and KOLs have some concerns about this¹,” according to notes from the ad comm meeting, yet one analyst still believes peak sales of $3 billion will be achieved. 
• Once treated with Elevidys, a patient can't receive another gene therapy⁶ and will likely be unable to participate in clinical trials for other kinds of treatment 

Elevidys Approvals: Implications for the Life Science Industry 

Although this approval shows the willingness of the FDA to approve gene therapies based on a surrogate marker via accelerated approval, expect eyes to be on progress and results of confirmatory trials. Communicating and reinforcing value factors to external stakeholders  before  approval is a critical investment to mitigate pricing criticism at approval. As seen with this approval, reporters captured statements and quotes around these various factors (such as high unmet need and the potential to restore mobility).  

Looking ahead, as the number of cell and gene therapy approvals increase⁵, the argument that these therapies will have a minimal budget impact will need to be revisited and adjusted. Life science companies that do the work ahead of time to quantify the holistic and positive impact of their innovation will be at less risk of getting trapped in the “budget impact” argument.  

Check out additional recent Syneos Health Reputation & Risk Management perspectives on related topics and learn more about Syneos Health on Insights Hub.  

References 

1. Center for Biologics Evaluation and Research. 2023 meeting materials, CTGTAC. U.S. Food and Drug Administration.  https://www.fda.gov/advisory-committees/cellular-tissue-and-gene-therapies-advisory-committee/2023-meeting-materials-cellular-tissue-and-gene-therapies-advisory-committee.  

2. Fidler B. Sarepta prices Duchenne gene therapy at $3.2M. BioPharma Dive. June 22, 2023.  https://www.biopharmadive.com/news/sarepta-duchenne-elevidys-price-million-gene-therapy/653720/.  

3. Perrone M. First gene therapy for deadly form of muscular dystrophy gets FDA approval for Young Kids. AP News. June 22, 2023. https://apnews.com/article/fda-gene-therapy-duchenne-muscular-dystrophy-approval-82a82cfb67ef9fb6cf75cd3b5865c138.  

4. Tirrell M. FDA approves $3.2 million gene therapy for Rare muscular dystrophy in kids ages 4 and 5. CNN. June 22, 2023. https://www.cnn.com/2023/06/22/health/fda-sarepta-muscular-dystrophy/index.html. 

5. The age of cell and gene therapy is here. Charles River. https://www.criver.com/eureka/age-cell-and-gene-therapy here#:~:text=By%20the%20end%20of%202022,and%20In%20Vivo%20Pharma%20Intelligence 

6. Miller H. The FDA’s approval of a new gene therapy for Duchenne Muscular Dystrophy won’t help me - but it gives me hope. STAT. June 22, 2023.  https://www.statnews.com/2023/06/22/sarepta-duchenne-muscular-dystrophy-genetic-therapy/?utm_campaign=rss.