Back in mid-May, we provided our POV around the FDA ad comm's narrow positive vote in favor of Sarepta’s gene therapy candidate for Duchenne Muscular Dystrophy (DMD). On June 22nd, the moment many have been eagerly awaiting occurred ---the FDA approved Sarepta's Elevidys for DMD.  As anticipated, the approval has received coverage by all usual trade outlets (STAT, Reuters, etc.) and mass media (CNN, NPR, WSJ).

Media’s Main Soundbite

The prevailing soundbite across media was "first gene therapy for Duchenne Muscular Dystrophy". The majority of media coverage didn’t waste any time calling out the list price for this one-time treatment, $3.2 million (a few articles within the headline). This was usually followed by some commentary (typically, that it represents the second most expensive drug in the world now after Hemgenix).

Of note, there has not been strong criticism of the price among top tier or top trade outlets. Many media outlets are including comments that contextualize the price:

  • CNN explained that the cost is not unusual for a gene therapy: “While eye-popping, such a price tag isn’t out of step with other one-time gene therapies.”
  • AP News tried to explain that the list price is not what patients will pay: “Like most medicines in the U.S., the cost will be mostly paid by insurers — not patients — including private plans and government programs.”
  • BioPharma Dive even quoted Sarepta CEO Dough Ingram: “The price reflected a “conservative” approach to valuing the benefit to patients and their families of Elevidys, which Sarepta claims would be cost-effective at prices between $5 million and $13 million.”

Mention of price was almost always paired with an acknowledgment of the high unmet need for this incurable muscle disease that largely affects boys. Many times, this was paired with a heart-warming patient vignette of a boy who had received the treatment during clinical trials or a quote from a passionate patient advocacy organization (once again highlighting the influence of patient advocacy).

Lingering Concerns:  Efficacy, Narrow Indication, Clinical Trial Participation

Outside of pricing and unmet need, there was some concern expressed about:

  • 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. Patient advocacy and KOLs have some concerns about this, yet one analyst still believes peak sales of $3 billion will be achieved.
  • Once treated with Elevidys, a patient can't receive another gene therapy and will likely be unable to participate in clinical trials for other kinds of treatment. 

Implications for the Life Sciences 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 increase, 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.

Authored by Leigh Ann Bruhn and Kate O'Driscoll

About the Author:

We are a team of healthcare communicators, policy-shapers and crisis response specialists. Drawing upon professional experiences from Congress, CMS, HHS, hospitals, and health technology—and our collective work in rare disease, oncology, diabetes, gene therapy, pain management and infectious disease—we provide unique solutions to the evolving messaging challenges in today’s healthcare industry. We support our clients with evidence-based approaches to preventing pricing pushback, protecting brands from modern activism, establishing and communicating clear policies surrounding expanded access to medicines, and a proactive approach to value frameworks. Our offerings also include product safety, litigation, regulatory risks, ex-U.S. considerations and policymaker investigations.