Nancy Bradish Myers, President

Today the FDA granted its first-ever approval for a Duchenne Muscular Dystrophy (DMD) treatment; Sarepta received Accelerated Approval for its drug Exondys 51 (eteplirsen) to treat a subset of boys with DMD.

The agency’s move caps off years of back and forth between FDA reviewers, the company and patient advocates. As with any Accelerated Approval, in order to gain full FDA approval, the company must verify the product’s clinical benefit in confirmatory trials.

My initial thought is that clearly, this is a triumph for patient advocates and the parents of boys with DMD, who have been facing dire unmet medical needs and clamoring for treatment options.

What are the other takeaways from this controversial product review?  A few topline thoughts:

  • This is a megaphone moment for FDA; the agency is signaling that it’s willing to consider patient preferences seriously.
  • This decision is a clear example of FDA actually using the regulatory flexibility it has touted; it demonstrates that FDA is indeed approaching orphan drugs for life threatening diseases on a case-by-case basis, and that it’s willing to accept very small data sets when warranted.
  • This move is likely going to send a reverberation through the industry: it may change how companies and patients interested in developing orphan products approach the FDA.
  • It would not surprise me if it sends a number of orphan drug developers into meetings to explore what is the smallest sample size FDA will need to grant an approval. In this case, FDA seemed to consider the circumstances and the extreme unmet need as well as the science.
  • Clearly not every orphan drug will follow this path, but it will be studied by strategists to see if any learnings can be gleaned for their rare disease drug development playbook.