UPDATE: BioMarin's Advisory Committee meeting did not appear to go very well for the company. Despite testimony from dozens of public speakers such as patients and advocates begging the FDA to approve drisapersen, the independent advisers were still simply not swayed by BioMarin's trial data.
While the advisers did not have an up-or-down vote on whether the FDA should recommend the drug, they did have a series of votes on whether BioMarin's trial data were persuasive. The committee member's votes on those questions tended to range from negative to neutral at best.
The FDA could still potentially approve the drug, given that there is no available therapy for Duchenne and that the agency doesn't have to follow the advisory committee's advice. But things are not looking good at this juncture.
BioMarin competitor Sarepta has a similar meeting for its own Duchenne candidate coming up in the winter, and is certain to be looking back at Tuesday's meeting to better prepare for some potentially intense criticism. However, the company also likely has an advantage as it appears its drug has a better safety and efficacy profile.
- After a series of setbacks, including a highly critical and discouraging letter posted on the FDA's site on Friday, an FDA advisory panel will be voting on whether to approve BioMarin's drisapersen for treatment of Duchenne Muscular Dystrophy (DMD) next week (UPDATED ABOVE).
- DMD is a rare genetic disease, which mainly affects young men. Because the body does not produce dystrophin in those affected by DMD, muscle tissue does not function properly, the body weakens and eventually the patient is paralyzed and dies. Most patients die before age 30.
- There have been many setbacks for BioMarin in the face of the FDA's criticism of the small size of their clinical trials. There has also been a great deal of support from DMD advocates, mainly parents and the DMD treatment community.
There are a lot of people out there, from BioMarin exces, to researchers, parents of boys with DMD, physicians, and advocates, who are hopeful about this drug. But a letter posted on the FDA's site this morning has dampened enthusiasm for a possible approval.
In the letter, the FDA said that efficacy data from three clinical trials "does not reach the level of substantial evidence" necessary for approval. Moreover, the letter also references "severe toxicity across many organ systems."
This meeting would probably not be happening if it were not for last June's new guidance on DMD clinical trials. The new guidance takes into consideration increased risk tolerance among some DMD patients involved in trials. Acceptable endpoints may now include functional, cardiac, or respiratory metrics, though the FDA has asserted that the ideal trial design still involves a randomized, placebo-controlled design—even if the placebo-controlled part involves using historical data.
In a previous phase 2 trial drisapersen failed; however, there were also previous studies showing positive drisapersen-related outcomes---but the FDA deemed them too small. Now there is data from a placebo-controlled, phase 2 trial with positive outcomes. BioMarin is hoping this data will convince the panel to approve the drug.
And then there is the issue of Sarepta Pharmaceuticals' competitor drug for DMD, which was once the frontrunner in this race, but failed to convince the FDA that its data was sufficient for approval. It's not clear what will happen next week, but many hope that the FDA will approve the drug anyway, with the idea that anything in terms of better functionality and physical well-being for DMD patients, who have nothing, provides something worthwhile.
It should be noted that the FDA's letter should not automatically lead to the foregone conclusion of a rejection by the panel, but truthfully it is not encouraging. This question comes to mind: If the FDA can approve Addyi, why not approve drisapersen?