FDA usually does not make comments on pending new drug applications (NDAs) nor do they usually publicly discuss conversations with sponsors. However, in this instance, FDA has made a rather interesting disclosure relative to a pending NDA for a serious and devastating disease, Duchenne Muscular Dystrophy (DMD). See FDA’s full statement here. However, FDA says it “is addressing questions the agency has received from DMD patients, their families, and others in the community who are concerned about the timing of the filing of an NDA for eteplirsen.”
FDA notes that they recognize “the unmet medical need in Duchenne muscular dystrophy (DMD), the devastating nature of the disease for patients and their families, and the urgency to make new treatments available. We remain committed to working with all companies to expedite the development and approval of safe and effective drugs to treat this disease.” FDA goes on to describe (as best they can to assure nondisclosure of confidential information) the discussions it has had with the sponsor, Sarepta Therapeutics . The FDA statement came on the heels of an October 27, 2014 press release from the sponsor.
In FDA’s statement, they go on to describe (in general) the conversations and advice that they have provided to Sarepta over the course of their discussions and outlined (again, in general terms) the additional information that FDA notes that would be needed for filing of the NDA.
The Agency also indicates its willingness to conduct a rolling review of the firm’s application and agrees that the product will likely receive a priority review. But the sponsor must provide the required data and analysis upon which the Agency has counseled the firm. While I have not seen the Agency make many of these statements over my 40 year career, I am happy to see that the Agency is more than just a regulatory body and has compassion for both the caregivers and patients stricken with this disease. Remembering the early years of the AIDS epidemic and the controversy with the cancer community, it appears that FDA has learned a good communication and public relations lesson.