3 moms FDA met with Katz, Woodcock, Temple on Monday
confirmed by jenn: "consider it confirmed. was a very positive meeting"
Office of Drug Evaluation I - Division of Neurology Products (DNP)
The Division of Neurology Products (DNP) regulates and reviews Investigational New Drug (IND) applications and marketing applications for drug and biologic products for the treatment of neurological diseases and conditions, such as Alzheimer’s disease, stroke, Parkinson’s disease, Huntington’s disease, epilepsy, migraine headaches, muscular dystrophy, amyotrophic lateral sclerosis, multiple sclerosis, cerebral palsy, dementia, narcolepsy, lennox-gastaut syndrome, and insomnia.
Director: Russell Katz, M.D.
The naysayers will write off these moms meeting w the FDA as meaningless, but the recent FDA presentation on AA by Dr. Temple confirmed that "patient reported outcomes" (i.e. how the drug has impacted the daily lives of the patients) are becoming more and more important to them in assessing approvals in rare diseases, where the trials provide limited statistical support because of the small patient enrollments. These meetings are not about "anecdotal evidence" that will be tossed aside when it comes down to a decision - they are providing the FDA critical information that could sway the decision. IMO, the dystrophin production data combined with the pristine safety record are sufficient for an AA, so when you add the moms speaking about how their children are flourishing on this drug, it is a slam dunk.