Please keep our friends in the DMD community in your thoughts and prayers. The FDA will decide this upcoming week whether or not to approve the drug, Eteplirsen. Given the positive meeting that FAST had with FDA officials, we are hopeful that this safe drug will be approved for the DMD community.
“FDA has been successful at applying flexibility in oncology and HIV/AIDS to speed patient access to apparently safe treatments, and the need and opportunity to adopt innovative and flexible approaches to the review of rare disease drugs has never been greater than it is today. Patients are waiting.”