Aug. 4 Quick Takes: HHS opens investigation into accelerated approval pathway

HHS’s Office of the Inspector General will review how FDA implements the accelerated approval pathway and make recommendations based on a sample of drugs that were approved via the pathway, including Alzheimer’s therapy Aduhelm aducanumab-avwa from Biogen Inc. (NASDAQ:BIIB) and Eisai Co. Ltd. (Tokyo:4523). The announcement comes after acting FDA Commissioner Janet Woodcock requested an independent investigation of FDA’s interactions with Biogen during Aduhelm’s review by FDA. In a tweet, Woodcock wrote, “The FDA intends to fully cooperate with the HHS OIG review and welcomes the opportunity to provide clarity regarding the science-based application of the accelerated approval pathway.” 

Sarepta Therapeutics Inc. (NASDAQ:SRPT) added a sixth limb-girdle muscular dystrophy gene therapy to its pipeline through an in-licensing deal with the Abigail Wexner Research Institute at Nationwide Children’s Hospital for a program that targets LGMD Type 2A, which accounts for nearly one third of LGMD cases. Sarepta’s LGMD candidates, all of which use the rh.74 adeno-associated viral vector to target skeletal muscle, address six LGMD subtypes, which combined represent more than 70% of known LGMDs. ...