I wasn't crazy about Sarepta defying FDA brass, but I could see why they did. The company was already working through proper channels at the agency, including disclosure of the Phase 1 death in LGMD. Then the media got involved and the newly hired FDA generals felt the need to pull rank. Commissioner Makary's official statement was cringe-worthy: "Today, we’ve shown that this FDA takes swift action when patient safety is at risk.” My hero.
Sarepta CEO Ingram was questioned about this type of contingency at the Q1 Earnings Call in May, specifically regarding the appointment of Prasad as head of CBER that very day. Ingram replied, "What I do remain confident about is that the FDA is going to be the FDA that it's been for the last 100 years, which is an organization dedicated to following great science and fulfilling its mission of bringing life-enhancing therapies that are safe and efficacious to patients. And there's no reason to believe that that should change or that anyone would permit that to change."
Later, yet another concerned analyst asked about the appointment of Prasad. Ingram said, "Let me speak to what we know today. Of course, that appointment occurred only today. So what I can tell you is that subsequent to the change of Commissioner Makary and then the departure of Dr. Marks, we've had a significant number of interactions with our primary reviewer on the biologics side, which is OTP at CBER, and we have seen so far no changes in the approach they're taking. In fact, Dr. Verdun appears to be quite innovative in her goal of moving therapies along and relying upon modern tools for drug development. I would also note that Dr. Makary hasn't made many public statements, so it's difficult to deduce precisely what his views are on things."
Two weeks later, Verdun was placed on administrative leave, along with her deputy, Dr. Rachael Anatol. From Chatgpt: "This action has caused industry-wide concern, as Verdun was seen as a key leader in cell and gene therapy regulation." I was unaware of this when I said, "Those in the know already know." Sounds like they have some catching up to do. Bottom line, the Phase 1 death was already reported to the agency in June. |
