I believe the opposite. Newly enacted federal legislation encourages and empowers FDA to accelerate the approval of new, innovative drugs for rare and life-threatening diseases. If the 48-week eteplirsen data confirm the positive benefit for Duchenne muscular dystrophy patients observed already, FDA would be well within its regulatory purview to grant accelerated approval.
FDA is constantly under political attack for slowing the development of life-saving drugs. With eteplirsen, the agency has an opportunity to show that it is responsive to Congress and the needs of kids with a debilitating and deadly disease.
Also, don't underestimate the influence that families affected by DMD will have on FDA's decision to approve eteplirsen early. Already, one Vermont mother, Jenn Mcnary, is receiving a lot of media attention for her efforts to obtain eteplirsen on a compassionate-use basis for her son Austin, 13, who suffers from DMD. Another of Mcnary's sons, Max, 10, also suffers from DMD but he's literally running around the house after being treated with eteplirsen in Sarepta's phase II trial.
Max is one of the four patients in the trial treated with the 50-mg dose of eteplirsen. Mcnary doesn't know Max's actual results from the trial because the data remain blinded but the change in his health is unmistakable, she says."Max today is more like Tigger than Eeyore," says Mcnary. "My husband and I have to tell him to stop running in the house. We never had to do that before." Some of the recent press accounts of Mcnary's efforts to obtain eteplirsen for Austin have painted Sarepta as the bad guy -- a drug company refusing to help a child patient in need. But Mcnary says she has no animosity towards Sarepta, which doesn't have the financial resources today to ramp up manufacturing enough to provide eteplirsen via a compassionate-use program. "What everyone wants is for FDA to grant early approval of eteplirsen," she says. Towards that end, Mcnary plans to meet with FDA in October, around the same time that Sarepta will be meeting with FDA to discuss accelerated approval. She hopes to have other DMD patient advocates with her. The next big catalyst for Sarepta will be the release of the 48-week eteplirsen data in mid-October at the World Muscle Society conference.
D. Jones asks, "Do you have an opinion on Delcath Systems (DCTH)? They are submitting their NDA [New Drug Application] later this year and I was wondering if it's time to buy before a potential run-up." I'm not a Delcath fan, never been one, mainly because of the small commercial market for the company's Chemosaturation (ChemoSat) liver-tumor treatment system. I haven't had big concerns about the FDA approvability of ChemoSat, more that Delcath could never deliver sufficient revenue to generate a meaningful profit. [Sales to date in Europe, where ChemoSat is approved, have been minimal.]
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