Sarepta’s phase IIb study of only 12 DMD patients, 2 of which sadly dropped out of the walking portion early, was not robust enough to seek early approval, I think the time had arrived to go for it. For example, political support for speeding up what I believe is exactly this type of program could not be any clearer. In July of 2012, Congress passed the FDASIA legislation. Among other things, FDASIA argued for expedited paths to approval for breakthrough therapies (Sarepta technically did not apply for this specific designation), it had a provision that explicitly asked FDA’s Center for Drug Evaluation and Research (CDER) and Centers for Biologics Evaluation and Research (CBER) to come up with policy related to patient outreach and advancing drug development for rare diseases, and offered a pediatric rare disease review voucher that was meant as extra incentive for companies to go after these diseases. Those signals show how there is a lot of outside support for programs like this, and a need for change, so I think publicly seeking them was entirely appropriate.
Second, you cannot ignore the human factor. There are no approved treatments that seek to correct the cause of DMD, so as you can imagine, families have an incredible sense of urgency to do anything to help their children who have this condition. In fact, if you discuss biotech on Twitter, you may have come across a DMD patient advocate, many of whom sadly use the hourglass as their avatar and a symbol of what they are up against. It is heartbreaking to think what these families must be going through.
Given that, put yourself in Mr. Garabedian’s position. You can bet he receives phone calls and letters, probably every day, from families urgently asking him for help. In my opinion, if you are in his shoes, receiving calls for help like that every day, and you believe your drug works, the only choice is to push for rapid approval. I follow this very closely and can tell you that he has always done so in an entirely appropriate, ethical, professional, and level way with both those families and the regulators. He has been careful not to overhype the results Sarepta has seen, and not to try to bulldoze his way through the FDA.
Third, it is not his fault that a competitor’s drug (Prosensa and GlaxoSmithKline’s drisapersen) with similar, but very different, characteristics failed a large study a couple of months ago. Those that criticize Sarepta’s goal of seeking early approval seem to forget that FDA signaled this summer that they were open to accepting such a filing for review. It wasn’t until after the competitor failed (and a natural history study from another DMD candidate was recently published) that FDA changed its mind and insisted that the 12-patient study doesn’t provide enough evidence for Sarepta to seek approval. I do not see how any of that is Garabedian’s fault. I also do not see how Sarepta could have kept FDA’s previous willingness to review a filing private considering how patient interest in this issue is so high and urgent. In my opinion, he did the right thing to seek the go ahead to file, he basically received it, and it is not his fault that outside circumstances have so unfortunately changed the situation. None of that was foreseeable, so you cannot hold it against him.
I would just like to conclude by saying that I think Chris Garabedian and his team deserve not only a break on this, but tons of gratitude and respect for their hard work, under an insane amount of pressure, over the last year and a half. There is no doubt this has been a very disappointing week. However, his leadership has already moved mountains to get us where we are, and while this week’s news frankly stinks, I cannot think of a better person to manage the company back to a path of ultimate success. Hindsight is always 20/20, but I still would not change a thing about the way this was managed. I’m immensely proud and supportive of the work he has done.
