Biotech Values

[jb_118]

but totally ignore the real DMD experts' opinion

I sat down with one of the "DMD experts" to talk about another subject, but we ended up drifting to this topic and hashed it out. He ultimately agreed with the conclusion that the company had completely mismanaged the development of the drug (unfortunately) and that efficacy wasn't well enough established to meet the rigor the FDA requires for approval, and that it would be bad precedent for the FDA to approve the drug based on the current information. He seemed hopeful that the drug would work eventually but had a healthy amount of skepticism.

At the very conclusion of our conversation I gathered that he (or his institution) has had at least one patient enrolled in one of the Etep studies, but there was specific discussion about this.

When I went back and checked the open letter to the FDA from DMD experts calling for approval, I found his name on the list. I was somewhat surprised to find his name there given our conversation. I lean to the conclusion that there is such a strong advocacy from the patient groups that it was just easier to sign than to become a target. Or maybe he's just as conflicted as the rest of everybody between hope and well-designed trials.

Anyway, my point is that I don't think "listening to the DMD experts" is as straightforward as you might think.

My disclosure is that I have no position in SRPT or any related companies, although I am a former SRPT long and got out when it became apparent that they were going to try and lobby their way to approval rather than properly develop the drug.