I recently wrote a review of Sarepta Pharmaceuticals drug eteplirsen for the treatment of Duchenne muscular dystrophy. The gist of it is that the FDA is about to review the drug and vote whether to approve or reject it, on April 26. There is no significant proof that the drug works. Evidence suggests that it is safe though. Just to get the disclosure out of the way for legal reasons, I own no stake in Sarepta and don’t plan on buying any.
Muscular dystrophy is a horrible horrible disease. I wouldn’t wish it on anybody except members of Congress and the Knesset, but since it’s genetic that’s only rhetorical.
People who have it will die by the time they are 25 or so. Their muscles will decay and they will not be able to move, and then eventually they will not be able to breathe and that’s it.
Eteplirsen tries to repair the broken gene that codes for dystrophin, the protein that most of us have that enables us to move. It might work, it might not. It’s hard to tell. But without it, everyone with muscular dystrophy on exon 54 will die. With it, maybe they will, and maybe they won’t.
The meeting takes place on April 26. There will be record attendance at the meeting, I just read. 1,200 people will watch a group of government doctors determine if dying people will be allowed to take eteplirsen. The entire addressable market for this form of the disease is about 1,560 in the US.
The entire question is absolutely insane. If you don’t realize that, you are amoral, or immoral, or both. If these people want to take the damn drug, they should be able to take it at the agreed upon price, even if it were extremely dangerous, which it isn’t.
The thought of self righteous government scientists who do not have muscular dystrophy deciding for a group of people whether or not they can try the drug, just drives me mad. It makes me want to cry for them.
They will probably reject approval. For the greater good of course. Because if they are God forbid allowed to even try a drug that has no proven efficacy, the entire reason d’etre of the FDA will cease to exist.
Newsflash. There is no reason for the FDA to exist. All they do is kill people in the name of safety.