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Eteplirsen received approval for use as a Duchenne muscular dystrophy therapy despite the FDA review team concluding that the treatment was unlikely to show any benefit for patients.
Dr. Janet Woodcock's (Director of the Center for Drug Evaluation and Research) decision was heavily influenced by the "parading diseased children in front of the cameras" and was made before the FDA's review team completed their analysis.
Part of Dr. Woodcock's rational for approval included the stock price of Sarepta (the pharmaceutical company responsible for eteplirsen):
She opined that Sarepta in particular "needed to be capitalized." She noted that [Sarepta's] stock went down after the AC meeting and went up after FDA sent the June 3, 2016 letter. Dr. Woodcock cautioned that, if Sarepta did not receive accelerated approval for eteplirsen, it would have insufficient funding to continue to study eteplirsen and the other similar drugs in its pipeline.
FDA Commissioner Dr. Robert Califf, Acting Chief Scientist Dr. Luciana Borio, and Dr. Ellis Unger, the Director of the Office of Drug Evaluation, all opposed the approval but Dr. Califf declined to overrule Dr. Woodcock's decision.
Dr. Unger argued that the approval was unethical and counterproductive:
By allowing the marketing of an ineffective drug, essentially a scientifically elegant placebo, thousands of patients and their families would be given false hope in exchange for hardship and risk.
Dr. Borio argues:
Granting accelerated approval here on the basis of the data submitted could make matters worse for patients with no existing meaningful therapies — both by discouraging others from developing effective therapies for DMD and by encouraging other developers to seek approval for serious conditions before they have invested the time and research necessary to establish whether a product is likely to confer clinical benefit.
[...] [Sarepta] has exhibited serious irresponsibility by playing a role in publishing and promoting selective data during the development of this product. Not only was there a misleading published article with respect to the results of Study 201/202147 –which has never been retracted—but Sarepta also issued a press release relying on the misleading article and its findings.
Dr. Derek Lowe, from In The Pipeline, agrees with Dr. Unger and Dr. Borio that the drug is "unlikely to provide much benefit, and is reasonably likely to provide none at all" and that the drug "may well be [$300,000 per year] worth of placebo".
Note: Bold was added by the submitter.
http://endpts.com/senior-fda-officials-warned-that-eteplirsen-ok-would-lower-fda-standards/
http://blogs.sciencemag.org/pipeline/archives/2016/09/20/sarepta-gets-an-approval-unfortunately
https://en.wikipedia.org/wiki/Eteplirsen
(Score: 2) by butthurt on Thursday September 22 2016, @09:46PM
My understanding of this is scanty, but I see a possible way that it could be ineffective. As I understand, the drug is an RNA molecule that's been modified so it won't be metabolised. It binds to messenger RNA that codes for an unwanted protein. So when a cell makes that messenger RNA, the drug binds to it, forming a combination that, I assume, resists decomposition by the cell. That would use up the drug. Whatever regulates the expression of genes may sense that the mRNA or the protein aren't present. Since the cell's DNA remains unchanged, that DNA can direct the production of additional messenger RNA. The amount of the drug that's administered is limited, but the "ingredients" for RNA are abundant so the cell may be able to produce more mRNA molecules than there are available Eteplirsen molecules (stoichiometry), and dystrophin will be made.
(Score: 0) by Anonymous Coward on Friday September 23 2016, @12:50PM
You're probably right. The idea should work, which is why it was called a "scientifically elegant placebo", but the levels of mRNA restored with the frequent injections are still too low for reliable detection.