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 Not only was there a misleading published article with respect to the results of Study 201/202147 –which has never been retracted—but Sarepta also issued a press release relying on the misleading article and its findings.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
Harsh but true: if your kid has DMD, they don't have a chance either way. They will die of suffocation or on a hospital ventilator if they're lucky enough to get that far.
The correct but unpalatable politically/socially decision is to support them while expending as little resources as possible on minor therapies for people who are already dead. We need an actual generic modification to fix the disease; focus research there. Until there's a cure, though, drugs like this, especially with immense price tags asked to be borne by society, should never be approved for general use.
Best answer, don't approve it, but don't forbid it either. Simply rate it's safety (or lack thereof) for human use with no endorsement for effectiveness and let doctors and their patients decide case by case.
I assume you meant to type "genetic modification" not "generic modification." As I understand it, this drug is complementary to messenger RNA (mRNA) that codes for an undesirable protein. The intention was that the drug would combine with the mRNA, inactivating the mRNA before it could reach ribosomes, where the protein would otherwise be made. It isn't genetic modification but it is an attempt to impede the expression of a gene.