(Total Views: 366)
Posted On: 09/07/2019 4:50:30 AM
Post# of 155188
Very interesting, yeah, that likely, copied below. Those reasons seemed lame. Leronlimab offers huge advantages: weekly dosing, lower toxicity, much better results over what is already approved. Why is weekly dosing an advantage? Many in this mdr2 group have picked up resistance by not complying with their daily pill regimen. So having one drug dose weekly should cover them if they forget to take their pills one day.
The real reason for rejection much more likely has to do with the FDA being pressured into approving Exondys 51 back in 2016. A drug in the neighborhood of $300,000 with no solid proof of clinical benefit is almost never approved, and the FDA was pressured into approval from many different directions at the time. It ultimately led to resignations at the top of the agency, and Sarepta got only a conditional approval requiring future clinical data demonstrating clinical benefit to patients. That data is still pending.
A reasonable guess is that the FDA found itself in a quandary over golodirsen. It couldn't say explicitly that it was rejecting golodirsen based on lack of data proving clinical benefit, because then why was Exondys 51 conditionally approved? It also couldn't just approve golodirsen straight up because doing so would telegraph to biotech companies that the Exondys 51 approval was not a special case.
The real reason for rejection much more likely has to do with the FDA being pressured into approving Exondys 51 back in 2016. A drug in the neighborhood of $300,000 with no solid proof of clinical benefit is almost never approved, and the FDA was pressured into approval from many different directions at the time. It ultimately led to resignations at the top of the agency, and Sarepta got only a conditional approval requiring future clinical data demonstrating clinical benefit to patients. That data is still pending.
A reasonable guess is that the FDA found itself in a quandary over golodirsen. It couldn't say explicitly that it was rejecting golodirsen based on lack of data proving clinical benefit, because then why was Exondys 51 conditionally approved? It also couldn't just approve golodirsen straight up because doing so would telegraph to biotech companies that the Exondys 51 approval was not a special case.
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