Sarepta Therapeutics Inc. (SRPT) Is Sinking After FDA Rejects BioMarin Drug

On Thursday, the U.S. Food and Drug Administration (FDA) turned down a New Drug Application (NDA) for BioMarin’s treatment of Duchenne muscular dystrophy (DMD).

A full copy of the FDA’s eteplirsen review can be found here. Mostly DMD patients lose their battle against the disease by the age of 30.

The drug, eteplirsen, is created to treat a particular subset of Duchenne muscular dystrophy (DMD) patients.

In the review released on Friday, regulators said that Sarepta’s 6-minute walk test results were unreliable; expressed concerns about the extremely small clinical trial size; noted a lack of efficacy at higher doses early on in the treatment process (suggesting even less efficacy at lower doses and later on in the process); and highlighted the failure to meet the primary endpoint.

As for BioMarin Pharmaceutical Inc., it’s not the end of the world for them as they still await the approval of Kyndrisa by the regulating bodies in Europe.

Kyndrisa was regarded by some people as a potential treatment and would have been the first of its kind in the US for the rare muscle-wasting condition had it not been rejected by the FDA. BioMarin expects to hear back from the European regulatory advisory committee in April or May, with a final decision in the third quarter.

“FDA has concluded that the standard of substantial evidence of effectiveness has not been met”, BioMarin said.

Shares of Sarepta were down 57% to $13.60 Friday morning, with a consensus analyst price target of $46.75 and a 52-week trading range of $11.42 to $41.97.

BioMarin also will be watching the FDA’s ruling on a possible competitor, Sarepta Therapeutics’ (NASDAQ:SRPT) eteplirsen. And with a rival put on the shelf, Sarepta’s chances of gaining an approval-despite facing a major challenge to its own limited data set-likely just got better as patients and their families clamor for an approval.