Drug Development

FDA’s controversial OK for eteplirsen spurs second thoughts for a rejected rival at BioMarin


Jean-Jacques Bienaimé, BioMarin CEO

Sarepta’s regulatory win for Exondys51 (eteplirsen) left one group of analysts and FDA insiders more than a little flabbergasted.

There was only a minuscule amount of data to look at. And after all, PTC never even got in the doors at the FDA, while the agency had no trouble slapping down BioMarin’s application for drisapersen, which had more data — including its failure in clinical studies and troubling safety signs.

Inspired by the eteplirsen decision, BioMarin $BMRN is rethinking its decision to heave drisapersen off to the scrap yard. The rare disease specialist is mulling the notion of appealing its FDA rejection for its rival Duchenne muscular dystrophy drug, acquired in the Prosensa buyout for $680 million. In a statement to Endpoints News, the company said:

The decision by the FDA to issue a Complete Response Letter for drisapersen is one that can be appealed.  BioMarin is exploring its options.  The company has not determined if it will pursue an appeal.

The FDA’s approval for Exondys51 is shaping up as one of the most controversial on record. Janet Woodcock’s steadfast support for Sarepta $SRPT and its drug triggered a war inside the agency, as senior-level officials ridiculed the lack of evidence on efficacy and questioned the safety profile that Sarepta had presented. The FDA commissioner even chided Sarepta for using a clearly dodgy study to back its application, but deferred to Woodcock’s experience in the field in allowing the approval to go through.

Even the suggestion of a new shot at an approval for drisapersen, though, triggered withering remarks on Twitter over the toxicity issues tied to the drug. Sarepta’s share price soared on the FDA’s approval, leaving it in sole command of the market for a drug that costs $300,000 a year. Any perceived threat to that, however slight, by bringing back this troubled application was in line for a backlash.

It may be a long shot, but BioMarin also has nothing much to lose at this point if it decides to move ahead.

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