Sarepta Craters as FDA Considers Requesting Halt to Elevidys Shipments

A 3rd loss of life linked to Sarepta Therapeutics’ gene remedy platform reported Thursday will be the ultimate straw for the FDA, which can request that Sarepta voluntarily cease all shipments of its Duchenne muscular dystrophy therapy Elevidys, in accordance with Reuters, which quoted a supply conversant in the matter.

In an e mail to BioSpace, a Well being and Human Providers consultant confirmed the Reuters report. Moreover, an HHS official informed BioSpace in the identical e mail, “We’re taking a tough take a look at pulling it from the market.”

In one other report, FDA Commissioner Marty Makary informed Bloomberg Information that the regulator is contemplating whether or not Elevidys, an adeno-associated virus (AAV) vector–based mostly gene remedy that was first accepted in June 2023, ought to keep available on the market. Sarepta informed CNBC, nevertheless, that it had not heard from the FDA.

Shares of the biotech have been down almost 37% on the time of publication.

This newest information follows a tough season for Sarepta, which beforehand reported the deaths of two teenage sufferers taking Elevidys from acute liver failure. On Wednesday, Sarepta added a boxed warning to Elevidys’ for acute liver harm and acute liver failure. On Thursday, information broke of a 3rd loss of life, that of a 51-year-old affected person in a Part I trial for Sarepta’s limb-girdle muscular dystrophy (LGMD) gene remedy SRP-9004, which makes use of the identical underlying platform expertise as Elevidys. Like the 2 teenage boys, the person additionally died of acute liver failure.

It’s been a rollercoaster of every week for Sarepta, which on Wednesday noticed its inventory leap 18% after buyers responded with cautious optimism to a enterprise overhaul that noticed the layoffs of round 500 staff and a pivot from gene remedy to its siRNA platform belongings.

“The one danger we see transferring ahead is the loss of life of a 3rd Elevidys affected person,” BMO mentioned in a word to buyers on the time.

Whereas the third loss of life wasn’t related to Elevidys, Sarepta’s lack of disclosure earlier this week didn’t sit effectively with buyers. “We expect the LGMD affected person loss of life may amplify affected person hesitancy to make use of business Elevidys and enhance investor mistrust because the firm didn’t disclose the occasion on its name,” William Blair wrote in a word to buyers early Friday morning.

In a word to buyers Friday afternoon, BMO mentioned Sarepta revealed throughout a administration name that the disclosure of this loss of life was not made throughout Wednesday’s name as a result of given the discontinuation of the SRP-9004 program.