Sarepta Refused FDA’s Request to Halt Elevidys Shipments

(Bloomberg) – Food and Pharmaceutical Administration said on Friday, Sarepta Therapeutics Inc.’s three deaths refused to pause all posts after the company’s gene therapies.

Two young men died of acute liver failure after taking Elevidys in recent months. They were treated for Duchenne muscle dystrophy and could not walk due to muscle increase. The company said on Friday, a 51 -year -old patient last month from acute liver failure, extremity belt to treat muscle dystrophy a gene treatment died in an early stage attempt, he said.

FDA leaders met with Sarepta and the agency said in a statement and asked to voluntarily stop all posts of the drug, the biggest product. “The company rejected it,” the Agency said.

In his own statement on Friday, Sarepta said that “new or changing security signals that do not show new or changing security signals decided to continue to send to Evlıdys”.

Sarepta shares have fallen 36% on Friday since 2016. The company has lost about $ 8.5 billion since March 18, when the first patient death was reported.

Everidys is an important drug for Sarepta and in the second quarter of this year, it constitutes more than half of the company’s net product revenue. In June, Sarepta suspended the posts of the drug for patients who can no longer walk.

Given the new security information, FDA said that gene therapy should be given to men who can only walk. The company said that since the launch of about 85% of patients treated with Elevidys.

Developments also created doubts about the future of the drug.

Baird Analyst Brian Skorney in a note on Friday, “FDA’nın Sarepta’dan Elevidys’in all the posts that will want to stop all posts, following the reports of the FDA’s completely reinforced risk of the drug from the market is now strongly strengthened.” He said.

The newest patient death occurred in the study of a gene therapy using a similar viral transmission method with Elevidys, ie safety effects for patients receiving the drug in the market.

In an interview on Friday, the FDA commissioner Marty Makary said that the organizer examined whether Elevidys will be on sale. FDA was already investigating the death of the previous two patients. Makary commented on a question of Bloomberg News and did not give any more details.

The regulator is also under pressure for Sarepta’s posture for gene therapy. Deaths are one of the first major tests for Vinay Prasad, the new president of the regulator’s gene therapy department. As an academician at the University of California San Francisco, he criticized the accelerated approval process for the treatment of Sarepta before his current role.

The latest death launched an unusual tense change between analysts and Sarepta executives in a call on Friday. Two days ago, the company had made a long call with the same analyst group, but he could not explain that a third patient had recently died in a similar treatment with Elevidys. On Friday, the two analysts asked if there were other deaths that the company had not announced.

Douglas Ingram, Chairman of the Sarepta Executive Officer, said that he did not defend the company’s registration and added the following and added: uz We are historically a very transparent organization. ”

Ingram said that the company did not explain the death of the third patient at the beginning of the week, because it was “neither material nor center”. In a note before the Friday call, Leerink Analyst Joseph P. Schwartz said that Sarepta did not talk about the last death on Wednesday, “deeply disturbing and weakening the reliability further”. William Blair Analyst Sami Corwin said he could increase the “investor insecurity”.

Sarepta said on Wednesday that his doctors and patients agreed to warn Elevidys about the risk of liver failure at the request of FDA. Sarepta executives said the warning label solved the FDA’s concerns about using gene therapy to treat children with Duchenne.

Inek I think that the answer is quite clearly in the general question about whether Elevidys will be on the market as therapy, ”Ingram said in a statement on Wednesday.

Duchenne muscle dystrophy is a genetic disorder that affects men who interfere with the production of protein dystrophin used by muscle cells. As it gets older, it causes weakening and atrophy of severe muscles with patients who need to use wheelchairs. The extremity bird muscle dystrophy is a different condition that often weakens muscles around hips and shoulders.

Elevidys has been one of the most popular gene therapies on the market. In May, more than 800 patients were treated with Evlıdys in commercial environments and clinical studies. Many families are desperate for anything that can help children with deadly muscle disease, despite the lack of data indicating that Sarepta’s treatment is actually slowing down the overall progress of the situation.

Debra Miller, the founder of the Patient Defense Group Cureduchenne, said that families “deserve clear and transparent information” about the safety and effectiveness of a treatment and that some have less or other options.

“Families with Duchenne have no time to waste,” he added.

However, the death series can make patients more reluctant to use Elevidys. On Wednesday, Ingram said that the company should “assess the effect of hesitation associated with these events”.

On the same day, the company said that the labor force had cut more than one -third and paused several drugs on the pipeline. The company said that moves will contribute to an estimated $ 400 million in annual cost savings.

-Subrat Patnaik’ten help.

(It is updated by the comments of Sarepta expression in the fourth paragraph.)

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