Sarepta stock falls on FDA gene therapy Elevidys comment

Shares Sarepta therapeutics The future of approved gene therapy treatment dived more than 30% on Friday because the future of the treatment appeared at risk.

The Food and the Drug Administration will ask the company to voluntarily stop all of the treatment, which is a person familiar with CNBC.

Sarepta told CNBC that he did not hear from FDA.

Separately, FDA Commissioner Marty Makary Told to Bloomberg News The agency thinks that the company’s gene therapy will remain in the market.

FDA is investigating the deaths of two patients with Elevidys, which constitute more than half of Sarepta’s total net product revenue. The company also reported a third death of a separate experimental gene therapy.

Elevidys has been united in discussions even since it was approved. Gen therapy has not made it clear that it can benefit people with Duchenne muscle dystrophy, a condition that erodes muscle function over time.

People with disease eventually lose their ability to walk and die in the beginning of their 20s, so there is a great treatment that is not met. The FDA in 2023 initially gave an Everidys a conditional approval for patients between the ages of 4 and 5, who were only the group that was the most beneficial in clinical studies.

The following year, the agency gave full approval for 4 or older patients who could still walk to treatment and approved for patients who can no longer walk. The second decision is particularly controversial, because Elevidys had fewer evidence that it could help people who already advanced.

Plus, Everidys could not meet his goal in a phase 3 attempt, but the company argued that the drug promised other metrics in the study. Then the head of the FDA’s Biological Evaluation and Research Center Peter Marx accepted Sarepta’s evaluation and FDA personnel were rejected to expand the approval of Everidys.

Earlier this year, Sarepta announced that two young children died of liver failure after taking Elevidys. Later, this week, in the Phase 1 attempt, another person died of one of Sarepta’s gene therapies for a different disease.

Two therapies are different, but share the same delivery method and increase safety concerns around Elevidys. BMO Analyst Kostas Biliouris said that Elevidys’ security risks are particularly important, considering uncertain benefit.

For example, novartis’s gene therapy has also caused liver toxicity and death for zolgensma and spine muscle atrophy, but the benefit of this treatment is clear.

Biliouris said, “That’s why the deaths here are very important to Zolgensma, for example,” he said.

And for a large company like Zolgensma, Novartis, many are just a drug. Elevidys for Sarepta is everything.

This week, managers tried to assure investors that the therapy should bring at least 500 million dollars a year in cases where deaths were not only reported and deaths are not reported. Sarepta last month, while exploring a safer way to apply treatment, stopped raising to patients who can no longer walk.

At this point, Biliouris is the greatest concern of investors whether the FDA is taken into consideration. The company’s shares fell more than 87% this year.

“If FDA withdraws Elevidys from the market,” he says, “Sarepta is over.”