Douglas Ingram, President and Chief Executive Officer of Sarepta Therapeutics Inc., during the Forbes Healthcare Summit in New York, USA, on Tuesday, December 5, 2023.
Michael Nagle | Bloomberg | Getty pictures
Shares of Sarepta therapeutics More than 30%fell on Friday when the future of approved gene therapy was endangered.
The Food and Drug Administration will apply for the company to voluntarily stop all programs, Eldys, a person who is familiar with the matter, said CNBC.
Sarepta said CNBC that the FDA did not hear it.
Regardless of this, the FDA Commissioner Marty Makary said in an interview with Bloomberg News that the agency was considering whether the company's gene therapy should remain on the market.
The FDA examines two patients bound by Elefidy's deaths, which makes up more than half of the total income from Sarepta. The company also reported a third death that was bound to separate experimental gene therapy.
Eldys was involved in controversy in controversy before approval. Gent therapy must still clearly prove that it can benefit people with Duchenne muscle dystrophy, an illness that undermines muscle function over time.
After all, people with the disease lose the ability to run, and most of them die in their early 20s, which means that there is a large, uncovered need for treatment. In 2023, the FDA originally granted an elemented eligent for patients between 4 and 5 years, the group, which had the greatest benefit in clinical studies.
The following year, the agency of treatment gave patients 4 and older who still go and the approval for patients 4 and on accelerated people who could no longer run. The latter decision was particularly controversial because there was fewer indications that Elefidy's people could help people whose illness had already made so much progress.
In addition, Elefidys did not confront his goal in a phase -3 study, although the company claimed that the drug had performed promising metrics in the study. Then Peter Marks, Head of the FDA Center for Biologics, agreed with Sarepta's assessment and overlooked the FDA employee from FDA to expand the approval of Eltidys.
At the beginning of this year, Sarepta announced that two young boys died of liver failure after receiving elkae. Then this week reports that another person died during a phase -1 study in which another genetic therapies of Sarepta were examined for another disease.
The two therapies are different, although they have the same delivery method, which increases the security concerns around the element closure. The security risks of ELEDYS are particularly important in view of the uncertain benefit, said the BMO analyst Kostas Bilioouris.
For example, Novartis' gene therapy for the muscular atrophy of the spine also causes liver oxicity and death, but the benefits of this treatment is clear.
“That is why deaths here, for example, are so important to Zengensma,” said Biliouris.
And Zengensma is only a drug of many for a large company like Novartis. Elefidys is everything for Sarepta.
This week managers tried to calm investors that the therapy should bring in at least 500 million US dollars a year, even if they can only treat patients who can still run where deaths were not reported. Sarepta hired the shipping of elegips to patients who can no longer run while examining a safer way to give treatment.
At that time, the top concern for investors is whether the FDA is pulling the medication, said Biliouris. The company's share has fallen more than 87% this year.
“When the FDA moved to ELEDS on the market,” he says, “Sarepta is ready.”
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Jennifer Hand, whose son was diagnosed with Duchenne Muskeldystrophy at the end of 2020, said that it was “heartbreaking” that other patients have no treatment option to turn when the shipments are convinced by ELEDYS.
Her son Charlie was dosed in electrodes in 2022 as part of the experiment with Sarepta and found improvements in six to 12 months, including increased endurance and more liquid movements. The drug also facilitated a treacherous symptom of the disease, which is referred to as Gowers signs and causes the children to find difficulties if they get up from a seat or lying position.
She said her son was “completely stable” from his dose for three years. Handt said she was aware of the liver toxicity risks before Charlie was inscribed in the process.
“We don't have the luxury not to take the risk,” said Handt. “There are families who have dealt with this disease because this would have done something to have an option, even if there are risks.”
“Every family should have the choice to make this jump with this medication and possibly see advantages,” she added.
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