Douglas Ingram, president and chief executive officer of Sarepta Therapeutics Inc., at the Forbes Healthcare summit in New York, in the United States, Tuesday, December 5, 2023.
Michael Nagle | Bloomberg | Getty images
Actions of Sarepta Therapeutics Plunged more than 30% on Friday while the future of its approved treatment in gene therapy appeared at risk.
The Food and Drug Administration will ask the company to voluntarily arrest all the treatment of treatment, Elenidys, a person familiar with the case told CNBC.
Sarepta told CNBC that he had not heard of the FDA.
In addition, the FDA commissioner Marty Makary told Bloomberg News that the agency examined if the company’s gene therapy should remain on the market.
The FDA investigated two deaths of patients related to Elenidys, which represents more than half of the total income of the Total Net Product of Sarepta. The company also reported a third death linked to a separate experimental gene therapy.
Eledys was bogged down in controversy even before it approved. Gene therapy has not yet clearly proven that it can benefit people with Duchenne muscular dystrophy, a condition that erodes muscle function over time.
People with the disease finally lose the ability to walk and most die at the start of the twenties, which means there is a huge need for unsatisfied treatment. The FDA in 2023 initially granted a conditional approval to patients for patients only between 4 and 5 years old, the group that has seen the most advantages in clinical trials.
The following year, the agency granted complete treatment to the approval of patients 4 years and over who could still walk and accelerate the approval of patients 4 and more who could no longer walk. The latter decision was particularly controversial because there was less evidence that elevations could help people whose disease had already progressed so much.
In addition, Eledys did not achieve its objective in a phase 3 test, although the company argued that the drug was promising on other metrics in the study. Then, the chief of the Center for Biologics Evaluation and Research of the FDA, Peter Marks, agreed from the evaluation of Sarepta and rejected the FDA staff to extend Elédys’ approval.
Earlier this year, Sarepta revealed that two adolescents died of liver failure after receiving elements. Then, this week, reports emerged that another person died during a phase 1 test investigating another Sarepta genetic therapies for a different disease.
The two therapies are different, although they share the same delivery method, increasing security concerns around Elédys. Elédys’ security risks are particularly important given the uncertain profit, said BMO Kostas Biliouris analyst.
For example, Novartis Zolgensma gene therapy for spinal muscle atrophy has also caused hepatic toxicity and death, but the benefit of this treatment is clear.
“This is why the dead here count so much against Zolgensma, for example,” said Biliouris.
And Zolgensma is only a drug of many for a large company like Novartis. For Sarepta, Elenidys is all.
This week’s leaders have tried to reassure investors that even if they can only deal with patients who can still walk, where deaths have not been reported, therapy should bring in at least $ 500 million per year. Last month, Sarepta stopped sending waiting elements to patients who can no longer walk while he explores a safer way to administer the treatment.
The main concern for investors at this stage is whether the FDA draws the drug, said Biliouris. The actions of the company have now dropped by more than 87% this year.
“If the FDA draws elevations from the market,” he says, “Sarepta is over.”