Douglas Ingram, president and executive director of Sarepta Therapeutics Inc., during the Forbes Health Summit in New York, USA, on Tuesday, December 5, 2023.
Michael Nagle | Bloomberg | Getty images
Actions of Therapeutic Sarepta More than 30% fell on Friday, since the future of its approved gene therapy treatment appeared at risk.
The Food and Medicines Administration will request that the company voluntarily stop all the treatment of treatment, a person familiar with the matter told CNBC.
Sarepta told CNBC that he had not had news from the FDA.
Separately, the FDA Commissioner Marty Makary, said in an interview with Bloomberg News that the agency is considering whether the company's gene therapy should remain in the market.
The FDA has been investigating two deaths of patients linked to Elevidys, which represents more than half of the net income of net products from SAREPTA. The company also reported a third death linked to a separate experimental gene therapy.
Elevidys has been involved in controversy even before being approved. Gene therapy has not yet demonstrated that people with Duchenne muscle dystrophy can benefit, a condition that erodes muscle function over time.
People with the disease eventually lose the ability to walk, and most die at age 20, which means that there is a great unsatisfied need for treatment. The FDA in 2023 originally granted EREVidys a conditional approval for patients only between the ages of 4 and 5, the group that saw the greatest benefit in clinical trials.
The following year, the agency granted the total approval of the treatment for patients of 4 years or more who could still walk and accelerate approval for patients from 4 years and older who could no longer walk. The last decision was especially controversial because there was less evidence that Elevidys could help people whose disease had already progressed so much.
In addition, Elevidys failed to fulfill its objective in a phase 3 essay, although the company said the medicine was promising from other metrics in the study. Then, the head of the Evaluation and Research Center of the Biological Center of the FDA, Peter Marks, agreed with the evaluation of SAREPTA and annulled the FDA staff to expand the approval of Elevidys.
Earlier this year, Sarepta revealed that two teenagers died of liver failure after receiving Elevidys. Then, this week, reports arose that another person died during a phase 1 trial that investigates another of Sarepta's genetic therapies for a different disease.
The two therapies are different, although they share the same delivery method, increasing security concerns around Elevidys. The safety risks of the Elevidys are especially important given the uncertain benefit, said BMO Kostas Biliouris analyst.
For example, the gene therapy of Novartis Zolgensma for spinal muscular atrophy has also caused toxicity and liver death, but the benefit of that treatment is clear.
“That is why the deaths here matter so much versus Zolgensma, for example,” said Biliouris.
And Zolgensma is just a drug of many for a large company like Novartis. For Sarepta, Elevidys is everything.
This week, executives tried to assure investors that even if they can only treat patients who can still walk, where deaths have not been reported, therapy should generate at least $ 500 million a year. Last month, Sarepta stopped sending Elevidys to patients who can no longer walk while exploring a safer way to administer treatment.
The main concern for investors at this time is whether the FDA pulls the drug, said Biliouris. The company's shares have now fallen more than 87% this year.
“If the FDA gets out of the market,” he says, “Sarepta is made.”
Jennifer Handt, whose son was diagnosed with Duchenne's muscular dystrophy at the end of 2020, said he was “heartbreaking” that other patients do not have a treatment option to resort if Elevidys shipments stop.
His son, Charlie, was dosed with Elevidys in 2022 as part of the Tardia stage of Sarepta and noticed improvements in six to 12 months, including the increase in resistance and more liquid movements. The drug also relieved a revealing symptom of the condition called Gowers sign, which causes difficulties for children when rising from a sitting or liar position.
She said her son is “completely stable” three years after her dose. Handt said he was aware of the risks of toxicity of the liver before Charlie enrolled in the trial.
“We don't have the luxury of not running the risk,” said Handt. “There are families who dealt with this disease before I had done something to have an option, even if there are risks.”
“Each family must have the option to make this leap with this medicine and potentially see benefits,” he added.
