Sarepta stock falls on FDA gene therapy Elevidys comment

Shares of Sarepta Therapeutics plunged greater than 30% on Friday as the long run of its approved gene therapy treatment appeared in danger.

The Food and Drug Administration will request that the corporate voluntarily stop all shipments of the treatment, Elevidys, an individual conversant in the matter told CNBC.

Sarepta told CNBC it had not heard from the FDA.

Individually, FDA Commissioner Marty Makary said in an interview with Bloomberg News that the agency is considering whether the corporate’s gene therapy should stay in the marketplace.

The FDA has been investigating two patient deaths tied to Elevidys, which accounts for greater than half of Sarepta’s total net product revenue. The corporate also reported a 3rd death tied to a separate experimental gene therapy.

Elevidys has been mired in controversy even since before it was approved. The gene therapy has yet to obviously prove it might probably profit individuals with Duchenne muscular dystrophy, a condition that erodes muscle function over time.

Individuals with the disease eventually lose the flexibility to walk, and most die by their early 20s, meaning there is a huge unmet need for treatment. The FDA in 2023 originally granted Elevidys a conditional approval for patients only between the ages of 4 and 5, the group that saw probably the most profit in clinical trials.

The next 12 months, the agency granted the treatment full approval for patients 4 and older who could still walk and accelerated approval for patients 4 and up who could not walk. The latter decision was especially contentious because there was less evidence that Elevidys could help people whose disease had already progressed a lot.

Plus, Elevidys failed to satisfy its goal in a Phase 3 trial, though the corporate contended that the drug showed promise on other metrics within the study. Then head of the FDA’s Center for Biologics Evaluation and Research, Peter Marks, agreed with Sarepta’s assessment and overruled FDA staff to expand approval of Elevidys.

Earlier this 12 months, Sarepta disclosed that two teenage boys died from liver failure after receiving Elevidys. Then this week, reports emerged that one other person died during a Phase 1 trial investigating one other one among Sarepta’s gene therapies for a special disease.

The 2 therapies are different, though they share the identical approach to delivery, heightening the protection concerns around Elevidys. The security risks of Elevidys are especially essential given the uncertain profit, said BMO analyst Kostas Biliouris.

For instance, Novartis’ gene therapy Zolgensma for spinal muscular atrophy has also caused liver toxicity and death, but the advantage of that treatment is evident.

“That is why deaths here matter a lot versus Zolgensma, for instance,” Biliouris said.

And Zolgensma is only one drug of many for a big company like Novartis. For Sarepta, Elevidys is all the pieces.

Executives this week tried to reassure investors that even when it might probably only treat patients who can still walk, where deaths have not been reported, the therapy should usher in at the least $500 million a 12 months. Sarepta last month stopped shipping Elevidys to patients who can not walk while it explores a safer technique to administer the treatment.

The highest concern for investors at this point is whether or not the FDA pulls the drug, Biliouris said. The corporate’s stock has now fallen greater than 87% this 12 months.

“If the FDA pulls Elevidys from the market,” he says, “Sarepta is completed.”

Jennifer Handt, whose son was diagnosed with Duchenne muscular dystrophy in late 2020, said it was “heartbreaking” that other patients won’t have a treatment choice to turn to if shipments of Elevidys are paused. 

Her son, Charlie, was dosed with Elevidys in 2022 as a part of Sarepta’s late-stage trial and noticed improvements in six to 12 months, including increased stamina and more fluid motions. The drug also eased a telltale symptom of the condition called Gowers’ sign, which causes children difficulty when getting up from a sitting or lying position. 

She said her son is “completely stable” three years out from his dose. Handt said she was aware of the liver toxicity risks before Charlie enrolled within the trial.

“We do not have the posh of not taking the chance,” Handt said. “There are families that handled this disease before that will have done anything to have an option, even when there are risks.” 

“Every family must have the selection to take this leap with this drug and potentially see advantages,” she added.