Sarepta shares plunge 40% as future of its gene therapy appears at risk

Shares of Sarepta Therapeutics plunged much than 30% connected Friday arsenic the aboriginal of its approved cistron therapy attraction appeared astatine risk.

The Food and Drug Administration volition petition that the institution voluntarily halt each shipments of the treatment, Elevidys, a idiosyncratic acquainted with the substance told CNBC.

Sarepta told CNBC it had not heard from the FDA.

Separately, FDA Commissioner Marty Makary said successful an interrogation with Bloomberg News that the bureau is considering whether the company's cistron therapy should enactment connected the market.

The FDA has been investigating 2 diligent deaths tied to Elevidys, which accounts for much than fractional of Sarepta's full nett merchandise revenue. The institution besides reported a 3rd decease tied to a abstracted experimental cistron therapy.

Elevidys has been mired successful contention adjacent since earlier it was approved. The cistron therapy has yet to intelligibly beryllium it tin payment radical with Duchenne muscular dystrophy, a condition that erodes musculus relation implicit time.

People with the illness yet suffer the quality to walk, and astir dice by their aboriginal 20s, meaning there's a immense unmet request for treatment. The FDA successful 2023 primitively granted Elevidys a conditional support for patients lone betwixt the ages of 4 and 5, the radical that saw the astir payment successful objective trials.

The pursuing year, the bureau granted the attraction afloat support for patients 4 and older who could inactive locomotion and accelerated support for patients 4 and up who could nary longer walk. The second determination was particularly contentious due to the fact that determination was little grounds that Elevidys could assistance radical whose illness had already progressed truthful much.

Plus, Elevidys failed to conscionable its extremity successful a Phase 3 trial, though the institution contended that the cause showed committedness connected different metrics successful the study. Then caput of the FDA's Center for Biologics Evaluation and Research, Peter Marks, agreed with Sarepta's assessment and overruled FDA unit to grow support of Elevidys.

Earlier this year, Sarepta disclosed that 2 teenage boys died from liver nonaccomplishment aft receiving Elevidys. Then this week, reports emerged that different idiosyncratic died during a Phase 1 proceedings investigating different 1 of Sarepta's cistron therapies for a antithetic disease.

The 2 therapies are different, though they stock the aforesaid method of delivery, heightening the information concerns astir Elevidys. The information risks of Elevidys are particularly important fixed the uncertain benefit, said BMO expert Kostas Biliouris.

For example, Novartis' cistron therapy Zolgensma for spinal muscular atrophy has besides caused liver toxicity and death, but the payment of that attraction is clear.

"That's wherefore deaths present substance truthful overmuch versus Zolgensma, for example," Biliouris said.

And Zolgensma is conscionable 1 cause of galore for a ample institution similar Novartis. For Sarepta, Elevidys is everything.

Executives this week tried to reassure investors that adjacent if it tin lone dainty patients who tin inactive walk, wherever deaths haven't been reported, the therapy should bring successful astatine slightest $500 cardinal a year. Sarepta past period stopped shipping Elevidys to patients who tin nary longer locomotion portion it explores a safer mode to administer the treatment.

The apical interest for investors astatine this constituent is whether the FDA pulls the drug, Biliouris said. The company's banal has present fallen much than 87% this year.

"If the FDA pulls Elevidys from the market," helium says, "Sarepta is done."

Jennifer Handt, whose lad was diagnosed with Duchenne muscular dystrophy successful precocious 2020, said it was "heartbreaking" that different patients won't person a attraction enactment to crook to if shipments of Elevidys are paused. 

Her son, Charlie, was dosed with Elevidys successful 2022 arsenic portion of Sarepta's late-stage proceedings and noticed improvements successful six to 12 months, including accrued stamina and much fluid motions. The cause besides eased a telltale grounds of the information called Gowers' sign, which causes children trouble erstwhile getting up from a sitting oregon lying position. 

She said her lad is "completely stable" 3 years retired from his dose. Handt said she was alert of the liver toxicity risks earlier Charlie enrolled successful the trial.

"We don't person the luxury of not taking the risk," Handt said. "There are families that dealt with this illness earlier that would person done thing to person an option, adjacent if determination are risks." 

"Every household should person the prime to instrumentality this leap with this cause and perchance spot benefits," she added.