Retail Bulls Stick With Sarepta After 8-Year-Old Patient’s Death Deemed Unrelated To DMD Gene Therapy

Retail sentiment around Sarepta Therapeutics remained upbeat on Monday after the company said Friday that the death of an eight-year-old boy in Brazil was determined to be unrelated to Elevidys, its gene therapy for Duchenne muscular dystrophy, amid an investigation by the U.S. Food and Drug Administration (FDA).

The company reported the death to the FDA on June 18 through its postmarketing safety database. The boy was not part of a clinical trial.

The death occurred on June 7, according to a statement from the U.S. Food and Drug Administration, which did not specify where the boy lived, according to a Bloomberg report. 

On Thursday, Roche Holding AG, which markets Elevidys outside the U.S., said a patient in Brazil had recently died after receiving the gene therapy for Duchenne muscular dystrophy.

Brazilian health officials stated that the cause was likely a viral infection that may have been exacerbated by immune suppression. Steroids that lower the immune response are commonly used in Duchenne treatment, including those used alongside Elevidys.

The FDA said on Friday that it is investigating the case. A Sarepta spokesperson confirmed the probe is tied to the Brazil death.

The update comes as Sarepta faces growing regulatory pressure. Last Monday, the company paused all U.S. shipments of Elevidys in response to an FDA request, reversing its earlier stance. 

The pause allows Sarepta time to update the therapy’s safety label following multiple recent deaths in separate gene therapy programs, including two boys who died from liver failure after taking Elevidys.

Elevidys remains approved in the U.S. for children aged four and older with Duchenne, including those who can no longer walk. However, its use in that group was granted under accelerated approval and is now being re-evaluated.

Meanwhile, Europe’s drug regulator recommended on Thursday that Elevidys not be approved for use in the EU. Roche, which filed for the approval, said it was disappointed by the outcome but intends to keep working with European regulators. 

"We are disappointed by the CHMP’s (Committee for Medicinal Products for Human Use) negative opinion, given the urgent need for disease-modifying therapies for children in the EU living with Duchenne," said Levi Garraway, Chief Medical Officer at Roche.

On Stocktwits, retail sentiment for Sarepta was ‘extremely bullish’ amid ‘high’ message volume, which shot up by 65% on Sunday.

Sarepta’s stock has declined 90.4% so far in 2025.

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