Sarepta Wins FDA Approval To Begin Study On Reducing Liver Injury Risk With Elevidys

• The fresh study cohort will enroll about 25 DMD patients in the U.S. who cannot walk.
• The company intends to initiate the study cohort by the end of this year.
• Elevidys, a medicine intended for the treatment of DMD, created safety concerns following the death of two patients after treatment earlier this year.

Sarepta Therapeutics, Inc. (SRPT) on Tuesday said that the U.S. Food and Drug Administration (FDA) has approved dosing in a study cohort to evaluate the use of an immunosuppressive regimen as part of treatment with Elevidys gene therapy for Duchenne muscular dystrophy patients.

SRPT shares gained nearly 1% on Tuesday morning at the time of writing.

Study Plans

The data from the study cohort will be used to determine whether administering Sirolimus before and after the Elevidys infusion can help reduce acute liver injury in patients who cannot walk, the company said. Siromilus belongs to a group of medicines known as immunosuppressive agents and has shown promise in managing certain aspects of liver disease and its complications.

The study cohort will enroll about 25 patients in the U.S. who are unable to walk. They will be treated with 14 days of peri-infusion Sirolimus dosing before Elevidys administration and 12 weeks after administration.  

“We plan to initiate Cohort 8 by the end of this year and pending enrollment, complete primary endpoint data collection in the second half of 2026,” said Louise Rodino-Klapac, president of research and development and technical operations at Sarepta.

Sarepta’s Rocky Road With Elevidys

Elevidys, a medicine intended for the treatment of Duchenne muscular dystrophy (DMD), created safety concerns following the death of two patients after treatment earlier this year. Both non-ambulatory patients suffered from acute liver failure. DMD is a severe, inherited genetic disorder that causes progressive muscle weakness and degeneration.

While Sarepta resumed shipments to DMD patients who can walk after a temporary pause earlier this year, it is now developing a risk-mitigation approach for patients who cannot walk to resume shipments in this particular patient segment.

However, a Citi analyst said in August that the U.S. Food and Drug Administration may determine that additional steps beyond Siromilus are recommended or required for non-ambulatory Duchenne muscular dystrophy patients on Elevidys. The agency may take a “more comprehensive look” at the level of immunomodulation required for Elevidys, the analyst had then said.

How Did Stocktwits Users React?

On Stocktwits, retail sentiment around SRPT stayed within the ‘bearish’ territory over the past 24 hours, while message volume remained at ‘low’ levels.

SRPT stock is down 84% this year and by 83% over the past 12 months. 

Also See: LNAI Stock Soars Pre-Market After Immune Cell Therapy Gains Licensee Interest

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