Sarepta Therapeutics' Reorganization Boosts Investor Confidence

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) stock is trading higher on Thursday after the company issued an after-hours update on ongoing label expansion efforts for Elevidys, its approved gene therapy for Duchenne muscular dystrophy.

The U.S Food and Drug Administration (FDA) has requested that the label include a black box warning for acute liver injury (ALI) and acute liver failure (ALF).

Sarepta agrees with this change, which appears to resolve any material issues with the ambulant portion of the Elevidys label. In June, Sarepta temporarily suspended shipments of Elevidys for non-ambulatory patients.

Also Read: Did The FDA Make A Mistake? Sarepta’s Elevidys Approval Under Scrutiny

A Sarepta committee is reviewing cases of ALF and exploring additional immunosuppression regimens. The committee agreed on an enhanced immunosuppressive regimen with sirolimus for Elevidys in non-ambulant patients.

Sarepta will submit the expert panel’s findings and proposed protocol to the FDA and will discuss a proposal to gather data on the regimen in a new cohort (Cohort 8) of the ENDEAVOR study (Study SRP-9001-103) as a pathway to re-establish dosing in the non-ambulant setting.

Additionally, Sarepta is assessing real-world data generation opportunities for ambulant patients through ...