Sarepta shares fall amid FDA’s CBER appointment and weak Elevidys gross sales

Sarepta additionally reported lower-than-expected gross sales for its DMD gene remedy Elevidys. Credit score: SOPA Photos through Getty Photos.

Sarepta Therapeutics noticed its inventory drop by 26% on 6 Could after the US Meals and Drug Administration (FDA) introduced that Vinay Prasad will lead its Middle for Biologics Analysis and Analysis (CBER), the division chargeable for regulating gene therapies and vaccines.

The market response mirrored investor considerations over Prasad’s long-standing public criticism of Sarepta’s Duchenne muscular dystrophy (DMD) therapies, together with Elevidys (delandistrogene moxeparvovec-rokl), the corporate’s flagship gene remedy.

Prasad has overtly questioned each the scientific proof supporting Sarepta’s therapies and the FDA’s choice to grant them accelerated approval, a regulatory pathway he has repeatedly criticised.

As well as, the corporate reported Q1 2025 monetary outcomes that missed expectations. Elevidys generated $375m in gross sales, falling wanting Wall Road’s $427m forecast. The corporate additionally decreased its 2025 complete product income steering to between $2.3bn and $2.6bn, from a earlier vary of $2.9bn to $3.1bn. Sarepta CEO Doug Ingram attributed the revised forecast to “headwinds within the quarter,” and informed buyers through the earnings name that Sarepta stays assured within the scientific rigour of its gene remedy programme.

Prasad will change Scott Steele, who has served as interim CBER director since Peter Marks stepped down in March 2025 amid reported stress from the Division of Well being and Human Providers (HHS). The FDA authorised Sarepta’s gene remedy in 2023 beneath the accelerated approval pathway and expanded the label in 2024. Nevertheless, Prasad has accused Marks of reportedly overruling inner FDA employees who raised considerations concerning the supporting knowledge. Prasad, a haematologist-oncologist and professor of epidemiology and biostatistics on the College of California, has been outspoken in his opposition to each choices.

It’s uncommon you can blame a single particular person for a regulatory failure, however Peter Marks at FDA overruled 3 assessment groups and pushed this product available on the market regardless of a failed trial.

He’s additionally the rationale why Biden mandated vaccines b/c he rammed a full approval thorugh, at the same time as… pic.twitter.com/x2nlTkVnV8

— Vinay Prasad MD MPH (@VPrasadMDMPH) March 18, 2025

In a 2024 letter co-authored with Timothée Olivier, Prasad criticised the approval of Elevidys, calling it “probably the most expensive medicine all through historical past”, with a $3.2m price ticket and potential for “extreme unwanted side effects”. He has additionally raised broader considerations concerning the FDA’s use of expedited pathways, arguing that they will result in the approval of therapies with out ample proof of scientific profit.

Throughout the Q1 earnings name, Ingram declined to remark instantly on Prasad’s appointment however stated he had confidence within the FDA: “What I do stay assured about is that the FDA goes to be the FDA that it’s been for the final 100 years, which is an organisation devoted to following nice science.

“There isn’t any purpose to imagine {that a} science-driven organisation and science-minded regulators can be contemplating something apart from the truth that they need to be proud that they approve this sensible remedy.”

Ingram additionally defended the security profile of Elevidys, noting that regardless of a current affected person dying, it stays “probably the most spectacular” amongst adeno-associated virus (AAV)-based gene therapies by way of security. Sarepta had disclosed in March 2025 {that a} teenage boy had died after receiving Elevidys, prompting questions from analysts through the name.

Shares of different gene remedy and uncommon disease-focused biotechs, together with Ultragenyx, Beam Therapeutics, and Verve Therapeutics, additionally declined following the announcement of Prasad’s appointment.

Cell & Gene Remedy protection on Pharmaceutical Know-how is supported by Cytiva.

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