SRPT Stock Down After EMA Places Clinical Hold on DMD Gene Therapy

Shares of Sarepta Therapeutics SRPT dropped more than 6% yesterday after the EMA placed a clinical hold on all studies evaluating Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD). The therapy has been developed in collaboration with pharma giant Roche RHHBY.

This hold has been placed in response to a patient’s death post-treatment with Elevidys, which was reported last month. Though the death was due to acute liver failure (a known risk of Elevidys), Sarepta believes that the patient’s recent CMV infection might have contributed to the outcome. Investigation is still ongoing.

In a letter to the World Duchenne Organization, Roche mentioned that the companies are pausing enrollment and dosing of study participants in three studies evaluating the therapy at the EU study sites. This hold will remain ‘until the analysis into the cause of death is complete.’

The paused studies include Sarepta-sponsored phase III ENVISION study (in boys aged 8-17 years), the Roche-sponsored phase II ENVOL study in infants and newborns, and Sarepta’s early-stage study in boys aged 4 to 9 years with pre-existing antibodies to a specific serotype. Roche has also paused enrolment and dosing for the ENVOL study being conducted at the U.K. study sites.

SRPT and RHHBY entered into a licensing agreement in 2019 to develop Elevidys. Per the agreement, Sarepta retains the rights to market the gene therapy in the United States while Roche has exclusive rights to launch and market the therapy outside the country.

SRPT Stock’s Performance

The downside in Sarepta stock was likely due to concerns that this latest development could make doctors even more hesitant to prescribe Elevidys, negatively impacting the therapy’s overall market adoption in the United States. Also, the clinical hold further delays the launch plans for the gene therapy in the European Union.

Elevidys is a major contributor to Sarepta’s topline. The therapy’s sales accounted for nearly 60% of the company’s total revenues in the fourth quarter of 2024. For full-year 2025, SRPT expects net product revenues in the range of $2.9-$3.1 billion, two-thirds of which are expected to come from Elevidys’ sales. Any further negative developments concerning the gene therapy could adversely impact both SRPT’s growth trajectory and stock performance.

Year to date, Sarepta’s shares have plunged nearly 52% against the industry’s 1% growth.

Sarepta’s Strong Commercial DMD Portfolio

Sarepta’s portfolio consists of four approved therapies, all targeting DMD indications.

Currently, Elevidys is the first and only one-shot gene therapy for DMD in the United States. Since its commercial launch in June 2023, the gene therapy has demonstrated blockbuster potential. Sarepta added about $821 million from Elevidys sales in 2024 compared with $200 million in the year-ago period.

Apart from Elevidys, Sarepta has three other therapies in its commercial portfolio targeting the DMD patient population. Exondys 51 is the first approved disease-modifying therapy for DMD in the United States as well as the company’s first product to receive marketing approval. Exondys 51 recorded impressive sales growth in the past few quarters despite the COVID-19 pandemic. Vyondys 53 and Amondys 45 have also seen strong demand since their launch. Per management, these three drugs have the potential to address nearly a third of all patients with DMD in the United States.

SRPT’s Zacks Rank

Sarepta currently carries a Zacks Rank #3 (Hold).

Sarepta Therapeutics, Inc. Price

Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote

Key Picks Among Biotech Stocks

Some better-ranked stocks from the sector are ANI Pharmaceuticals ANIP and CytomX Therapeutics CTMX, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2025 earnings per share (EPS) have risen from $5.54 to $6.35. EPS estimates for 2026 have increased from $6.75 to $7.21 during the same period. Year to date, shares of ANIP have risen 24%.

ANIP’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 17.32%.

In the past 60 days, estimates for CytomX Therapeutics’ 2025 EPS have improved from a loss of 31 cents to profit of 25 cents. During the same timeframe, estimates for loss per share for 2026 have narrowed from 65 cents to 31 cents. Year to date, shares of CytomX have lost 47%.

CTMX’s earnings beat estimates in three of the trailing four quarters and missed the mark once, delivering an average surprise of 180.70%.

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This article originally published on Zacks Investment Research (zacks.com).

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