Sarepta Therapeutics Faces FDA Challenges Amid Executive Departure, Analysts See 94.98% Upside Potential

Sarepta Therapeutics (SRPT) faces FDA challenges after the suspension of its Elevidys treatments due to safety concerns. Despite this, Wall Street analysts predict a substantial potential upside for SRPT's stock with a diverse range of price targets. GuruFocus estimates show a significant valuation gap, suggesting promising long-term investment potential.

Sarepta Therapeutics (SRPT) has been facing significant regulatory hurdles following the suspension of its Elevidys treatments due to safety concerns. Despite these challenges, Wall Street analysts predict a substantial potential upside for SRPT's stock, with a diverse range of price targets. GuruFocus estimates show a significant valuation gap, suggesting promising long-term investment potential.

The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) recently issued an opinion recommending against conditional marketing authorization for Sarepta’s Duchenne muscular dystrophy (DMD) adeno-associated virus (AAV) vector-based gene therapy delandistrogene moxeparvovec-rokl (marketed as Elevidys) for patients who are ambulatory [1]. Roche, which has commercial rights for the gene therapy product in markets outside the United States, expressed disappointment with the CHMP’s decision [2].

The CHMP’s opinion was based on data from clinical trials for Elevidys, including the pivotal phase 3 EMBARK clinical trial (NCT05096221). While the primary end point was not met, Roche noted statistically significant and clinically meaningful improvements on functional outcome measures compared to placebo [2]. Safety data indicated a consistent and manageable safety profile for ambulatory patients in the trial.

Following the CHMP’s decision, Roche stated its intent to continue discussions with the EMA to explore bringing Elevidys towards authorization in the EU. Notably, Elevidys and some of Sarepta’s other muscular dystrophy gene therapies have faced challenges following recent deaths of several patients [3-6]. The FDA had informally requested that Sarepta put shipment of all Elevidys product in the United States on hold, a request that Sarepta initially resisted but later complied with [3-6]. After determining that the death of an 8-year-old boy treated with Elevidys in Brazil was unrelated to the gene therapy, the FDA recommended resuming shipment of Elevidys for ambulant patients [4].

However, Sarepta had paused shipments to nonambulant patients after two deaths attributed to acute liver failure [5-7]. The FDA continues to work with Sarepta regarding nonambulatory patients, which remains subject to a voluntary hold [3]. Additionally, the FDA revoked the platform technology designation for the AAVrh74 viral vector used in Elevidys and placed holds on all trials evaluating Sarepta’s investigational gene therapy products SRP-9004 and SRP-9003 following the death of a 51-year-old patient treated with SRP-9004 [8-10].

Despite these challenges, Wall Street analysts have expressed optimism about SRPT’s stock. GuruFocus estimates show a significant valuation gap, suggesting promising long-term investment potential. Analysts predict a substantial potential upside for SRPT's stock, with a diverse range of price targets.

References:
[1] Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU. News release. Roche. July 24, 2025. Accessed July 31, 2025. https://www.roche.com/media/releases/med-cor-2025-07-25
[2] DMD gene therapy Elevidys dealt another blow, this time from EU panel. News article. Matthew Dennis. FirstWord PHARMA. July 25, 2025. Accessed July 31, 2025. https://firstwordpharma.com/story/5983866
[3] FDA recommends removal of voluntary hold for Elevidys for ambulatory patients. News release. FDA. July 28, 2025. Accessed July 31, 2025. https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients
[4] FDA investigating death of 8-year-old boy who received Elevidys. News release. FDA. July 25, 2025. Accessed July 31, 2025. https://www.fda.gov/news-events/press-announcements/fda-investigating-death-8-year-old-boy-who-received-elevidys
[5] Sarepta Therapeutics provides clarifying statement on Elevidys. News release. Sarepta Therapeutics, Inc. July 25, 2025. Accessed July 31, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-clarifying-statement-elevidys
[6] Sarepta Therapeutics provides statement on Elevidys. News release. Sarepta Therapeutics, Inc. July 18, 2025. Accessed July 31, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys
[7] FDA informs sarepta that it recommends that sarepta remove its pause and resume shipments of ELEVIDYS for ambulatory individuals with duchenne muscular dystrophy. News release. Sarepta Therapeutics, Inc. July 28, 2025. Accessed July 31, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/fda-informs-sarepta-it-recommends-sarepta-remove-its-pause-and
[8] Philippidis A. StockWatch: Sarepta shares nosedive after LGMD gene therapy patient dies. Genetic Engineering & Biotechnology News. July 20, 2025. Accessed July 31, 2025. https://www.genengnews.com/topics/genome-editing/stockwatch-sarepta-shares-nosedive-after-lgmd-gene-therapy-patient-dies/
[9] FDA requests Sarepta Therapeutics suspend distribution of Elevidys and places clinical trials on hold for multiple gene therapy products following 3 deaths. News release. FDA. July 18, 2025. Accessed July 31, 2025. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold
[10] U.S. FDA grants platform technology designation to the viral vector used in SRP-9003, Sarepta’s investigational gene therapy for the treatment of limb girdle muscular dystrophy type 2E/R4. News release. Sarepta Therapeutics Inc. June 4, 2025. Accessed July 31, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/us-fda-grants-platform-technology-designation-viral-vector-used