Recently, the FDA has requested Sarepta Therapeutics, Inc., a Massachusetts-based biopharmaceutical company, to suspend all distribution of its DMD gene therapy Elevidys (delandistrogene moxeparvovec), following the deaths of three patients—two non‑ambulatory teenagers and one adult—due to acute liver failure, as well as an 8‑year‑old child whose death was later deemed unrelated to the therapy. Furthermore, the FDA also placed clinical trials for limb‑girdle muscular dystrophy (LGMD) using the same AAVrh74 vector on hold and revoked its platform technology designation for Sarepta’s AAVrh74 gene platform.

This news has caused a cascade of further events, causing turmoil in the whole CGT space.

• On the 16th of July, the company announced layoffs of around 36% of its staff (~500 jobs), as part of a strategic restructuring aimed at focusing on core areas (e.g., siRNA pipeline) and cutting costs toward an estimated $400 million in savings by 2026.
• On the 21st of July, Sarepta agreed to a voluntary hold on Elevidys shipments for non-ambulatory patients, although it initially resisted full suspension.
• Crucially, after investigating the deaths, the FDA determined the 8‑year‑old’s death was unlikely related to Elevidys, and recommended that shipments be resumed for ambulatory (walking) DMD patients.
• The voluntary hold remains in effect for non‑ambulatory patients pending further safety review and data submission.
  

Reimbursement status of Elevidys as of 8/2025

Country	ELEVIDYS – Reimbursed?	ELEVIDYS – List Price / Comment
United States	Yes — commercial / payer coverage available, but conditional	$3.2 M (one-time) — often tied to outcomes- or staged-payment models
Germany	Not reimbursed — CHMP issued a negative EU opinion	No EU list price; future price would be negotiated if approved
France	Not reimbursed — no EU authorization	—
Italy	Not reimbursed — under assessment, no EU authorization	—
Spain	Not reimbursed — no EU authorization	—
United Kingdom	Not routinely reimbursed — NICE appraisal is in progress	—
Japan	Approved — MHLW (conditional/time-limited); reimbursement pending	No public price yet; expected after negotiation
Canada	Approved or under review — public reimbursement requires provincial HTA	No public price; pricing to be negotiated provincially
Brazil	Mixed — ANVISA approved, but sales currently paused due to safety	—
Australia	TGA registration noted — no PBS reimbursement yet	—

• Sarepta’s stock surged substantially, up to 50+%, on news of resumed shipments—although overall shares are still down ~85–90% year‑to‑date due to cumulative setbacks.
  
• Multiple securities class action lawsuits (Bleichmar Fonti & Auld; Levi & Korsinsky; Schall Law Firm; Hagens Berman) have been filed by investors citing material misstatements and stock drop losses stemming from the patient deaths and regulatory disruptions.

Outlook & Next Steps

• Following previous issues with Zolgensma, this is yet another event profoundly shaking investor and payer interest in the CGT space.
  
• Elevidys remains on the market for ambulatory DMD patients, with resuming U.S. and international shipments via partner Roche.
  
• Sarepta is planning a clinical study with enhanced immunosuppression regimens to address ALT/liver injury risks, aiming to lift the hold on non‑ambulatory patients pending FDA review and potential label changes, including a likely black box warning for liver failure risk.

Would you like to take a deeper look at the CGT space? Reach out at info@ambiom.com.

Sources:

(1) Reuters. (2025, July 28). Sarepta resumes shipping of gene therapy Elevidys to patients who can walk. https://www.reuters.com/business/healthcare-pharmaceuticals/sarepta-resumes-shipping-gene-therapy-elevidys-patients-who-can-walk-2025-07-28/

(2) Investopedia. (2025, July 29). Sarepta Therapeutics stock surges as FDA allows Elevidys sales to resume. https://www.investopedia.com/sarepta-therapeutics-stock-surges-as-fda-allows-elevidys-sales-to-resume-11780773

(3) GlobeNewswire. (2025, August 1). SRPT investor reminder: Sarepta Therapeutics, Inc. stock drop leads to class action; investors with losses urged to contact BFA Law by August 25. https://www.globenewswire.com/news-release/2025/08/01/3125782/0/en/SRPT-INVESTOR-REMINDER-Sarepta-Therapeutics-Inc-Stock-Drop-Leads-to-Class-Action-Investors-with-Losses-Urged-to-Contact-BFA-Law-by-August-25-NASDAQ-SRPT.html

(4) U.S. Food & Drug Administration. (2025, July). FDA requests Sarepta Therapeutics suspend distribution of Elevidys and places clinical trials on hold. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold

(5) Parent Project Muscular Dystrophy. (2025, July 29). Sarepta Therapeutics announces update on Elevidys label & company restructuring. https://www.parentprojectmd.org/sarepta-therapeutics-announces-update-on-elevidys-label-company-restructuring/

(6) STAT News. (2025, July 28). FDA lifts some restrictions on Sarepta’s gene therapy for Duchenne. https://www.statnews.com/2025/07/28/sarepta-duchenne-elevidys-hold-ambulatory/

(7) CGTLive. (2025, June). FDA activity recap June 2025: features new platform designation, IND clearance, more. https://www.cgtlive.com/view/fda-activity-recap-june-2025-features-new-platform-designation-ind-clearance-more