The controversy surrounding embattled gene therapy company Sarepta Therapeutics, including its drug, Elevidys, for Duchenne muscular dystrophy, continues to captivate industry observers—with the latest twist being Sarepta’s decision late Monday to voluntarily pause Elevidys shipments in the US.

A back-and-forth affair

Monday's action marked an about-face by the company from just a few days earlier, when it rejected the FDA’s request to voluntarily halt shipments following the reported deaths of three patients. Sarepta, in a statement Friday, commented: “Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship Elevidys to the ambulant population.” The company noted as well, while acknowledging the tragic nature of the most recent death—a 51-year-old non-ambulant limb-girdle muscular dystrophy patient (LGMD)—the event occurred in a Phase I clinical trial for SRP-9004, a clinical-stage gene therapy “intended to treat a different disease (LGMD type 2D), is administered using a different dose, and is manufactured using a different process.”1

Nevertheless, Sarepta subsequently reversed course. Its Big Pharma partner, Roche, in news reported yesterday,² is also stopping shipments in certain countries amid escalating safety concerns surrounding Elevidys.

To help you keep pace with these and preceding twists and turns in this saga, the following is a roundup of the key developments, the impact and context for Sarepta and FDA, and the potential broader implications that may impact gene therapy regulation.

Timeline and key events

FDA alert and request for halt

Multiple patient deaths—two non‑ambulatory teens treated with Elevidys and a 51‑year‑old adult in a separate LGMD trial—were attributed to acute liver failure. In response, the FDA (on July 18, 2025) formally asked Sarepta to suspend all Elevidys shipments and placed its LGMD gene therapy trials on clinical hold, citing safety concerns tied to the shared adeno-associated virus (AAV)rh74 viral vector.3

Sarepta pushback

Initially, as mentioned, Sarepta declined the request—continuing to ship Elevidys to ambulatory patients—arguing no new safety signals emerged in that group.4

Major market fallout

Following the third death announcement and FDA pressure, Sarepta’s stock plunged ~36% in one day and was downgraded by several analysts. Concerns over credibility and regulatory risk intensified.5

Sarepta reverses course

As referenced, by late Monday, Sarepta agreed to pause all US shipments of Elevidys—effective at market close on Tuesday—with hopes of rebuilding FDA relations while updating its label and risk‑management protocols.6

Global repercussions

Roche, responsible for ex‑US distribution, also halted shipments, as mentioned, in some markets that rely on FDA approval, although ongoing supplies remain in regions such as Japan and United Arab Emirates.7

Deeper context

Regulatory strategy under fire

The FDA not only revoked Sarepta’s AAVrh74 platform designation (stalling future gene therapies), but critics argue the agency may escalate to full market withdrawal—especially under growing public scrutiny.8

Safety and labeling updates

Sarepta is adding a bolded liver-risk warning and is investigating ways (such as sirolimus co-therapy) to mitigate liver toxicity, while the FDA evaluates risks even for ambulatory patients.9

Pipeline and restructuring fallout

On July 16, Sarepta axed 500 positions (~36% of workforce) in a major restructure aimed at saving $400 million a year. It also halted most LGMD programs, though SRP‑9003 remains under discussion.10

Financial stability in question

With revenue hinging heavily on Elevidys, the pause threatens a reported $500 million a year in sales. Sarepta faces about $1 billion in debt due in 2027, convertible bonds trading at deep discounts, and the risk of covenant breaches or funding disruptions.10

Industry and investor pressure

From doctors, hospitals, and analysts to patients and families, sentiments range from cautious optimism (with updated plans and transparency) to deep concern about delayed safety communication and lingering trust issues.11

Wider implications: Gene therapy regulation

Regulatory trust and oversight

The deaths tied to AAVrh74 (used in Elevidys and other Sarepta programs) may prompt the FDA to reevaluate safety assumptions across all AAV platforms, especially in large doses or non-ambulatory populations.

Other companies using AAV vectors may now face longer review cycles, enhanced preclinical demands, or clinical holds.

Platform-based caution

The FDA’s decision to revoke Sarepta’s AAVrh74 platform designation—once a fast-track advantage—signals a move away from platform generalizations toward individualized trial data per therapy, even within the same vector or capsid family.

Market access and labeling

Sarepta’s reversal and relabeling will likely set a precedent for boxed warnings and narrowed indications (e.g., ambulatory patients only), unless long-term safety data is robust.

Risk evaluation and mitigation strategies (REMS)

Expect broader implementation of REMS programs for gene therapies—requiring specialized training for providers, tighter control over distribution, and consent mechanisms addressing liver risks.

Potential for market withdrawals

The Elevidys episode could embolden the FDA to mandate full withdrawals of gene therapies that don’t meet evolving safety standards—even if efficacy remains promising.

Investor caution in gene therapy

Sarepta’s value collapse (~36% stock drop in one day) highlights the volatility of single-asset gene therapy bets, prompting investors to push for diversified pipelines and conservative assumptions.

Slowed approvals

The FDA may extend review timelines or request post-hoc subgroup analyses for already-approved gene therapies to reassess benefit-risk profiles, delaying revenue generation.

More complex CMC reviews

Chemistry, manufacturing, and controls specs for AAV vectors may now face deeper audits—especially regarding dose precision, batch consistency, and vector purity.

References

1. Sarepta Therapeutics Announces Voluntary Pause of ELEVIDYS Shipments in the US. Sarepta press release. July 21, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys

2. Joseph, Andrew. Roche Halts Some Shipments of Sarepta’s Duchenne Gene Therapy Outside US. STAT. July 23, 2025. https://www.statnews.com/2025/07/23/roche-sarepta-gene-therapy-elevidys-duchenne-safety/

3. FDA Investigating Deaths Due to Acute Liver Failure Following Treatment with Sarepta’s AAVrh74 Gene Therapies. FDA. July 18, 2025. https://www.fda.gov/vaccines-blood-biologics/safety-availability-biologics/fda-investigating-deaths-due-acute-liver-failure-following-treatment-sareptas-aavrh74-gene-therapies?

4. Gatlin, A. Sarepta's 'Unprecedented Times' Continue As Company Rebukes The FDA. Investor's Business Daily. July 21, 2025. https://www.investors.com/news/technology/sarepta-stock-elevidys-gene-therapy-fda-request/

5. Walker, J. Sarepta Therapeutics Stock Plunges After Another Patient Death Is Linked to One of Its Gene Therapies. The Wall Street Journal. July 18, 2025. https://www.wsj.com/livecoverage/stock-market-today-dow-sp-500-nasdaq-07-18-2025/card/sarepta-therapeutics-stock-plunges-after-another-patient-death-is-linked-to-one-of-its-gene-therapies-Ghp9p3b7Jphm9hgRkcv9?gaa_at=eafs&gaa_n=ASWzDAisnCyd-kVligmKhT3qtUvjPBNh6icBuR8UxYB8jExqYDDy0XUkqau05ZFGbEM%3D&gaa_ts=688147c3&gaa_sig=9-3IC2xX_6aP_QwsblrM4T1SiXwi676NV7MzWxwQaI42FmaPVYFPNzxu7gvZqXqt3CTHH6RWT5tbndAypuaGaw%3D%3D

6. Manalac, T. Sarepta Concedes to FDA Request, Suspends US Shipments of Elevidys. BioSpace. July 22, 2025. https://www.biospace.com/business/sarepta-concedes-to-fda-request-suspends-us-shipments-of-elevidys

7. Roche Pauses Shipments of Elevidys Gene Therapy Outside US. Reuters. July 23, 2025. https://www.reuters.com/sustainability/boards-policy-regulation/roche-pauses-shipments-elevidys-gene-therapy-outside-us-2025-07-22/

8. Kransteiner, F.; Masson, G. FDA Mulls Elevidys Market Withdrawal Following 3rd Death After a Sarepta Gene Therapy. Fierce Pharma. July 18, 2025. https://www.fiercepharma.com/pharma/fda-mulls-elevidys-market-withdrawal-following-3rd-death-after-sarepta-gene-therapy

9. Handt, J.; Maynard, K. We are Mothers of Duchenne Patients. Recent Setbacks with Sarepta Must Not Stop Progress. STAT. July 18, 2025. https://www.statnews.com/2025/07/18/sarepta-elevidys-deaths-duchenne-muscular-dystrophy-dmd-mothers-research-progress/

10. Fidler, B.; Pagliarulo, N. 5 Questions on Sarepta, the FDA, and a Duchenne Gene Therapy Crisis. BioPharma Dive. July 22, 2025. https://www.biopharmadive.com/news/sarepta-fda-elevidys-duchenne-gene-therapy-crisis-questions/753515/

11. Peebles, A.; Constantino, A.K.; Pramuk, J. Sarepta Shares Plunge 40% as Future of Its Gene Therapy Appears at Risk. CNBC. July 18, 2025. https://www.cnbc.com/2025/07/18/sarepta-stock-falls-on-fda-gene-therapy-elevidys-comment.html