Sarepta Closes Enrollment in Elevidys ENVISION Trial After More Than a Year on Hold

Sarepta Therapeutics is done enrolling patients in ENVISION, the Phase 3 trial of its Duchenne muscular dystrophy gene therapy Elevidys in non-ambulatory and late-ambulatory patients. The company disclosed the decision in a community letter around its third-quarter earnings report on Thursday, Nov. 5.

The trial has been on global hold for more than a year.

Why Sarepta pulled the plug

ENVISION was built to enroll 90 participants across 14 countries: 64 non-ambulatory and 26 late-ambulatory. Sarepta says that after weighing the length of the hold and the extra safety information needed to treat non-ambulatory patients, restarting would require "significant changes" to the original design. The company called that "ultimately not feasible."

Instead, Sarepta will close enrollment and analyze the blinded data collected in Part 1. The company says the goal is to share findings with the Duchenne community sooner. It has not given a date.

Trial sites are working with enrolled families on individual next steps and transition plans, according to Sarepta. The Parent Project Muscular Dystrophy, which relayed the letter, called the decision "disappointing news" for participants and families.

What replaces it

Sarepta says it remains committed to finding a treatment path for non-ambulatory patients. It continues to enroll them in ENDEAVOR Cohort 8, which tests an enhanced safety approach: oral sirolimus given alongside corticosteroids.

The company plans to meet with the FDA in the first quarter of 2027 to discuss that data and a possible U.S. path forward.

Tech Times, which covered the announcement, reported that Cohort 8 holds about 25 patients and is an open-label safety study, not one designed to demonstrate clinical benefit. Its reporting argues that data from such a small cohort will have to persuade an FDA that has been strict about confirmatory evidence. Sarepta has not said what size or kind of data it expects the agency to accept.

The backdrop is 2025, when multiple deaths were linked to Sarepta's gene therapy programs, including Elevidys. Tech Times reports that FDA removed the non-ambulatory indication from the label in November 2025 after three patients died of acute liver failure. Elevidys sales fell as the benefit-risk profile came under question.

The money picture

Sarepta's Q3 numbers show a company that has stopped the bleeding but is not growing.

• Total revenue was $381.4 million, down 5% from $399.4 million a year earlier.
• Product revenue was $324 million, down 13%. Elevidys brought in $83 million, and the PMO exon-skipping franchise $240 million.
• Collaboration and other revenue was $58 million, mainly contract manufacturing of Elevidys supply sold to Roche.
• Cost of sales rose 25% to $189 million, including $52 million in Elevidys take-or-pay charges, which cut product gross margin to 59%.
• GAAP operating loss was $9.0 million. Non-GAAP operating income was $22.9 million.
• GAAP net loss was $28.5 million, or $0.27 per share.
• Cash and investments ended the quarter near $1 billion.

For the first nine months, revenue was $1.51 billion, down 14% from $1.76 billion. GAAP net income for the period was $297.6 million, against a $301.2 million loss in the same period of 2025.

Sarepta narrowed its 2026 product revenue guidance to $1.25 billion to $1.30 billion, raising the low end by $50 million. Management expects Q4 Elevidys revenue to land broadly in line with Q3, while acknowledging variability for a one-time gene therapy.

A new CEO with a long list

Michael Severino, formerly an executive at AbbVie and Amgen, was on his first earnings call as CEO after 90 days in the job. In the release, he said the company has "a durable Duchenne franchise, a promising pipeline" and the financial strength to invest. He added: "We have work to do, and I want us to be measured by execution."

An analyst pressed him on Sarepta's "mixed" record of clean clinical trial wins. Severino answered that a positive study requires "the most substantial effect size you can" get. He said the company has learned how to pick strong neuromuscular sites and that its study design and statistical capabilities "are as good here as they are anywhere."

Sarepta's pipeline now leans on siRNA programs from its Arrowhead deal, signed in November 2024. Multiple ascending dose data for SRP-1003 in myotonic dystrophy type 1 and SRP-1001 in facioscapulohumeral muscular dystrophy are expected later in 2026. The company says it is preparing both for potential late-stage development.

Exon skippers also face a verdict

Sarepta also has a regulatory decision pending on its older drugs. The FDA's target date for traditional approval of Amondys 45 and Vyondys 53 is the end of February 2027. Both drugs received accelerated approval earlier, and BioSpace reports that both missed their confirmatory trials in November 2025. Sarepta resubmitted anyway.

What comes next

Two dates now matter: the Q1 2027 FDA meeting on non-ambulatory Elevidys, and the end-of-February 2027 decision on the exon skippers. Sarepta has not said when blinded ENVISION Part 1 results will be released, and the FDA has not publicly said what evidence would reopen access for non-ambulatory patients in the U.S.