Sarepta Gets FDA Green Light to File for Full DMD Drug Approvals

By FieldPulse Staff · March 19, 2026

Tags: launch, strategy

FDA confirms Sarepta can file supplemental NDAs for DMD drugs Vyondys and Amondys using 9-year ESSENCE data plus real-world evidence, averting potential withdrawal after confirmatory trial miss.

Sarepta Therapeutics announced on March 19, 2026 that the FDA confirmed the company can submit data from its 9-year ESSENCE confirmatory study — along with real-world evidence — in support of converting its Duchenne muscular dystrophy (DMD) exon-skipping drugs Vyondys (golodirsen) and Amondys (casimersen) from accelerated to full approval.

The move was seen as a significant lifeline for both drugs.

Sarepta had reported in November 2025 that Vyondys and Amondys failed to hit their confirmatory trial endpoints, raising the prospect of accelerated approval withdrawal.

The FDA's indication that it will consider supplemental NDA submissions using the broader data package — including 9 years of real-world outcomes — averts that scenario for now.

Sarepta's gene therapy Elevidys remains on the market under full approval for ambulatory patients under 8.

The exon-skipping portfolio serves a broader patient population and represents significant recurring revenue for the company's field team.

Source: https://www.fiercepharma.com/pharma/fierce-pharma-regulatory-tracker-2026