Sarepta Therapeutics announced on 16 July it would add a US Food and Drug Administration (FDA)-requested black box warning for acute liver injury/failure to its Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec), resolving disputes over ambulant patient use. However, the company continued shipping Elevidys despite an 18 July FDA halt request, citing no new safety signals in ambulant patients. The conflict escalated when Sarepta revealed a separate fatal liver failure case in a Phase I trial of investigational therapy SRP-9004 for limb-girdle muscular dystrophy.

On 21 July, Sarepta reversed course, voluntarily pausing all US Elevidys shipments to complete label negotiations. The dispute unfolded alongside Sarepta’s 36% workforce cuts (500 jobs) and USD 400 million cost reduction plan, prioritising siRNA programs. Elevidys generated USD 282 million in Q2 2025, underscoring its commercial importance amid financial pressures. CEO Doug Ingram emphasised maintaining FDA relations while addressing patient access needs.

According to PharmCube's NextBiopharm® database, has only four approved products in its US homeland (Elevidys plus three nucleotide therapies), making Elevidys a key source of revenue. Click here to request a free trial for NextBiopharm®.

Sources:
https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-strategic-restructuring-and
https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys
https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys