Sarepta Gets Thumbs Down From Regulators Regarding Elevidys Gene Therapy

Core Opinion - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization for Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD) in ambulatory children aged three to seven years [1][2]. Group 1: Clinical Data and Efficacy - The CHMP's opinion was based on data from the largest gene therapy clinical program for DMD, including the pivotal Phase 3 EMBARK study, which indicated that Elevidys provided sustained stabilization or slowing of disease progression [2]. - Over 900 individuals with DMD have been treated with Elevidys, with 760 being ambulatory; although the primary endpoint was not met in EMBARK after one year, significant improvements were observed in secondary endpoints compared to placebo [3]. - Longer-term efficacy data submitted to the EMA included two-year results from the EMBARK study and a three-year pooled efficacy analysis from other studies, showing clinically meaningful improvements in motor function [4]. Group 2: Regulatory and Market Impact - Sarepta Therapeutics announced a voluntary and temporary pause of all U.S. shipments of Elevidys effective July 22, following a senior FDA official's statement that additional clinical data would be required to validate Elevidys' safety profile [4][5]. - Analysts expressed uncertainty regarding the FDA's requirements for resuming shipments, noting that requiring another trial for ambulatory patients would be unusual given Elevidys' existing full approval for that group [6]. - The stock price of Sarepta Therapeutics fell by 7.53% to $11.91, reflecting market pressure due to the ongoing uncertainty surrounding the product's status [9]. Group 3: Partnerships and Financial Implications - Arrowhead Pharmaceuticals reported that Sarepta has faced setbacks in unrelated products and programs, but expects to earn the first $100 million soon and the remaining $200 million by year-end [8]. - Should Sarepta fail to meet payment milestones associated with the enrollment of ARO-DM1 cohorts, Arrowhead would have the right to terminate the agreement regarding ARO-DM1 [9].