Pharma BD Deal Intelligence
In December 2019, Roche licensed ex-US rights to Sarepta's microdystrophin gene therapy program for Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies, led by SRP-9001 (delandistrogene moxeparvovec). Roche paid ~$1.15B upfront (comprising $750M cash plus a $400M equity investment) plus an $85M near-term milestone, with up to $500M in additional milestones and significant ex-US royalties (~$1.735B total disclosed); Sarepta retained US rights and manufacturing while Roche took regulatory approval and commercialization outside the US. Post-close, SRP-9001 was approved by the FDA as ELEVIDYS on June 22, 2023 - the first gene therapy for DMD (accelerated approval, later expanded) - and Roche launched it across ex-US markets including Japan (first commercial sale and a $40M milestone to Sarepta in Q1 2026). In 2025 the program faced a serious safety crisis: deaths from acute liver failure tied to the shared AAVrh74 vector led the FDA to pause US shipments in July 2025, and Roche voluntarily paused ex-US shipments (July 23, 2025) in countries that reference FDA decisions, while continuing supply in independently-approving markets such as Brazil and Japan. The EMA issued a negative opinion in 2025, and Roche announced a new global Phase 3 confirmatory trial on April 16, 2026 to support ex-US use.