Anvisa and Roche Cancel Brazil’s Conditional Approval for Elevidys

Overview

• Anvisa published the cancellation on Monday, Aug. 24, 2026, saying the totality of data submitted for the gene therapy Elevidys did not meet Brazil’s regulatory requirements and that Roche requested the withdrawal.
• Elevidys is a one‑time gene therapy for Duchenne muscular dystrophy that uses a viral vector to deliver a shortened dystrophin gene, and its commercialization in Brazil had been under conditional approval since December 2024 and suspended for safety review in July 2025.
• Regulatory safety concerns include reports of acute liver failure and two deaths in the United States, which prompted Anvisa’s 2025 suspension and led the agency to demand consolidated safety data focused on hepatic risk.
• About ten Brazilian patients received Elevidys between December 2024 and July 2025; Roche must continue active safety monitoring and reporting for those patients and says it will keep running clinical programs and submit further evidence.
• The decision intensifies pressure on families and courts seeking access, raises questions about cost and public funding for ultra‑expensive advanced therapies, and could prompt tighter post‑market data requirements even as Anvisa said it may fast‑track any fully compliant future submission.