This Week’s Biopharma News: New Restrictions on Sarepta Gene Therapy
November 18, 2025
-
5 min
The FDA has restricted Sarepta's Elevidys gene therapy, which is now limited to ambulatory patients aged four and older with a confirmed Duchenne muscular dystrophy mutation, following reports of severe liver injuries. Additionally, a new regulatory pathway for personalized therapies is in development, allowing innovative treatments targeting specific biological mechanisms. Advancements in non-viral cell therapy manufacturing are being pursued by Kytopen and Excellos while Sandoz secures licensing for a biosimilar to pertuzumab for breast cancer. Meanwhile, Novartis achieved promising results from its malaria treatment phase III trial, highlighting a potential breakthrough in the field.
1. Sarepta's Elevidys gene therapy now indicated for ambulatory patients. 2. New FDA boxed warning for acute liver injury. 3. Novel pathway for personalized therapies introduced by FDA. 4. Kytopen and Excellos collaborating on non-viral cell therapy methods. 5. Sandoz gains rights for a pertuzumab biosimilar for breast cancer. 6. Novartis reports successful Phase III trial for KLU156 against malaria.
Listen Tab content
