This Week’s Biopharma News: New Restrictions on Sarepta Gene Therapy
November 18, 2025
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5 min
6 Key Takeaways
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1
Sarepta's Elevidys gene therapy now indicated for ambulatory patients.
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2
New FDA boxed warning for acute liver injury.
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3
Novel pathway for personalized therapies introduced by FDA.
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4
Kytopen and Excellos collaborating on non-viral cell therapy methods.
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5
Sandoz gains rights for a pertuzumab biosimilar for breast cancer.
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6
Novartis reports successful Phase III trial for KLU156 against malaria.
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.
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