The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve), a gene therapy for spinal muscular atrophy (SMA), for use in patients aged two years and older with a confirmed mutation in the SMN1gene.
Itvisma delivers a functional copy of the SMN1 gene using an adeno-associated virus (AAV) vector directly into the cerebrospinal fluid via a single intrathecal injection. Because the therapy is administered into the central nervous system, it avoids the need for weight-based dosing.
Clinical evidence from a Phase 3 study demonstrated significant improvement in motor function, while additional support came from data on Zolgensma, which uses the same active ingredient.
The FDA noted that safety concerns include liver toxicity and cardiac issues, especially in adults with existing medical conditions. These risks are consistent with those observed in Zolgensma’s use.
The FDA regulators granted the therapy Fast Track, Breakthrough Therapy, and Priority Review designations. Itvisma also received Orphan Drug status, reflecting its role in treating a rare disorder.
With this approval, Itvisma becomes the first gene replacement therapy for a broader SMA population beyond infants, offering a treatment option to older children and adults previously ineligible for other gene therapies.
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