WASHINGTON, D.C. — The U.S. Food and Drug Administration approved Itvisma, a new adeno-associated virus vector-based gene therapy for spinal muscular atrophy, extending treatment eligibility to adults and children 2 years of age and older with confirmed mutations in the SMN1 gene.
The decision marks a major expansion in SMA care. Until now, the only FDA-approved gene therapy for the disorder — Zolgensma — was limited to patients under 2 years old. Itvisma uses the same active ingredient but is delivered through a single intrathecal injection directly into the central nervous system, independent of patient weight, allowing the treatment to reach motor neurons with a smaller dose.
FDA officials said the approval represents a breakthrough for patients living with a rare, devastating neurodegenerative disease that causes progressive muscle weakness, paralysis, and, in the most severe cases, early death.
“Today’s approval shows the power of gene therapies and offers treatment to patients across the SMA disease spectrum,” said Vinay Prasad, M.D., the FDA’s Chief Medical and Scientific Officer and Director of the Center for Biologics Evaluation and Research. He said the agency remains committed to accelerating therapies for conditions with significant unmet need.
SMA affects roughly 4 to 10 per 100,000 live births in the United States and was once among the leading genetic causes of infant mortality. Itvisma demonstrated substantial evidence of effectiveness in a Phase 3 study, supported by mechanistic data and prior safety and efficacy findings from Zolgensma.
The FDA noted that the expanded indication to adult patients is justified but carries additional risks. Regulators warned that individuals with preexisting chronic medical conditions may face higher risks of hepatotoxicity or cardiotoxicity. The Itvisma label includes a boxed warning for liver injury similar to that found on Zolgensma, adjusted to reflect new clinical findings.
By delivering gene therapy directly into cerebrospinal fluid, Itvisma targets the root cause of SMA with rapid onset, restoring SMN protein production and stopping further disease progression, according to the agency.
The FDA worked closely with Novartis Gene Therapies to leverage existing safety data from Zolgensma, expediting review through multiple regulatory pathways, including Fast Track, Breakthrough Therapy, Priority Review, and Orphan Drug designations.
“Significant unmet need remains in SMA, particularly for patients across various ages and motor function levels,” said Vijay Kumar, M.D., Acting Director of the Office of Therapeutic Products. He called the approval an important step forward for individuals long excluded from gene-based options.
Itvisma is manufactured by Novartis Gene Therapies, Inc.
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