The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of Spinal Muscular Atrophy in adult and pediatric patients aged two years and older who carry a confirmed SMN1 gene mutation. The decision expands treatment options across the SMA spectrum by offering a one-time gene therapy designed to correct the underlying genetic cause rather than only easing symptoms.
Itvisma uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the SMN1 gene directly into the central nervous system. The therapy is given as a single intrathecal injection into the cerebrospinal fluid surrounding the spinal cord, a delivery route intended to target motor neurons efficiently with a lower vector dose that does not depend on patient body weight. Although the active genetic construct matches that of Zolgensma, Itvisma’s concentrated formulation and intrathecal administration are tailored for older children and adults.
The FDA granted the application Fast Track and Breakthrough Therapy designations in recognition of the urgent unmet need for patients over two years of age. “Today’s approval shows the power of gene therapies and offers treatment to patients across the SMA disease spectrum,” said Vinay Prasad, M.D., M.P.H., the FDA’s Chief Medical and Scientific Officer, highlighting the clinical relevance of restoring SMN protein production to halt disease progression and improve motor function.
Regulators have also emphasized safety monitoring. Clinical data showed risks of hepatotoxicity and cardiotoxicity, particularly in adults with chronic underlying conditions, and the Itvisma label carries a boxed warning for liver injury. Healthcare providers are advised to monitor liver function closely after administration and remain vigilant for cardiac complications as part of post-treatment follow‑up.
For Pakistan, the approval brings both hope and stark realities. SMA is more common in populations with high rates of consanguineous marriage, but most Pakistani infants are not screened at birth and many cases go undiagnosed. Advocacy groups such as the Strive Eradication of Disability Foundation have raised alarm over the prohibitive cost of advanced gene therapies, describing a “billion-rupee” barrier: single-dose prices run into millions of dollars and are beyond the reach of nearly all families without major government subsidies or international aid.
Local health advocates are urging the Pakistani government to create a national SMA registry, expand newborn screening and engage manufacturers in access pricing talks to make treatments like Itvisma attainable. While the therapy marks a scientific milestone, translating that advance into real-world access in Pakistan will require coordinated policy action, financing solutions and stronger diagnostic infrastructure to ensure patients can benefit from this new option.
