{"id":64074,"date":"2026-04-27T21:39:50","date_gmt":"2026-04-27T13:39:50","guid":{"rendered":"https:\/\/flcube.com\/?p=64074"},"modified":"2026-04-27T21:39:51","modified_gmt":"2026-04-27T13:39:51","slug":"novartis-wins-chmp-positive-opinion-for-itvisma-intrathecal-onasemnogene-abeparvovec-in-5q-spinal-muscular-atrophy-across-all-ages-in-eu","status":"publish","type":"post","link":"https:\/\/flcube.com\/?p=64074","title":{"rendered":"Novartis Wins CHMP Positive Opinion for Itvisma (Intrathecal Onasemnogene Abeparvovec) in 5q Spinal Muscular Atrophy Across All Ages in EU"},"content":{"rendered":"\n

Novartis AG<\/strong> (NYSE: NVS<\/a>) announced that the Committee for Medicinal Products for Human Use (CHMP)<\/strong> of the European Medicines Agency (EMA)<\/strong> has issued a positive opinion<\/strong> recommending marketing authorization for Itvisma\u2122 (intrathecal onasemnogene abeparvovec)<\/strong> to treat children, adolescents, and adults aged 2 years and older<\/strong> with 5q spinal muscular atrophy (SMA)<\/strong> due to biallelic SMN1 gene mutations<\/strong>.<\/p>\n\n\n\n

Itvisma is a one-time, intrathecally administered gene therapy<\/strong> based on an adeno-associated virus serotype 9 (AAV9)<\/strong> vector engineered to deliver a functional copy of the human SMN1 gene<\/strong> directly to the central nervous system. By enabling sustained expression of survival motor neuron (SMN) protein<\/strong>, the therapy addresses SMA at its genetic root cause<\/strong>, with clinical data showing meaningful improvements in motor function across age groups.<\/p>\n\n\n\n

Regulatory & Product Profile<\/h2>\n\n\n\n
Parameter<\/th>Detail<\/th><\/tr><\/thead>
Drug Name<\/strong><\/td>Itvisma\u2122 (onasemnogene abeparvovec, intrathecal formulation)<\/td><\/tr>
Indication<\/strong><\/td>5q SMA with biallelic SMN1<\/em> mutations, age \u22652 years<\/td><\/tr>
Mechanism<\/strong><\/td>AAV9-mediated delivery of functional SMN1<\/em> gene via single intrathecal injection<\/td><\/tr>
EMA Status<\/strong><\/td>CHMP positive opinion \u2192 marketing authorization decision expected Q3 2026<\/strong> from European Commission<\/td><\/tr>
U.S. Status<\/strong><\/td>Already approved and marketed as Itvisma<\/strong> (same product, formerly OAV101B)<\/td><\/tr>
Differentiation<\/strong><\/td>First intrathecal<\/strong> (vs. intravenous) AAV9-SMN1 gene therapy approved globally; designed for older\/larger patients where IV dosing faces vector load limitations<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n

Clinical & Strategic Significance<\/h2>\n\n\n\n