The U.S. FDA has granted Novartis' gene therapy Itvisma approval for the treatment of spinal muscular atrophy (SMA) in children aged two years or older with an SMN1 mutation. The approval allows Itvisma to be the first CNS-directed gene replacement therapy for a broader SMA group, thus extending Novartis' treatment choices beyond IV Zolgensma. The company is already preparing for commercial launch and doing market access activities.
Source Reference:
FDA Official Press Release:
https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-itvisma-spinal-muscular-atrophy
