The US FDA has approved onasemnogene abeparvovec-brve, an adeno-associated virus (AAV) vector-based gene therapy, for treating spinal muscular atrophy in adult and pediatric patients aged two years and older with mutations in the survival motor neuron (SMN1) gene. This single, fixed-dose therapy is the first and only gene-replacement option authorized for such a broad patient group. Clinical evidence In a clinical trial involving 136 patients, onasemnogene abeparvovec-brve produced significant improvements in motor function and stabilization of motor abilities, as measured by the Hammersmith Functional Motor Scale – Expanded.
The mean change from baseline was 2.39 compared to 0.51 with sham treatment. Dosage and administration Single-dose intrathecal injection over 1 to 2 minutes Postpone treatment in patients with active infections until the infection has resolved and the patient is…
