ARTICLE
The FDA approved the first gene therapy for pediatric patients with spinal muscular atrophy (SMA). The treatment comes with a wholesale acquisition cost of $2.125 million, making it the most expensive drug in the world. The cost can be spread out over a 5-year installment plan, drugmaker Novartis said. The treatment, known as onasemnogene abeparvovec-xioi (Zolgensma), is approved for children under age 2 with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. The therapy targets SMA by replacing a defective or missing SMN1 gene with a single, one-time intravenous infusion to deliver a fully functional copy into target motor neuron cells. Data from the ongoing phase III STRIVE trial showed children with SMA Type 1 experienced prolonged event-free survival, increases in motor function, and significant milestone achievements with the drug. The most common side effects seen in the STRIVE trial were elevated liver enzymes and vomiting. The drug comes with a boxed warning that acute serious liver injury can occur. Source: https://www.medpagetoday.com/neurology/generalneurology/80076