The FDA approved Novartis’ Itvisma (onasemnogene abeparvovec-brve), the company’s first and only gene replacement therapy for spinal muscular atrophy in children 2 and older, as well as for teens and adults.
The therapy is designed to address the genetic root cause of SMA by delivering a functional copy of the SMN1 gene through a single, fixed intrathecal dose. Itvisma is approved for patients with a confirmed SMN1 gene mutation and does not require dosing adjustments based on age or body weight, according to a Nov. 24 news release from the FDA.
Approval was based on results from the phase 3 STEER and phase 3b STRENGTH studies, which demonstrated significant motor function improvement and stabilization over 52 weeks of follow-up, according to a Nov. 24 news release from Novartis.
Approximately 9,000 people in the U.S. are living with SMA, according to the company release. Novartis said Itvisma may reduce the need for chronic treatment among eligible patients and is expected to be available in the U.S. in December.
