BRIEF—Novartis files for US approval for Zolgensma

The US regulator has accepted a Biologics License Application from Novartis for Zolgensma (onasemnogene abeparvovec-xxxx), an investigational gene replacement therapy for the treatment of spinal muscular atrophy Type 1.

The therapy has previously received Breakthrough Therapy designation and has also been granted Priority Review, and a decision is expected by May 2019.

Novartis has also filed for approval in Japan and in Europe.