Zolgensma is an adeno-associated virus vector-based gene therapy.
Drugs in the Pipeline
The BLA submission is supported by data from the PATHFINDER clinical program.
The Food and Drug Administration (FDA) has approved Saphnelo (anifrolumab-fnia) for the treatment of adults with moderate to severe systemic lupus erythematosus who are receiving standard therapy. Type I interferons (IFNs) play a role in the pathogenesis of systemic lupus erythematosus (SLE). Anifrolumab-fnia, a human immunoglobulin G1 kappa monoclonal antibody, binds to subunit 1 of…
The BLA submission included data from the pivotal phase 3 NAVIGATOR trial that assessed tezepelumab in 1061 patients with severe, uncontrolled asthma.
The approval was based on data from the randomized, double-blind, placebo-controlled phase 3 VOYAGE trial.