Industry Updates
Scholar Rock Resubmits Biologics License Application (BLA) for Apitegromab to the FDA
Today, Scholar Rock announced that it has resubmitted its Biologics License Application (BLA) to the Food and Drug Administration (FDA) for apitegromab, an investigational therapy […]
Biogen Receives FDA Approval of High Dose SPINRAZA for the Treatment of SMA
Biogen today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for the High Dose regimen of SPINRAZA (nusinersen) to […]
Evrysdi Risdiplam Tablet Is Now Approved For Broader Use In SMA
We’re excited to share an important update for the spinal muscular atrophy (SMA) community. The U.S. Food and Drug Administration (FDA) has approved a label […]
Biogen Announces Results from the Phase 2/3 DEVOTE Study
Today, Biogen announced the publication of results from the pivotal Phase 2/3 DEVOTE study in Nature Medicine, highlighting the potential benefits of a high-dose regimen […]
Genentech Releases Winter SMA Community Update Letter
Genentech recently released an update on their continued collaboration with the SMA community and Evrysdi® tablet updates. They will continue to provide timely updates as […]
Cure SMA Shares Updates on the Phase 10 SMA Industry Collaboration
Earlier this year, Cure SMA launched an expanded Phase 10 of the SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together […]
Novartis Receives FDA Approval of Itvisma for the Treatment of SMA
Novartis today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Itvisma (onasemnogene abeparvovec-brve) to treat adults and children 2 years of age and older with […]
Scholar Rock and Cure SMA Share Update on Apitegromab and FDA Meeting
Read the Scholar Rock community statement here. Scholar Rock recently held an in-person Type A meeting with the FDA on November 12 to discuss […]
Novartis Releases Fall SMA Community Update Letter
Novartis recently released an update on their continued collaboration with the SMA community through fundraising events and community support programs, and they will continue to […]
Scholar Rock Releases SMA Community Update Letter on Apitegromab
Yesterday, Scholar Rock shared an SMA community update letter on their Biologics License Application (BLA) for apitegromab in SMA. As shared on September 23, 2025, […]