Biopharma Bites: FDA Approves High-Dose Spinraza for SMA, J&J's $500M Autoimmune Deal, and Updates from BMS and Boehringer

on

The FDA approved a new high-dose regimen of Biogen's SPINRAZA (nusinersen) for treating spinal muscular atrophy (SMA) in all ages, based on the Phase 2/3 DEVOTE study showing significant motor function improvements in treatment-naïve infants (mean CHOP-INTEND difference:
26.19 points, p<0.0001 vs. sham).125

High-dose SPINRAZA delivers higher drug concentration in loading and maintenance phases, with a safety profile consistent with the established low-dose regimen; it will be available in the US soon and is already approved in the EU, Switzerland, and Japan.1

The DEVOTE study included pivotal data from 75 treatment-naïve infantile-onset SMA patients and supportive data from 24 later-onset patients, demonstrating clinical benefits.1

Organizations like Muscular Dystrophy Association (MDA) and Cure SMA celebrated the approval as a milestone advancing SMA care, with MDA noting its roots in funded research.23

Note:
Search results detail Spinraza approval but lack specifics on J&J's $500M autoimmune antibody deal, BMS, or Boehringer updates.

Sources:

1. https://www.globenewswire.com/news-release/2026/03/30/3264519/0/en/FDA-Approves-New-High-Dose-Regimen-of-SPINRAZA-nusinersen-for-Spinal-Muscular-Atrophy.html

2. https://www.mda.org/press-releases/fda-approves-high-dose-regimen-of-spinraza-for-spinal-muscular-atrophy

3. https://www.curesma.org/biogen-receives-fda-approval-of-high-dose-spinraza-for-the-treatment-of-sma/

5. https://www.rarediseaseadvisor.com/news/fda-approves-high-dose-regimen-nusinersen-spinal-muscular-atrophy/