BRIEF—US FDA to consider vamorolone for DMD

The US regulator has accepted a new drug application (NDA) for vamorolone, submitted by Santhera Pharmaceuticals and ReveraGen BioPharma, with a decision expected by October 26.

The European Medicines Agency is already reviewing a marketing authorization submission for the candidate, a first-in-class option for Duchenne muscular dystrophy (DMD).

The firms hope to improve on the current standard of care in DMD, corticosteroids, with a treatment which binds to the same receptor but could offer a better safety profile.

If approved, Santhera plans to launch vamorolone in the USA in the last quarter of 2023.

The submission is backed by data from the pivotal Phase IIb VISION-DMD study, plus results from four open-label studies.

Vamorolone has Orphan Drug status in the USA and in Europe, as well as Fast Track and Rare Pediatric Disease designations in the USA.