BRIEF—Vamorolone data at Muscular Dystrophy Association congress

Swiss firm Santhera Pharmaceuticals and its US development partner ReveraGen BioPharma are presenting new efficacy and tolerability data for vamorolone in Duchenne muscular dystrophy (DMD).

At the 2022 Muscular Dystrophy Association (MDA) conference, the firms will outline results from the pivotal Phase IIb VISION-DMD study, which met its primary efficacy endpoint.

Vamorolone has a novel mode of action, binding to the same receptor as corticosteroids but modifying downstream activity.

In the VISION-DMD trial, the candidate showed sustained efficacy across multiple endpoints over 48 weeks and was well tolerated, with a favorable safety profile.

A rolling new drug application (NDA) submission to the US Food and Drug Administration for vamorolone for the treatment of DMD is planned to start at the end of March 2022.