With Duchenne Filing Due, Santhera Moves Vamorolone Into Becker Muscular Dystrophy

Rare Disease Trial Receiving FDA Funding

Santhera is bouncing back after a Duchenne drug failure two years ago with idebenone, and is looking to gain approval of vamorolone in this indication as well as the much rarer Becker muscular dystrophy.

Muscular dystrophy wheelchair user
Becker muscular dystrophy is more rare and slower in onset than Duchenne, but still leaves individuals unable to walk. • Source: Shutterstock

Santhera Pharmaceuticals and Reveragen BioPharma are gearing up to submit vamorolone to the US regulator as a new Duchenne muscular dystrophy treatment by the end of this year, but have also just announced the start of Phase II study of the drug in the related Becker muscular dystrophy (BMD).

Caused by the mutations in the same gene as Duchenne, which cause a loss of the muscle-building protein dystrophin, BMD is usually milder and the progressive muscle weakness slower

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