Vamorolone Fast Track - access to treatments

Vamorolone is an investigational drug that was developed as a dissociative steroid for people with Duchenne muscular dystrophy, manufactured by the pharmaceutical company Santhera

Vamorolone is a first-in-class treatment that binds to the same receptors as corticosteroids but modifies the downstream activity of the receptors. High dose corticosteroids have significant side effects that detract from patient quality of life. Due to Vamorolone’ s potential to ‘dissociate’ efficacy from typical steroid safety concerns, this treatment could be a valuable alternative to corticosteroids.

MDUK is currently responding to NICE’s scoping of the appraisal, which looks to identify the variables that will be assessed during the full appraisal. MDUK will also be attending a scoping workshop at the end of October 2022.

More information on NICE’s assessment of Vamorolone can be found here.