FSHD drug granted orphan drug status by FDA

Published Date
29/01/2020
Author
Research Team
Category
Research

Fulcrum Therapeutics has announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) to losmapimod for the treatment of patients with FSHD. This designation gives Fulcrum certain financial benefits that will help to lower the cost of developing the drug.

Losmapimod has been shown to “switch off” DUX4 in cells originating from people with FSHD. The safety and efficacy of the drug is currently being tested in a Phase 2 clinical trial. The results from this trial are expected later in 2020.

For more information, please read Fulcrum’s press release.

If you have any research questions, please contact our Research Line on research@musculardystrophyuk.org 

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