FDA grants orphan drug designation to branaplam for Huntington’s disease

The U.S. Food and Drug Administration has granted orphan drug designation to  branaplam (LMI070; Novartis) to treat Huntington’s disease.

Branaplam has been shown to reduce levels of mutant huntingtin protein in preclinical models. Novartis said it will start a development program for branaplam to explore the possibility of it as a treatment for people living with Huntington’s disease.

Branaplam is currently under investigation for the treatment of spinal muscular atrophy, where it was found to reduce huntingtin messenger RNA.