1. Oct 21, 2020

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.

Read the full press release here.