Clinical Trial Phase 2 Begins for Novartis Huntington’s Disease Drug Branaplam

Good News for the Huntington’s Disease Community from Novartis Pharmaceutical Company

On December 16, 2021, Novartis was granted by the US Food and Drug Administration (FDA) the Fast Track designation for branaplam (LMI070) for the treatment of Huntington’s disease (HD). Fast Track designation facilitates the development and expedites the review of drugs to treat serious conditions and fill unmet medical needs. Branaplam is being developed as a potential first in class orally administered disease modifying therapy for HD. See News Release

Branaplam is an oral drug that was originally developed by Novartis to treat a childhood disorder called spinal muscular atrophy. It was also found to lower huntingtin protein by interfering with the genetic “recipe” in a process known as RNA splicing.

Information provided by:

Novartis ( Novartis Pharmaceuticals )

“This is the first study of branaplam in adults with Huntington’s Disease (HD) to determine the correct dose required to lower mutant huntingtin protein (mHTT) levels in the cerebrospinal fluid (CSF) to a degree expected to be efficacious over longer periods of time.

This study is a randomized, double-blind, placebo-controlled study which will be conducted in approximately 75 early manifest HD participants.”