Novartis has temporarily suspended dosing in a mid-stage study of an experimental Huntington’s disease drug, writing Wednesday in a letter to patients that its treatment was linked to nerve damage.
The VIBRANT-HD study began in early 2022 and was a long-awaited trial of a huntingtin-lowering drug, branaplam, that could be taken by mouth. On Monday, August 8th, we learned that dosing has been temporarily suspended at the recommendation of an independent committee that is monitoring the data from the trial. This decision was made because of signs that some of the participants taking branaplam may be experiencing new problems with their nerves, known as peripheral neuropathy. Let’s talk more about what happened and what’s next.
Basel, October 21, 2020 — Novartis today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for branaplam (LMI070) in Huntington’s disease (HD). An Orphan Drug Designation grants special status to a drug that treats a rare disease or condition, and provides companies certain benefits to encourage the continued development of medicines that bring novel solutions to patients with these severe diseases.
ZURICH (Reuters) - Novartis is seeking to repurpose its investigational oral spinal muscular atrophy (SMA) drug branaplam to treat Huntington’s disease, the Swiss drugmaker said on Wednesday, as it plans a clinical trial after winning U.S. orphan drug designation.
Novartis research head Jay Bradner said on Thursday the Swiss drugmaker is focusing efforts against spinal muscular atrophy (SMA) on gene therapy Zolgensma and retreating from oral therapy like its molecule LMI070, also called branaplam.
After getting hit with a setback that forced the pharma giant Roche to abandon its lead drug for spinal muscular atrophy, company execs are back with a new drug in-licensed from PTC $PTCT that they believe has legs.