Verdiperstat Receives Orphan Drug Designation for Fatal, Fast-Acting Neurodegenerative Disease

Drug Has Potential Role In Oxidative Stress, Inflammation, and Neurodegeneration

Drug candidate verdiperstat (previously BHV-3241, Biohaven Pharmaceutical Holding Company Ltd.) has received orphan drug status from the FDA for the treatment of multiple system atrophy (MSA). 

Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of myeloperoxidase (MPO), a major driver of pathological oxidative stress and inflammation in the brain.

Results from clinical trials showed that after 12 weeks of treatment, patients receiving placebo worsened by 4.6 points on the Unified MSA Rating Scale compared to verdiperstat-treated patients, who worsened by 3.7 points at the 300-mg twice-daily dose and by 2.6 points at the 600-mg twice-daily dose. In approximately 250 patients, verdiperstat proved generally safe and was well tolerated. 

MSA, a rare and fatal neurodegenerative disease, causes death within 6 to 10 years of disease onset. Symptoms include slow movement, rigid muscles, tremor, and poor balance; cerebellar ataxia; and problems with blood pressure control, bladder function, and digestion. There is no cure for the disease and only symptomatic and palliative therapies are available.

Source: Biohaven Pharmaceutical Holding Company Ltd, February 19, 2019