The Food and Drug Administration (FDA) has granted Fast Track designation to verdiperstat (BVH-3241; Biohaven) for the treatment of multiple system atrophy.

Multiple system atrophy (MSA) is a rare, rapidly progressive, and fatal neurodegenerative disease characterized by parkinsonism, cerebellar ataxia, and impaired involuntary functions. 

Verdiperstat is a potential first-in-class, oral, brain-penetrant, myeloperoxidase inhibitor. Myeloperoxidase is an enzyme that acts as a key driver of pathological oxidative stress and inflammation in the brain. 

The Company is currently evaluating the efficacy of verdiperstat in the ongoing phase 3 M-STAR study in patients with MSA. The primary efficacy end point of the trial is the change from baseline in the Unified MSA Rating Scale Part I (an assessment of functioning across various areas) and II (motor examination) at week 48. 

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“We are extremely pleased that the FDA has granted Fast Track designation for verdiperstat, acknowledging the high unmet medical need for people suffering with MSA,” said Irfan Qureshi, MD, Vice President of Neurology at Biohaven. “The Fast Track designation may help accelerate the development of verdiperstat as the first treatment aimed at slowing progression of this devastating disease.”

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