Biohaven Pharmaceutical announced the Company's myeloperoxidase (MPO) inhibitor, verdiperstat, received Fast Track designation from the US Food and Drug Administration (FDA) for the treatment of multiple system atrophy (MSA).
"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. The Fast Track designation may help accelerate the development of verdiperstat as the first treatment aimed at slowing progression of this devastating disease," Irfan Qureshi, MD, Vice President of Neurology at Biohaven, said.
The FDA's Fast Track designation provides for an expedited review of potential new drugs intended to treat serious conditions with high unmet need, allowing important new drugs to become available more quickly to patients suffering from serious conditions. Benefits of Fast Track designation include enhanced interaction with the FDA as well as the eligibility to obtain accelerated approval and priority review at the time of a New Drug Application (NDA) filing if relevant criteria are met.
"We are grateful to the FDA for recognizing the serious and urgent need to accelerate the development of a potential new treatment for people living with MSA," Pamela Bower, Secretary of the MSA Coalition said. "Each day that is saved through the Fast Track process brings us one day closer to providing hope to the MSA community," Philip Fortier, Executive Director of the Defeat MSA Alliance said.
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Verdiperstat is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of myeloperoxidase, an enzyme that acts as a key driver of pathological oxidative stress and inflammation in the brain. A phase 3, multinational clinical trial is currently ongoing to evaluate the efficacy of verdiperstat in MSA across approximately 50 sites in the United States and Europe.
MSA is a rare, rapidly progressive, and fatal neurodegenerative disease. Only symptomatic and palliative therapies are currently available. MSA causes Parkinson's disease-like movement problems (slow movement, rigid muscles, tremor, and poor balance), cerebellar ataxia, as well as problems with involuntary (autonomic) functions, including blood pressure control, bladder function, and digestion. MSA usually leads to death after an average of 6 to 10 years from the onset of symptoms.
Biohaven licensed verdiperstat (BHV-3241) from AstraZeneca AB in September 2018, where it was known as AZD3241. The M-STAR study is a currently ongoing phase 3 clinical trial designed to evaluate the efficacy of verdiperstat in MSA. Verdiperstat received Orphan Drug designation from the US FDA as well as the European Medicines Agency due to the unmet medical need in MSA. Verdiperstat also has the potential to be developed in a number of other diseases associated with oxidative stress, inflammation, and neurodegeneration. A clinical trial designed to evaluate the efficacy of verdiperstat in amyotrophic lateral sclerosis (ALS) is being planned in collaboration with the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital.