The Food and Drug Administration (FDA) granted a Fast Track designation to the myeloperoxidase (MPO) inhibitor, verdiperstat (Biohaven Pharmaceutical, New Haven, CT) for the treatment of multiple system atrophy (MSA).
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 (NCT03952806) is currently ongoing to evaluate the efficacy of verdiperstat in MSA across approximately 50 sites in the US and Europe.
"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," commented Pamela Bower, secretary of the MSA Coalition. "Each day that is saved through the Fast Track process brings us one day closer to providing hope to the MSA community," added Philip Fortier, executive director of the Defeat MSA Alliance. More information about the MSA Coalition and Defeat MSA Alliance is available at www.multiplesystematrophy.org and www.defeatmsa.org.
Irfan Qureshi, MD, vice president of Neurology at Biohaven, commented, "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."
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 individuals 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.
Rashmi B. Halker Singh, MD, FAHS, FAAN
Jessica Ailani, MD, FAHS, FAAN
Lee I. Kubersky, MD