Multiple System Atrophy Drug Verdiperstat Wins Fast Track Designation from FDA


The ongoing phase 3 trial of the Biohaven drug is expected to be completed October 20, 2021.

Irfan Qureshi, MD

Irfan Qureshi, MD

Biohaven announced that company’s myeloperoxidase (MPO) inhibitor, verdiperstat, has been granted fast track designation by the FDA for the treatment of multiple system atrophy (MSA).1

Verdiperstat, also known as BHV-3241, is an oral, brain-penetrant, irreversible inhibitor of MPO, and has made visits to the FDA on multiple occasions, dating back to February 2019. At the time, the drug received orphan drug designation from the agency after completing its phase 1 and preliminary phase 2a clinical trials.2

"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, Biohaven, said in a statement.

In phase 1 and phase 2a, patients with MSA treated with 900 mg of verdiperstat twice daily showed numerical improvements in their change from baseline on the Unified MSA Rating Scale compared with placebo. Additionally, researchers noted positive benefits in other outcome measures, such as the Composite Autonomic Symptom Score and MSA-Quality of Life scale.

Results from the phase 2a trial showed evidence of target engagement and favorable trends over 12 weeks in patients with MSA on the Unified Multiple System Atrophy Rating Scale (UMSARS). After those 12 weeks, placebo-treated patients (n = 17) worsened by 4.6 points (standard error [SE], 1.1) on the Unified MSA Rating Scale, whereas patients treated with 300-mg twice daily verdiperstat (n = 18) only worsened by 3.7 points (SE, 1.2) and those treated with the 600-mg twice daily dose (n = 18) only worsened by 2.6 points (SE, 1.4). The drug was also proved to be generally safe and well tolerated.2

In July 2019, Biohaven enrolled its first patient into the phase 3 M-STAR study (NCT03952806), which aims to compare the efficacy of verdiperstat versus placebo in patients with MSA. The ongoing, double-blind, parallel-group study, randomized patients to either 300 mg of verdiperstat twice daily or matching placebo. The trial is still currently recruiting, with an estimated enrollment of 250 participants.3

Patients included in the study are between the ages 40 to 75 years, have a diagnosis of MSA, including either subtype (MSA-P or MSA-C), are able to ambulate without the assistance of another person, and have an anticipated survival of at least 3 years at the time of screening. Other neurologic disorders, or conditions that would interfere with a subject’s ability to comply with study instructions, are reason for exclusion from the study.

Researchers are using changes from baseline at week 48 in the UMSARS Part I and II total score as a way to test the efficacy of verdiperstat, as the primary end point. Additional secondary outcome measures included change in baseline at week 48 on the Clinical Global Impression (CGI-C) score and impact of verdiperstat on quality of life, measured by the changes in baseline at week 48 in the MSA-Quality of Life (MSA-QoL) score.

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Verdiperstat is currently being tested in the Healey-ALS trial, the world’s first platform study evaluating multiple drugs in patients with amyotrophic lateral sclerosis (ALS). Started in March 2020, the ongoing trial will use a combination of both verdiperstat, zliucoplan, and nanocrystalline gold, and aims to find results using the ALS Functional Rating Scale-Revised (ALSFRS-R) as a primary endpoint. That trial is expected to conclude in March 2022.

"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, Defeat MSA Alliance, said in a statement. Biohaven has not informed when they plan to release its next set of data, but the trial is expected to conclude October 20, 2021.


1. Biohaven’s verdiperstat receives fast track designation for the treatment of multiple system atrophy [news release]. New Haven, CT: Biohaven Pharmaceuticals; Published March 18, 2020. Accessed March 18, 2020.

2. Biohaven’s verdiperstat receives orphan drug designation from FDA for multiple system atrophy [news release]. New Haven, CT: Biohaven Pharmaceuticals; Published February 19, 2019. Accessed March 18, 2020.

3. Biohaven enrolls first patient in phase 3 clinical trial of verdiperstat, oral myeloperoxidase inhibitor, for the treatment of multiple system atrophy [news release]. New Haven, CT: Biohaven Pharmaceuticals; Published July 31, 2019. Accessed March 18, 2020.

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