Toll free: 1-855 542-5672 (1-855-KICK-MSA)

UPDATE – Information from Biohaven RE: Verdiperstat

Published by defeatmsa

June 11, 2021

JUNE 1, 2021

Defeat MSA Alliance Research Program Advisors:

Important Questions & Answers from Biohaven

RE:   Clinical Trial on Verdiperstat


  • Can I still join the trial?

Answer:  Enrollment for the phase 3 study closed in July of 2020.

  • When are we expected to know the results of the Phase 3 study?

Answer:  Biohaven’s expectation is that Top-Line results will be available by the end of the third quarter in 2021.

  • If the results are positive, what happens next?

Answer: If the Phase 3 study results are positive and show that verdiperstat is effective, then Biohaven will engage with the FDA to discuss next steps.

  • Is there anything we can do as patients or caregivers to help?

Answer: Continued advocacy work to raise awareness about MSA, including diagnosis and treatment gaps, as well as the burden of the disease on patients and families.

  • What is the plan for patients who live outside the United States?

Answer: If the Phase 3 study results show a statistically significant effect of slowing disease progression with Verdiperstat, Biohaven will engage with the health authorities in Europe and other countries to discuss next steps.

  • How does the drug work?

Answer: Verdiperstat is believed to inhibit an enzyme called myeloperoxidase that catalyzes the production of cytotoxic compounds. Buildup of these reactive compounds leads to brain tissue damage and neuronal death. Verdiperstat binds to Myeloperoxidase and is believed to reduce the production of reactive compounds and protect nerve cells from damage.

Learn more in this video:

  • Could you tell us anything about how many people have been in the Phase 3 study and where they are from? In the United States or outside of the United States?

Answer: There are 336 people in the blinded phase 3 trial; half are on placebo and half are on verdiperstat. 35% of patients are in the E.U. (Austria, France, Germany, Italy and UK) and 65% are in the United States.

  • If people who are taking verdiperstat have questions or concerns, who should they contact?

Answer: They should contact their study physician.

You May Also Like…


  1. Sandra Robbins

    I have MSA. Can I get in on a clinical trial. or is it too late. I’ve had it for nine years I’m still in the first stages. At least that’s what the doctor says.

    • defeatmsa

      Please write to us:
      and if you are in North America, you can call us toll free during daylight hours: 1 855 542-5672 (855 Kick-MSA)
      We are not operating the any trials but we can provide information about research and some drug trials.


Submit a Comment

Your email address will not be published.

Copy link
Powered by Social Snap