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Biohaven’s Verdiperstat Receives Fast Track Designation for the Treatment of Multiple System Atrophy

Biohaven’s Verdiperstat Receives Fast Track Designation for the Treatment of Multiple System Atrophy

At the world fights a global pandemic some encouraging news for the multiple system atrophy community is very welcome. Though we must still await the results of the Verdiperstat phase 3 clinical trial (currently underway), should the results be positive the FDA decision to fast track Verdiperstat could pave the way for a faster approval of the drug. We are grateful to Biohaven and the FDA! #MSACoalition #BuildingHopeforMSA


We know the MSA community have many questions about Verdiperstat. See below for the latest updates directly from the drug company Biohaven Pharmaceuticals.


*** UPDATE (as of June 11, 2021)

Q. Can I still join the trial?

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

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

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

Q. If the results are positive, what happens next?

A. 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.

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

A. 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.

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

A. 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.

Q. How does the drug work?

A. 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:

Q. 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?

A. 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.

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

A. They should contact their study physician.

*** UPDATE (as of November 6, 2020):

The MSA Coalition have received many questions about accessing the investigational drug Verdiperstat. Biohaven Pharmaceuticals have provided some answers:

Q. I live outside of the USA. Can I request expanded access or compassionate use to get Verdiperstat in my country?


A. Unfortunately no. Biohaven confirms that the only way to access Verdiperstat outside of the USA is if you are already enrolled in the phase 3 trial at a site outside of the USA.

Q. Can I still enroll in the clinical trial?

A. No, the trial is now closed to new participants at all study sites globally.

See Biohaven press release from July 21,2020

Q. When will the study be completed?

A. It is expected that all currently enrolled patients will have finished this blinded study by July of 2021.

Q. When will the study results be available?

A. No results will be available until after every enrolled patient has completed the trial. Topline results are expected by late 2021.

Q. What is Expanded Access?

A. Expanded Access, also known as “Compassionate Use”, is a pathway through which patients with serious diseases may be able to obtain access to an investigational drug or other therapy outside of a clinical trial.

A qualified physician would need to request access to the drug from the drug company on behalf of a patient. The drug company may agree to allow access if certain conditions are met. In the US the Food and Drug Administration (FDA) oversees this program.

You can learn more about the program at the FDA website.


Q. I live in the USA, how do I request expanded access to Verdiperstat?
A. Biohaven Pharmaceuticals provides detailed information on their expanded access policy at their website:



*** UPDATE (as of April 9, 2020): We have reached out to Biohaven for clarification about how they are addressing concerns regarding administering the trial during the global pandemic. Their answers are included below.

Q. What does fast track designation mean? Is the drug available now by prescription from my doctor?

A. Fast track designation does NOT mean that the drug is already approved for use to treat MSA. Fast track designation means that the FDA has promised to expedite the review process once the drug company has results to show them from the phase 3 trial. Both the drug company and the FDA will want to examine the results of the phase 3 trial very carefully before there is any decision made on whether to make this drug available to patients. The FDA cannot promise to approve it without first examining the trial results.

Q. What is the timeline for approval? Does this mean the drug could be approved within months or years (if phase 3 works)?

A. If the phase 3 trial is positive we can’t know for sure how long the next steps in the process of FDA approval will take. The phase 3 trial is estimated to complete October 20, 2021.

Q. Why does it take so long to get a drug approved?

A. We understand the timeline is frustrating however there are strict FDA protocols that must be followed. Drugs that are meant to slow disease progression have to be evaluated over the course of time (for a year in the case of MSA). This makes these research studies longer to complete. It takes considerable time to ramp up a study of this size and to find qualified patients to participate. There were over 50 clinical centers that had to be mobilized to recruit and enroll the 250 patients needed. This high number of patients is needed in order to show statistical significance in the results. Patients enrolled must each be on the drug or placebo for one year will need to be monitored closely at regular intervals.

Q. In light of the Covid-19 pandemic, how is the phase 3 study now being handled? Are different sites handling things differently, or is there a directive from Biohaven on what will happen?

A. Our top priority is to ensure the health, safety, and well-being of our study participants and site staff. As such, we have offered general principles and guidance to M-STAR study sites in response to the COVID-19 pandemic. As there are many people who depend on the work we do, Biohaven will support sites’ efforts to ensure that participants can safely continue in the study and receive their study medication. We encourage sites to follow institutional policies and procedures regarding conducting in- person study visits. We have asked sites to please communicate any potential issues to us as soon as they are aware of them, and we will work with them on a case-by-case basis to address such issues. Biohaven has implemented workaround measures that we believe are in compliance with guidance from health authorities, including the US Food and Drug Administration and European Medicines Agency. Biohaven has provided guidance to sites regarding alternatives, focusing largely on safety measures. We have asked sites that cannot see patients in person in their clinics due to the COVID-19 pandemic to perform remote visits via phone or telemedicine video, collect safety laboratories at local labs, etc. The Institutional Review Boards (IRBs) for the M- STAR study sites have been informed about these potential modifications to the study conduct.

Q. Each study visit involves two blood draws, several hours apart. Is Biohaven expecting people to go to local labs two times in one day for blood draws? If so, this puts people at risk during the global pandemic.

A. During the COVID-19 Pandemic, Biohaven is following the Health Authority guidelines. If patients are unable to go into sites for visits, a remote visit may occur (while the specific details of the patient’s safety are taken into consideration) and blood samples may be collected and analyzed at a local laboratory (such as Quest). These blood draws will be limited to assessing safety laboratories in order to ensure that the participant is tolerating the study drug without negative effects on major organ systems. The two blood draws—one before and one after the dose of study medication—that you are specifically asking about are intended to assess drug levels in the blood. These measurements will not be conducted when blood is drawn at local laboratories during the COVID-19 pandemic. Thus, there will only need to be 1 blood draw at a local laboratory per remote visit.

Q. Will the principal investigators be doing neurological exams virtually? (Each visit involves rating scales, and this could realistically be done virtually.)

A. Remote study visits may be conducted by the site via phone or telemedicine video. Assessments performed during these remote visits will focus largely on ensuring safety of the study participant. If deemed appropriate by the investigator, some aspects of the neurological exam may be performed via telemedicine video. However, we do not

anticipate requiring completion of all the study rating scales via telemedicine video. We are mindful that some participants may not have the same technology resources available. Moreover, the validity and reliability of performing all of these assessments remotely is not ensured.

Q. Will the people currently enrolled stay in the study longer than expected as they won’t be able to have visits with their doctors? (This relates to the validity of the study).

A. Currently, we are focused on ensuring the safety of trial participants and site staff. We believe that the workaround measures that we have implemented (such as remote visits) are in compliance with guidance from health authorities including the US Food and Drug Administration and should not impact the validity of the study. We do not anticipate that participants will remain in the study longer than anticipated, however extended windows to allow flexibility for conduct of visits may be allowed.

Q. Does participating in a clinical trial affect the care someone would receive if sick with Covid 19? If a hospital had to make a choice between who gets a ventilator, can it be assumed that the person in a clinical trial (or with a fatal neurodegenerative disease) would not be selected?

A. Every hospital likely has its own policies regarding the medical care that someone would receive if they develop COVID-19. We do not believe that a patient’s medical care would be affected based on clinical trial participation.

Q. How many slots are still available, and will people have to wait until the quarantine is lifted to try to participate in the trial?

A. Evolving governmental and institutional restrictions implemented due to safety concerns related to the COVID-19 pandemic have limited screening and enrollment of new participants at most, if not all, M-STAR study sites. Once these restrictions are lifted, screening and enrollment will resume. We estimate that approximately half of the target number participants have been enrolled in the study. This study has not officially been put on hold but, due to the restrictions of the institutions involved, the rate of study enrollment has decreased.

Q. As of April 2020, the Verdiperstat Biohaven trial is underway in the USA but has not yet started in Europe. In light of Covid 19 is the trial still planned to take place in Europe?

A. Unfortunately, the M-STAR trial has been significantly impacted due to the unprecedented COVID-19 pandemic. M-STAR study has a planned 250 randomized participants, among all countries enrolling in the M-STAR study. We estimate that approximately half of the target number of participants have been enrolled in the study. This study has not officially been put on hold but, due to the government and institution COVID-related restrictions, the rate of study enrollment has decreased. At this point it is difficult to determine when the global study enrollment limits will be reached. The Europe sites continue to prepare for launch of the M-STAR study once it is safe and feasible to resume clinical activity and currently there are sites open to enrollment in France, Austria and the UK. Biohaven continues to work behind the scenes with study centers in the US and the EU in the hope of moving forward expeditiously with the study as soon as possible, once the safety of trial participants is ensured.

More Information About Verdiperstat:

More information on the Verdiperstat phase 3 clinical trial can be found here:

More information on the FDA’s drug approval process can be found here:

More information on fast track approvals can be found here:

Verdiperstat video:

Biohaven Pharmaceuticals expanded access policy: