Biohaven’s Verdiperstat Receives Fast Track Designation for the Treatment of Multiple System Atrophy
 
Read the press release here:
https://www.biohavenpharma.com/investors/news-events/press-releases/03-18-2020
 
At this most difficult time some encouraging news for our 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 this FDA decision could pave the way for a faster approval of the drug.
Grateful to Biohaven and the FDA! #MSACoalition #BuildingHopeforMSA
 
We have compiled some questions and answers about this news 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.
 
More information on the Verdiperstat phase 3 clinical trial can be found here: https://clinicaltrials.gov/ct2/show/NCT03952806
More information on the FDA’s drug approval process can be found here: https://www.fda.gov/drugs/development-approval-process-drugs.
More information on fast track approvals can be found here:
https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track