NEW HAVEN, Conn., May 15, 2017 /PRNewswire/ -- (NYSE: BHVN) -- Biohaven Pharmaceutical Holding Company Ltd. (Biohaven) announced today that the U.S. Food and Drug Administration (FDA) has granted the company Fast Track Designation for Biohaven's product candidate trigriluzole (BHV-4157) for the potential treatment of Spinocerebellar Ataxia (SCA). Trigriluzole previously received Orphan Drug Designation from the FDA for the treatment of SCA in 2016. Biohaven is currently conducting a Phase 2/3 clinical trial in patients with SCA, with topline results expected in 2018.
SCA is a rare, debilitating neurodegenerative disorder that is estimated to affect approximately 22,000 people in the United States. Standard of care treatment is supportive and no medications are currently approved for patients with this debilitating condition.
"This Fast Track Designation from the FDA recognizes the high unmet medical need in patients with SCA," said Vlad Coric, M.D., CEO of Biohaven. "Fast track designation will help facilitate Biohaven's development of trigriluzole, and potentially expedite future regulatory review of trigriluzole for patients with SCA."
Phase 2/3 Clinical Trial of Trigriluzole
Biohaven initiated a randomized clinical trial of trigriluzole in patients with SCA in December 2016. Target enrollment is approximately 120 patients, with topline results expected in the first quarter of 2018. The study is designed to evaluate the safety and efficacy of trigriluzole for acute symptomatic treatment in this patient population. We believe that, if successful, this Phase 2/3 clinical trial will be the only pivotal trial necessary to support regulatory approval in SCA.
"Given the severe nature of this disease and the inadequacy of palliative treatments for patients with SCA we are endeavoring to complete our study as soon as possible," said Robert Berman, M.D., Chief Medical Officer at Biohaven.
Trigiluzole is a novel tripeptide prodrug being developed by Biohaven and represents more than six years of chemistry development and research into over 300 drug candidates. Biohaven anticipates that trigriluzole will be designated as a new chemical entity (NCE) and has received orphan drug designation from the FDA for the treatment of SCA.
About Fast Track Designation
As one of its Expedited Programs for Serious Conditions, the FDA grants Fast Track Designation to "facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need" in order to make important new drugs available to patients earlier. According to the FDA, a drug that receives Fast Track Designation is eligible for some or all of the following:
- More frequent meetings with FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval;
- More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers;
- Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met; and
- Eligibility for Rolling Review, which means that a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. NDA review usually does not begin until the drug company has submitted the entire application to the FDA.
Biohaven is a clinical-stage biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases, including rare disorders. Biohaven has licensed intellectual property from companies and institutions including Bristol-Myers Squibb Company, AstraZeneca AB, Yale University, Catalent, ALS Biopharma LLC and Massachusetts General Hospital. Biohaven is a company organized under the laws of the British Virgin Islands and its United States operations are based in New Haven, Connecticut.
This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of the Company's management. All statements, other than statements of historical facts, included in this press release regarding the potential effects of Fast Track Designation, including the possibility of expedited regulatory review, as well as the Company's plans and objectives, expectations and assumptions of management are forward-looking statements. The use of certain words, including the words "estimate," "intend," "expect," "believe," "anticipate," "will, "potential," "plan," "could," "may" and similar expressions are intended to identify forward-looking statements. The Company may not actually achieve the plans, intentions or expectations disclosed in the forward-looking statements and you should not place undue reliance on the Company's forward-looking statements. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by our forward-looking statements including the fact that the grant of Fast Track Designation does not guarantee that we will experience a faster development process, review or approval of trigriluzole compared to conventional FDA procedures, the possibility that the FDA could withdraw Fast Track Designation if it believes that it is no longer supported by data from our trigriluzole clinical development program and those factors described in the "Risk Factors" section of the Company's final prospectus dated May 3, 2017, filed with the Securities and Exchange Commission on May 5, 2017. The forward-looking statements are made as of this date and the Company does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Dr. Vlad Coric
Chief Executive Officer
Biohaven Pharmaceutical Holding Company Ltd.
Phone: (203) 404-0410
SOURCE Biohaven Pharmaceutical Holding Company Ltd.