Biohaven Ltd. announced that the US Food and Drug Administration (FDA) has accepted for review the company's New Drug Application (NDA) for troriluzole for the treatment of adult patients with spinocerebellar ataxia (SCA) and has granted Priority Review. This designation is assigned to applications for drugs that would offer a significant improvement over other available treatments for a given disorder or would provide a treatment option where none exists. In the case of SCA, a rare, genetic, neurodegenerative disease, troriluzole would be the first and only FDA-approved treatment for this life-threatening disorder. The FDA's decision regarding the NDA is expected within 6 months of filing (during 3Q2025). Based on FDA Priority Review timelines and if ultimately approved, Biohaven is prepared to commercialize troriluzole for SCA in the US in 2025.
Melissa Beiner, M.D., SCA Clinical Development Lead at Biohaven, commented, "Our NDA filing is the culmination of over 8 years of clinical research and represents an important collaboration across the SCA community. The troriluzole NDA reflects rigorous scientific collaborations between advocacy groups, patients and their families, clinical experts in SCA and Biohaven. The FDA decision to grant Priority Review demonstrates the extremely high unmet need in this rare neurodegenerative disease. Time is of the essence for patients with SCA, who are suffering relentless and irreversible functional decline including impairments in coordination and balance leading to falls, loss of ambulation, and difficulties with vision, speech and swallowing." Dr. Beiner added, "The robust clinical data presented in the NDA demonstrate sustained and compelling treatment benefit in SCA patients treated with troriluzole, a once-daily, oral pill. We look forward to working closely with the FDA throughout the review process to bring the very first treatment to patients and families suffering from SCA."
Jeremy Schmahmann, M.D., Professor of Neurology at Harvard Medical School and Founding Director of the Ataxia Center and the Martha and Robert Fogelman Endowed Chair in Ataxia and Cerebellar Neurology at Massachusetts General Hospital commented, "The FDA acceptance for review of this NDA represents a critical milestone for SCA patients. Since the discovery of the first gene for SCA in 1993, patients and families affected by SCA have watched generation after generation suffer severe, progressive disability and premature death with no treatment options. The need for an intervention that can slow disease progression and help patients maintain their independence is urgent. The delay in disease decline shown in the real-world evidence study is a watershed in the history of the SCAs. This is what patients have been waiting for. It is what the doctors who have been powerless, have been waiting for. Additionally, the importance of troriluzole's effects on reducing falls in this patient population cannot be overstated. I applaud the FDA for recognizing this urgency by granting a Priority Review and look forward to using troriluzole in the clinic if approved."
Biohaven's troriluzole clinical development program in SCA collected data over 8 years, including a robust long-term safety profile, and was the first industry trial conducted in SCA. The external control arms used in Biohaven's BHV4157-206-RWE Study were provided from objective, third-party data gathered from two independent natural history cohorts: one in the United States and one in Europe (EUROSCA Natural History Study). The National Ataxia Foundation (NAF) sponsored the Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) that served as the basis for the US natural history cohort. The data from the CRC-SCA is managed by the University of South Florida Health Informatics Institute. A total of 35 clinical sites provided data in the US and European natural history cohorts that served as the external controls in BHV4157-206-RWE. As per instructions from FDA on the real-world evidence study design and statistical analysis plan, the external control arm was determined using a Propensity Score Matching (PSM) method to ensure that untreated subjects from the comparator natural history cohort were rigorously matched to treated subjects from the troriluzole arm of Study BHV4157-206. PSM was used on all prognostic, demographic, and baseline characteristics known to be associated with disease progression in SCA, including baseline f-SARA, age, sex, age at symptom onset, genotype, and trinucleotide repeat length (by genotype).
Andrew Rosen, Chief Executive Officer of the National Ataxia Foundation (NAF), stated, "Biohaven's SCA program reflects years of dedicated clinical research and collaboration with leading world experts and advocacy groups to advance the ataxia field. We are proud that our multi-year effort to fund and support the Clinical Research Consortium for the Study of Cerebellar Ataxia played such a critical role in providing the external control arm of Biohaven's study. The goal of CRC-SCA is to improve our understanding of SCA disease progression and to promote the development of disease-modifying therapies for SCA." Mr. Rosen added, "On behalf of patients and families, who have watched generations of family members succumb to this devastating disease and have been waiting for decades for a treatment that could slow disease progression, I thank the FDA for not only accepting this NDA for review, but recognizing the need for urgency for our community in the form of a Priority Review."
Biohaven previously received both Fast-Track and Orphan Drug Designation (ODD) from the FDA, and ODD from the European Medicines Agency, where a troriluzole MAA is currently under review.
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