Biohaven Ltd. announced that the Division of Neurology within FDA's Office of Neuroscience informed the company that they are extending the PDUFA date for the troriluzole new drug application (NDA) for the treatment of spinocerebellar ataxia (SCA) by three months to provide time for a full review of Biohaven's recent submissions related to information requests from the FDA. The Division also informed Biohaven that it is currently planning to hold an advisory committee meeting to discuss the application, but no date has been scheduled. The FDA did not raise any new concerns in the letter. The FDA's decision regarding the NDA is now expected in 4Q 2025.
Biohaven previously received Fast-Track, Orphan Drug Designation (ODD) and Priority Review from the FDA regarding troriluzole for SCA. Priority Review 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.
Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven stated, "SCA is a devastating neurodegenerative disease that has affected generations of families and has no current approved therapy. We are committed to bringing the first treatment to patients and families affected by SCA. The clinical data presented in the NDA show a highly favorable benefit-risk profile with troriluzole, a once-daily oral pill, slowing disease progression by 50-70%, as measured by the f-SARA scale, and reducing the risk of falls. We look forward to a meeting with the advisory committee to discuss troriluzole's potential to improve the lives of individuals with SCA. We are especially grateful to the patients, families, and leading neurologists who participated in our studies over an 8-year period and the broader SCA community and patient advocacy groups, including the National Ataxia Foundation, who supported the review of troriluzole as the first potential therapy for SCA."
The Company recently announced it has completed the FDA mid-cycle review meeting and regulatory inspections of Biohaven and key clinical research sites for troriluzole in the treatment of SCA. The mid cycle review concluded that there were no previously unidentified major safety concerns, and it does not appear a Risk Evaluation and Mitigation Strategy (REMS) is needed.
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