Core Insights - Biohaven Ltd. announced the receipt of a Complete Response Letter (CRL) from the FDA regarding the New Drug Application (NDA) for VYGLXIA (troriluzole) aimed at treating spinocerebellar ataxia (SCA) [2][3] - The CRL was issued despite positive data from a real-world evidence study showing a 50-70% slowing of disease progression in patients treated with troriluzole compared to untreated controls [2][3] - Biohaven plans to meet with the FDA to discuss the evidence needed for a future NDA submission and is initiating strategic portfolio and cost-optimization measures to focus on key clinical programs [2][4][6] Regulatory and Clinical Data - The FDA's CRL cited concerns regarding potential bias and design flaws in real-world evidence studies, despite the study achieving statistical significance in its primary and secondary endpoints [2][3] - Study 206-RWE demonstrated a significant reduction in the risk of falls and delayed progression to wheelchair dependence in patients treated with troriluzole [2][3] - Biohaven's NDA included data from multiple independent studies supporting the efficacy of troriluzole, which received Orphan and Fast Track designations from the FDA [2][3] Strategic Focus and Financial Management - In response to the CRL, Biohaven is restructuring its business priorities to achieve a 60% reduction in annual direct R&D spending, focusing on three key clinical-stage programs: BHV-1400 for IgA nephropathy, Opakalim for epilepsy and depression, and Taldefgrobep alfa for obesity and SMA [4][6][7] - The company emphasizes its commitment to advancing innovative treatments for rare diseases and plans to present new data from its priority programs at an upcoming healthcare conference [2][4][6] Background on SCA and Troriluzole - Spinocerebellar ataxia is a rare neurodegenerative disorder affecting approximately 15,000 people in the U.S. and 24,000 in Europe and the UK, with no current FDA-approved treatments available [8] - Troriluzole is a novel prodrug that modulates glutamate levels, addressing the neurodegeneration associated with SCA [8]