Biohaven Ltd. has announced a significant development in the pursuit of a treatment for
spinocerebellar ataxia (SCA), a rare and life-threatening genetic neurodegenerative disorder. The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for
troriluzole, a promising candidate for treating adult patients with SCA, and has granted it Priority Review status. This acknowledgment by the FDA underscores the potential of troriluzole to significantly improve the treatment landscape for SCA, where no approved therapies currently exist.
Spinocerebellar ataxia is characterized by a progressive loss of
motor control due to
degeneration of the cerebellum and brainstem. This condition, affecting around 15,000 individuals in the United States and 24,000 in Europe, leads to severe disabilities such as
impaired mobility, speech difficulties, and premature death. Patients with SCA experience relentless functional decline, underscoring the urgent need for an effective treatment option.
Troriluzole, a novel prodrug, modulates glutamate, the most abundant excitatory neurotransmitter in the human body. It works by enhancing glutamate uptake from synapses, thereby potentially addressing the glutamate deregulation responsible for neurodegeneration in SCA patients. If approved, troriluzole would be the first FDA-sanctioned treatment for this debilitating disorder, a significant advancement for patients and their families.
The NDA submission is supported by positive results from Study BHV4157-206-RWE, which showed that troriluzole significantly slowed SCA disease progression by 50-70% over a three-year period. This study involved patients with various SCA genotypes and demonstrated a statistically significant delay in disease progression when compared to an external control group. The data indicate that troriluzole could delay disease progression by 1.5 to 2.2 years, offering hope for improved quality of life for patients.
Biohaven's comprehensive clinical research program, spanning over eight years, has been pivotal in reaching this milestone. The program includes long-term safety data and used external control arms derived from third-party data to bolster the study's findings. The real-world evidence from Study BHV4157-206-RWE underscores the potential of troriluzole to become a groundbreaking treatment option for SCA, reinforcing the importance of FDA’s Priority Review designation.
Dr. Melissa Beiner, SCA Clinical Development Lead at Biohaven, emphasized the collaborative effort behind this achievement, highlighting the partnerships with advocacy groups, patients, families, and clinical experts. These collaborations have been instrumental in progressing toward a viable treatment option for SCA. Dr. Beiner expressed optimism about working with the FDA to bring troriluzole to market and provide much-needed relief to those impacted by SCA.
Dr. Jeremy Schmahmann from Harvard Medical School also remarked on the significance of the FDA's acceptance of the NDA, noting it as a pivotal moment for SCA patients who have long awaited a treatment that could alter the course of their disease. He acknowledged the importance of troriluzole's impact on reducing falls among patients, which is a critical factor in maintaining their independence.
Biohaven's commitment to addressing the unmet needs in SCA is further demonstrated by its earlier receipt of Fast-Track and Orphan Drug Designation from the FDA, as well as an ongoing Expanded Access Protocol to provide early access to troriluzole. This initiative reflects Biohaven's dedication to improving the lives of those affected by SCA and advancing the field of ataxia research.
In conclusion, the acceptance of troriluzole’s NDA for Priority Review by the FDA marks a major step forward in the treatment of spinocerebellar ataxia. If approved, troriluzole will provide a new ray of hope for patients and families battling this challenging condition, paving the way for improved patient outcomes and quality of life.
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