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FDA Reconsiders Rare Disease Drug
23 Oct
Summary
- FDA will review Vyglxia, a rare disease drug, for a second time.
- Biohaven's drug Vyglxia aims to treat spinocerebellar ataxia.
- Vyglxia could be the first medicine marketed for this rare condition.
The U.S. Food and Drug Administration has accepted a resubmitted application for Vyglxia, a drug developed by Biohaven for rare genetic disorders. This marks a second review for the treatment, which targets spinocerebellar ataxia, a condition causing progressive neuronal damage leading to issues with balance, coordination, and speech.
Biohaven's initial application was rejected by the FDA in November of last year, citing concerns about the design of a key experiment. European regulators also questioned the supportive evidence, leading Biohaven to withdraw its application there. Despite these setbacks, Biohaven has submitted new analyses with its refreshed application, aiming for regulatory clearance.
The FDA's decision to review Vyglxia again has boosted Biohaven's stock by approximately 25%. Analysts are optimistic about the drug's prospects, particularly given recent shifts at the FDA towards greater flexibility in reviewing rare disease medicines and reversals of decisions made by former leadership.
A panel of experts will convene to discuss the findings, with the FDA expected to issue a new verdict in the first quarter of next year. If approved, Vyglxia could become the first available medication for spinocerebellar ataxia, with some analysts projecting annual sales exceeding $1 billion. The outcome may depend on the extent of the FDA's flexibility changes.
