Regulatory DevelopmentsThe late-cycle meeting with the FDA was collaborative, and the cancellation of the AdCom is seen as a favorable development, suggesting the NDA is on pace for approval.
Regulatory SupportNew FDA leadership at the rare disease division expressed support for an NDA, as they appeared to appreciate the totality of the data and high unmet need.
Stock PotentialThere was an expected share price return of over 310%, indicating significant potential upside.