Shares of Longboard Pharmaceuticals, Inc. surged on Thursday after the United States Food and Drug Administration (FDA) granted both Rare Pediatric Disease designation and Orphan Drug designation to its investigational drug, bexicaserin, for the treatment of Dravet syndrome. Pre-market trading saw shares rise by 4.2%, reaching $30.53, a continuation of the company’s upward trend over the last year, where shares increased fourfold.

The FDA’s dual designation is significant for Longboard, a clinical-stage biopharmaceutical company specialising in neurological diseases. The company aims to develop transformative treatments for conditions with unmet medical needs, such as Dravet syndrome, a rare and severe type of epilepsy that begins in infancy.

Dr. Randall Kaye, Chief Medical Officer at Longboard, noted that the FDA’s support is crucial as the company prepares to launch its global Phase 3 trial for bexicaserin, starting with Dravet syndrome. Kaye emphasised the value of receiving both designations, particularly the potential for a Priority Review Voucher (PRV), which can either be used for another compound’s priority review or sold.

FDA designations: Rare Pediatric Disease and Orphan Drug

The Rare Pediatric Disease designation aims to promote the development of treatments for rare paediatric conditions. This designation qualifies Longboard for a Priority Review Voucher, which can expedite the FDA review process for a different product in the future. The Orphan Drug designation, on the other hand, supports the development of treatments for rare diseases, providing benefits such as tax credits for clinical trials, exemption from user fees, and the possibility of seven years of market exclusivity upon approval.

Longboard’s investigational drug, bexicaserin, targets Developmental and Epileptic Encephalopathies (DEEs), including Dravet syndrome. The company is also advancing another compound, LP659, for the potential treatment of rare neuroinflammatory conditions.

These FDA designations mark a milestone in the company’s journey to bring effective therapies to patients with rare neurological diseases.

  bexicaserin, clinical trials, Dravet Syndrome, FDA, Longboard Pharmaceuticals, Orphan Drug, Pharmaceutical Stocks, Rare Pediatric Disease