Longboard Pharmaceuticals Gains Rare Pediatric Disease and Orphan Drug Status for Bexicaserin in Dravet Syndrome

Longboard Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company listed on Nasdaq as LBPH, has made a significant advancement in the treatment of neurological diseases. The company recently announced that the FDA has granted both Rare Pediatric Disease designation and Orphan Drug designation to its investigational drug, bexicaserin, for the treatment of Dravet syndrome.

Dr. Randall Kaye, Longboard’s Chief Medical Officer, expressed the company's excitement over receiving these designations. He emphasized that these regulatory supports are crucial as the company continues to explore bexicaserin as a potential treatment option for patients with Developmental and Epileptic Encephalopathies (DEEs), who have substantial unmet medical needs. The Orphan Drug designation particularly aids in the regulatory progression of bexicaserin's development. Additionally, the Rare Pediatric Disease designation provides an opportunity for Longboard to receive a Priority Review Voucher. This voucher can be used to expedite the review process for another Longboard compound or can be monetized.

The Rare Pediatric Disease program is designed to encourage the development of treatments for rare pediatric conditions. Under this program, a sponsor whose drug receives approval may be eligible for a Priority Review Voucher that can be redeemed for a priority review of a different product. Sponsors also have the option to sell or transfer this voucher.

The Orphan Drug designation is a critical initiative by the FDA to support the development and assessment of new treatments for rare diseases. This designation offers incentives such as tax credits for qualified clinical trials, exemption from user fees, and potentially seven years of market exclusivity post-approval.

Longboard Pharmaceuticals is dedicated to discovering transformative medicines for neurological diseases. The company is advancing a portfolio of product candidates that act on specific G protein-coupled receptors (GPCRs). These small molecule product candidates are the result of over two decades of GPCR research. One of their leading candidates, bexicaserin (LP352), is an orally administered, centrally acting 5-hydroxytryptamine 2C (5-HT2C) receptor superagonist. It does not affect the 5-HT2B and 5-HT2A receptor subtypes. Longboard plans to initiate a global Phase 3 program for bexicaserin. Earlier this year, positive topline data was reported from a Phase 1b/2a clinical trial (the PACIFIC Study) that evaluated bexicaserin in participants aged 12 to 65 with DEEs, including Lennox-Gastaut syndrome and Dravet syndrome.

Another investigational compound by Longboard is LP659, an oral, centrally acting modulator of sphingosine-1-phosphate (S1P) receptor subtypes 1 and 5. This compound is being developed as a potential treatment for rare neuroinflammatory conditions. A Phase 1 single-ascending dose (SAD) clinical trial has been completed for LP659 in healthy volunteers.

It is important to note that both bexicaserin and LP659 are investigational compounds and have not yet received marketing approval from the FDA or any other regulatory authority. Longboard Pharmaceuticals continues to work diligently to bring these potential treatments to market, aiming to address significant unmet medical needs in the field of neurological diseases.

In summary, the recent FDA designations for bexicaserin mark important milestones for Longboard Pharmaceuticals. These designations not only facilitate regulatory support but also provide strategic opportunities for future development and commercialization of their investigational drugs aimed at treating rare and severe neurological conditions.