Longboard Pharmaceuticals Receives Rare Pediatric Disease Designation and Orphan Drug Designation for Bexicaserin (LP352) in Dravet Syndrome
Longboard Pharmaceuticals (Nasdaq: LBPH) has received Rare Pediatric Disease designation and Orphan Drug designation from the FDA for bexicaserin (LP352) in Dravet syndrome. This follows the company's earlier Breakthrough Therapy designation for treating seizures associated with Developmental and Epileptic Encephalopathies (DEEs). The Rare Pediatric Disease designation offers an opportunity for a Priority Review Voucher, which can be used for priority review of another Longboard compound or monetized. Longboard plans to initiate its global Phase 3 DEEp program, starting with DEEp SEA in Dravet syndrome, in the coming weeks.
Longboard Pharmaceuticals (Nasdaq: LBPH) ha ricevuto la designazione di Malattia Pediatrica Rara e la designazione di Farmaco Orfano dalla FDA per bexicaserin (LP352) nel contesto della sindrome di Dravet. Questo segue la precedente designazione di Terapia Innovativa della società per il trattamento delle crisi associate alle Encefalopatie Sviluppo e Epilettiche (DEEs). La designazione di Malattia Pediatrica Rara offre un'opportunità per un Voucher di Revisione Prioritaria, che può essere utilizzato per la revisione prioritaria di un altro composto di Longboard o monetizzato. Longboard prevede di avviare il suo programma globale Phase 3 DEEp, a partire con DEEp SEA nella sindrome di Dravet, nelle prossime settimane.
Longboard Pharmaceuticals (Nasdaq: LBPH) ha recibido la designación de Enfermedad Pediátrica Rara y la designación de Medicamento Huérfano de la FDA para bexicaserin (LP352) en el síndrome de Dravet. Esto sigue la designación de Terapia Innovadora de la compañía para tratar las convulsiones asociadas con las Encefalopatías del Desarrollo y Epilépticas (DEEs). La designación de Enfermedad Pediátrica Rara ofrece una oportunidad para un Voucher de Revisión Prioritaria, que puede usarse para la revisión prioritaria de otro compuesto de Longboard o monetizarse. Longboard planea iniciar su programa global Phase 3 DEEp, comenzando con DEEp SEA en el síndrome de Dravet, en las próximas semanas.
롱보드 제약(나스닥: LBPH)은 드라베 증후군에 대한 베시카세린 (LP352)의 희귀 소아 질병 지정 및 희귀 약물 지정을 FDA로부터 받았습니다. 이는 발달성 및 간질성 뇌병증(DEEs)과 관련된 발작 치료를 위한 회사의 돌파구 치료 지정에 이은 것입니다. 희귀 소아 질병 지정은 다른 롱보드 화합물의 우선 검토를 위해 사용할 수 있거나 수익화할 수 있는 우선 검토 바우처의 기회를 제공합니다. 롱보드는 향후 몇 주 안에 드라베 증후군에서 DEEp SEA로 시작하여 전 세계 3상 DEEp 프로그램을 시작할 계획입니다.
Longboard Pharmaceuticals (Nasdaq: LBPH) a reçu la désignation de Maladie Pédiatrique Rare et la désignation de Médicament Orphelin de la FDA pour bexicaserin (LP352) dans le cadre du syndrome de Dravet. Cela fait suite à la précédente dénomination de Thérapie Innovante de la société pour le traitement des crises associées aux Encephalopathies de Développement et Épileptiques (DEEs). La désignation de Maladie Pédiatrique Rare offre une opportunité pour un Bon de Révision Prioritaire, qui peut être utilisé pour la révision prioritaire d'un autre composé de Longboard ou monétisé. Longboard prévoit de lancer son programme mondial Phase 3 DEEp, en commençant par DEEp SEA dans le syndrome de Dravet, dans les semaines à venir.
Longboard Pharmaceuticals (Nasdaq: LBPH) hat von der FDA die Rare Pediatric Disease designation und die Orphan Drug designation für bexicaserin (LP352) im Fall des Dravet-Syndroms erhalten. Dies folgt der früheren Breakthrough Therapy designation des Unternehmens zur Behandlung von Anfällen, die mit Entwicklungs- und Epileptischen Enzephalopathien (DEEs) verbunden sind. Die Rare Pediatric Disease designation bietet die Möglichkeit eines Priority Review Voucher, der für die vorrangige Überprüfung eines anderen Longboard-Verbindungsmittels verwendet oder monetarisiert werden kann. Longboard plant, in den kommenden Wochen sein globales Phase 3 DEEp Programm zu starten, beginnend mit DEEp SEA im Dravet-Syndrom.
- Received Rare Pediatric Disease designation for bexicaserin in Dravet syndrome
- Granted Orphan Drug designation for bexicaserin in Dravet syndrome
- Opportunity to receive a Priority Review Voucher
- Previously received Breakthrough Therapy designation for seizures associated with DEEs
- Planned initiation of global Phase 3 DEEp program for Dravet syndrome
- None.
The FDA's granting of Rare Pediatric Disease and Orphan Drug designations for bexicaserin in Dravet syndrome is a significant milestone for Longboard Pharmaceuticals. These designations offer several benefits:
- Enhanced regulatory support and guidance from the FDA
- Potential for 7 years of market exclusivity upon approval
- Tax credits for clinical trial costs
- Waiver of prescription drug user fees
The Priority Review Voucher opportunity could expedite future drug approvals or provide additional revenue if sold. This news strengthens bexicaserin's market potential and Longboard's position in the rare disease space, particularly in pediatric epilepsy treatments.
This regulatory breakthrough could significantly impact Longboard Pharmaceuticals' (NASDAQ: LBPH) financial outlook. The Orphan Drug designation's benefits, including tax credits and fee waivers, could reduce development costs. The potential Priority Review Voucher is a valuable asset, often selling for
The multiple FDA designations for bexicaserin position Longboard Pharmaceuticals favorably in the competitive landscape of Dravet syndrome treatments. Current market leaders include GW Pharmaceuticals' Epidiolex and Zogenix's Fintepla. Bexicaserin's unique mechanism of action as a 5-HT2C superagonist could differentiate it from existing therapies. The global Dravet syndrome market is projected to reach
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U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation and Rare Pediatric Disease designation for bexicaserin for the treatment of Dravet syndrome
“We are thrilled to receive both Rare Pediatric Disease and Orphan Drug designations for bexicaserin in Dravet syndrome. As the first company to receive Breakthrough Therapy designation (BTD) for the treatment of seizures associated with Developmental and Epileptic Encephalopathies (DEEs), we appreciate the regulatory support that BTD provides as we continue on our journey to study bexicaserin as a potential treatment option for DEE patients with significant unmet medical needs. Receiving Orphan Drug designation in Dravet syndrome provides us with further regulatory support as we progress the development of bexicaserin. Additionally, the granting of the Rare Pediatric Disease designation provides us with an opportunity to receive a Priority Review Voucher which can be used for priority review of another Longboard compound or monetized,” stated Dr. Randall Kaye, Longboard’s Chief Medical Officer. “We appreciate the FDA’s ongoing support and look forward to initiating our global Phase 3 DEEp program, starting with DEEp SEA in Dravet syndrome in the coming weeks.”
About Rare Pediatric Disease Designation and Priority Review Vouchers (PRVs)
The Rare Pediatric Disease program aims to incentivize drug development for rare pediatric diseases. Under this program, a sponsor who receives an approval for a drug or biological product for a rare pediatric disease may qualify for a Priority Review Voucher (PRV) that can be redeemed to receive priority review for a different product. The sponsor may also transfer or sell the PRV to another sponsor.
About Orphan Drug Designation
Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. The FDA has authority to grant Orphan Drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan Drug designation qualifies sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees, and the potential for seven years of market exclusivity after approval.
ABOUT LONGBOARD PHARMACEUTICALS
Longboard Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases. Longboard is working to advance a portfolio of centrally acting product candidates designed to be highly selective for specific G protein-coupled receptors (GPCRs). Longboard’s small molecule product candidates are based on more than 20 years of GPCR research. Longboard plans to advance bexicaserin (LP352), an oral, centrally acting 5-hydroxytryptamine 2C (5-HT2C) receptor superagonist, with no observed impact on 5-HT2B and 5-HT2A receptor subtypes, into a global Phase 3 program. Earlier this year, Longboard reported positive topline data from a Phase 1b/2a clinical trial (the PACIFIC Study) evaluating bexicaserin in participants ages 12 to 65 years old with Developmental and Epileptic Encephalopathies (DEEs), including Lennox-Gastaut syndrome, Dravet syndrome and other DEEs. Longboard is also evaluating LP659, an oral, centrally acting, sphingosine-1-phosphate (S1P) receptor subtypes 1 and 5 modulator, which is in development for the potential treatment of rare neuroinflammatory conditions. Longboard recently completed a Phase 1 single-ascending dose (SAD) clinical trial for LP659 in healthy volunteers.
Bexicaserin and LP659 are investigational compounds that are not approved for marketing by the FDA or any other regulatory authority.
FORWARD-LOOKING STATEMENTS
Certain statements in this press release are forward-looking statements that involve a number of risks and uncertainties. In some cases, you can identify forward-looking statements by words such as “focus”, “potential”, “opportunity”, “can”, “look forward”, “aim”, “may”, “working to”, “designed to”, “plan”, or the negative, plural or other tenses of these words, references to future dates or time periods, or other comparable language, and they may include, without limitation, statements about the following: Longboard’s product candidates and programs (including bexicaserin’s potential, development, advancement into a global Phase 3 program, and Breakthrough Therapy, Rare Pediatric Disease and Orphan Drug designations and their potential benefits, including eligibility for a Priority Review Voucher (PRV)), plans, focus and work. For such statements, Longboard claims the protection of the Private Securities Litigation Reform Act of 1995. Actual events or results may differ materially from Longboard’s expectations. Factors that could cause actual results to differ materially from those stated or implied by Longboard’s forward-looking statements include, but are not limited to, the following: the rare pediatric disease PRV program will begin to sunset after September 30, 2024, and if the program is not reauthorized, we will only be eligible to receive a PRV if bexicaserin receives marketing approval by September 30, 2026; the standards for Breakthrough Therapy, Rare Pediatric Disease and Orphan Drug designations are not the same as the standard for drug approval, the clinical evidence supporting Breakthrough Therapy designation is preliminary, and not all drugs designated as Breakthrough Therapies ultimately will be shown to have substantial improvement over available therapies; the FDA may later decide to rescind a designation if it determines the designation is no longer supported by subsequent data; Longboard’s product candidates are in a lengthy research and development process, the timing, manner and outcome of research, development and regulatory review is uncertain, and Longboard’s product candidates, including bexicaserin and LP659, may not advance in research or development or be approved for marketing; results of clinical trials and other studies are subject to different interpretations and may not be predictive of future results; topline or interim data may not accurately reflect the complete results of a particular study or trial and remain subject to audit, and final data may differ materially from topline or interim data; enrolling participants in clinical trials is competitive and challenging; risks related to unexpected or unfavorable new data; nonclinical and clinical data is voluminous and detailed, and regulatory agencies may interpret or weigh the importance of data differently and reach different conclusions than Longboard or others, request additional information, have additional recommendations or change their guidance or requirements before or after approval; risks related to Longboard’s limited operating history, financial position and need for additional capital; Longboard will need additional managerial and financial resources to advance all of its programs, and you and others may not agree with the manner Longboard allocates its resources; risks related to the development and commercialization of Longboard’s product candidates; risks related to relying on licenses or collaborative arrangements; other risks related to Longboard’s dependence on third parties; competition; product liability or other litigation or disagreements with others; government and third-party payor actions, including relating to reimbursement and pricing; risks related to regulatory compliance; and risks related to Longboard’s and third parties’ intellectual property rights. Additional factors that could cause actual results to differ materially from those stated or implied by Longboard’s forward-looking statements are disclosed in Longboard’s filings with the Securities and Exchange Commission (SEC). These forward-looking statements represent Longboard’s judgment as of the time of this release. Longboard disclaims any intent or obligation to update these forward-looking statements, other than as may be required under applicable law.
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CORPORATE CONTACT:
Megan E. Knight
VP, Head of Investor Relations
IR@longboardpharma.com
858.789.9283
Source: Longboard Pharmaceuticals, Inc.
FAQ
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