Soleno Therapeutics Announces U.S. FDA Acceptance for Filing and Priority Review of NDA for DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome
Soleno Therapeutics announced that the FDA has accepted for filing and granted Priority Review to its New Drug Application (NDA) for DCCR (Diazoxide Choline) Extended-Release Tablets for treating Prader-Willi syndrome (PWS) in individuals four years and older with hyperphagia. The PDUFA target action date is set for December 27, 2024. The FDA plans to hold an Advisory Committee meeting to discuss the application.
Priority Review is designated for drugs that could significantly improve treatment of serious conditions. DCCR has Breakthrough and Fast Track Designations in the U.S., and Orphan Drug Designation for PWS in both the U.S. and E.U. This acceptance marks a important milestone for Soleno and the PWS community, highlighting the potential impact of DCCR in addressing this rare and devastating condition.
Soleno Therapeutics ha annunciato che la FDA ha accettato la domanda per la registrazione e concesso una revisione prioritaria alla sua Nuova Domanda di Farmaco (NDA) per le compresse a rilascio prolungato di DCCR (Diazoxide Colina) per il trattamento della sindrome di Prader-Willi (PWS) in individui dai quattro anni in su con iperfagia. La data obiettivo per l'azione PDUFA è fissata per il 27 dicembre 2024. La FDA prevede di tenere una riunione del Comitato Consultivo per discutere la domanda.
La Revisione Prioritaria è designata per i farmaci che potrebbero migliorare significativamente il trattamento di condizioni gravi. DCCR ha Designazioni Breakthrough e Fast Track negli Stati Uniti, e Designazione di Farmaco Orfano per la PWS sia negli Stati Uniti che nell'UE. Questa accettazione segna un importante traguardo per Soleno e la comunità PWS, evidenziando il potenziale impatto di DCCR nel trattare questa rara e devastante condizione.
Soleno Therapeutics anunció que la FDA ha aceptado la presentación y otorgado revisión prioritaria a su Nueva Solicitud de Medicamento (NDA) para las tabletas de liberación prolongada de DCCR (Diazóxido Colina) para tratar el síndrome de Prader-Willi (PWS) en individuos de cuatro años o más con hiperfagia. La fecha objetivo de acción PDUFA está establecida para el 27 de diciembre de 2024. La FDA planea celebrar una reunión del Comité Asesor para discutir la solicitud.
La Revisión Prioritaria se designa para medicamentos que podrían mejorar significativamente el tratamiento de condiciones serias. DCCR tiene designaciones de Avance y Vía Rápida en EE.UU., y Designación de Medicamento Huérfano para PWS tanto en EE.UU. como en la UE. Esta aceptación marca un hito importante para Soleno y la comunidad PWS, destacando el potencial impacto de DCCR en el abordaje de esta rara y devastadora condición.
Soleno Therapeutics는 FDA가 DCCR(다이아조산 콜린) 지속 방출 정제의 새로운 약물 신청(NDA)을 접수하고 우선 심사를 승인했다고 발표했습니다. 이는 4세 이상 아동의 프라더-윌리 증후군(PWS)과 과식증을 치료하기 위한 것입니다. PDUFA 목표 행동 날짜는 2024년 12월 27일로 설정되어 있습니다. FDA는 신청서를 논의하기 위해 자문위원회 회의를 개최할 계획입니다.
우선 심사 지침은 심각한 상태의 치료를 실질적으로 개선할 수 있는 약물에 지정됩니다. DCCR은 미국에서 혁신 및 진행 속도 설계를 받았으며, 미국과 유럽 연합(EU) 모두에서 PWS에 대한 고아 약물 지정을 받았습니다. 이 수용은 Soleno와 PWS 커뮤니티에 중요한 이정표가 되며, DCCR이 이 희귀하고 파괴적인 상태를 해결하는 데 미칠 잠재적인 영향을 강조합니다.
Soleno Therapeutics a annoncé que la FDA a accepté le dossier et accordé une Revue Prioritaire à sa Demande de Nouveau Médicament (NDA) pour les Comprimés à Libération Prolongée de DCCR (Diazoxide Choline) destinés à traiter le syndrome de Prader-Willi (PWS) chez les individus de quatre ans et plus présentant de l'hyperphagie. La date d'action cible de la PDUFA est fixée au 27 décembre 2024. La FDA prévoit de tenir une réunion d'un Comité Consultatif pour discuter de la demande.
La Revue Prioritaire est désignée pour les médicaments qui pourraient améliorer de manière significative le traitement des conditions graves. DCCR a reçu des désignations de Percée et de Voie Accélérée aux États-Unis, ainsi qu'une désignation de Médicament Orphelin pour le PWS aux États-Unis et en UE. Cette acceptation marque une étape importante pour Soleno et la communauté PWS, mettant en évidence l'impact potentiel de DCCR dans le traitement de cette condition rare et dévastatrice.
Soleno Therapeutics gab bekannt, dass die FDA den Antrag zur Einreichung akzeptiert und eine Prioritätsprüfung gewährt hat für seinen Neuen Arzneimittelantrag (NDA) für DCCR (Diazoxid Cholin) Tabletten mit verlängerter Freisetzung zur Behandlung des Prader-Willi-Syndroms (PWS) bei Personen ab vier Jahren mit Hyperphagie. Das Ziel-Datum für die PDUFA-Aktion ist auf den 27. Dezember 2024 festgelegt. Die FDA plant, ein Beratungsgremium zu einberufen, um den Antrag zu erörtern.
Die Prioritätsprüfung wird für Medikamente vergeben, die die Behandlung schwerwiegender Erkrankungen erheblich verbessern könnten. DCCR hat in den USA die Auszeichnungen Durchbruch und Schnellbahn sowie die Orphan-Drug-Auszeichnung für PWS in den USA und der EU erhalten. Diese Annahme stellt einen wichtigen Meilenstein für Soleno und die PWS-Gemeinschaft dar und hebt die potenziellen Auswirkungen von DCCR bei der Bekämpfung dieser seltenen und verheerenden Erkrankung hervor.
- FDA accepted NDA filing for DCCR in Prader-Willi syndrome
- Priority Review granted, indicating potential significant improvement in treatment
- PDUFA target action date set for December 27, 2024
- DCCR has Breakthrough, Fast Track, and Orphan Drug Designations
- None.
Insights
The FDA's acceptance of Soleno's NDA for DCCR with Priority Review is a significant milestone in the drug development process. This designation, along with the December 27, 2024 PDUFA date, indicates the FDA's recognition of DCCR's potential to address an unmet medical need in Prader-Willi syndrome (PWS).
The planned Advisory Committee meeting suggests the FDA sees complexities or novelties in the application that warrant broader expert discussion. This could be due to the unique challenges of treating PWS or the drug's mechanism of action. While not necessarily negative, it adds an additional layer of scrutiny to the approval process.
The multiple designations (Breakthrough, Fast Track, Orphan Drug) underscore the drug's potential importance but don't guarantee approval. Investors should closely monitor the Advisory Committee meeting outcome, as it often influences the final FDA decision.
DCCR's potential approval for PWS patients aged four years and older with hyperphagia is noteworthy. PWS is a complex genetic disorder with treatment options, particularly for managing hyperphagia (excessive hunger), a hallmark symptom that significantly impacts quality of life.
The Priority Review status suggests that DCCR, if approved, could offer a substantial improvement over existing therapies. This is important for PWS patients and caregivers who currently lack effective treatments for managing hyperphagia.
However, it's important to note that while promising, the regulatory pathway for rare disease treatments can be challenging. The planned Advisory Committee meeting will be a critical juncture, where the risk-benefit profile of DCCR will be thoroughly evaluated. The outcome could significantly influence both the FDA's decision and market perception of DCCR's potential.
Soleno Therapeutics' progress with DCCR positions it favorably in the rare disease market. The Priority Review not only accelerates the regulatory timeline but also validates the unmet need in PWS treatment, potentially strengthening DCCR's market position if approved.
The orphan drug designation in both U.S. and E.U. markets could provide significant commercial benefits, including market exclusivity and potential tax credits. This dual-market potential enhances DCCR's commercial prospects.
However, investors should consider that rare disease drug markets, while potentially lucrative, can be challenging to penetrate due to small patient populations and complex reimbursement landscapes. The outcome of the Advisory Committee meeting will be important in gauging both regulatory sentiment and potential market reception. A positive FDA decision could significantly boost Soleno's market valuation, but any setbacks could have outsized negative impacts given the company's focus on this single asset.
PDUFA target action date set for December 27, 2024
FDA currently plans to hold an Advisory Committee meeting
REDWOOD CITY, Calif., Aug. 27, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for DCCR for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia. The FDA granted Priority Review for the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 27, 2024. Priority review is designated to applications for drugs that, if approved, would provide a significant improvement in safety or effectiveness of the treatment, prevention or diagnosis of a serious condition.
In its Priority Review Designation letter, the FDA also stated that it is currently planning to hold an advisory committee meeting to discuss the application for DCCR.
“PWS is a devastating, rare condition that significantly impacts the lives of patients and their families,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno. “The FDA’s acceptance of our NDA is a significant milestone, and designating our application for priority review reaffirms that the FDA views PWS as a serious condition. We are immensely grateful to the entire PWS community, including patients, caregivers and advocacy groups, for their ongoing support. We remain committed to continuing to work closely with the FDA through the NDA review process.”
Diazoxide choline has Breakthrough and Fast Track Designations in the U.S., as well as Orphan Drug Designation for PWS in the U.S. and E.U.
About PWS
The Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births. The hallmark symptom of this disorder is hyperphagia, a chronic and life-threatening condition characterized by feelings of intense, persistent hunger, food pre-occupation, and an extreme drive to seek and consume food, which can severely diminish the quality of life for individuals with PWS and their families. Additional characteristics of PWS include behavioral problems, cognitive disabilities, low muscle tone, short stature (when not treated with growth hormone), the accumulation of excess body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to significant mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior) and longer term, co-morbidities such as diabetes, obesity, and cardiovascular disease. In a global survey conducted by the Foundation for Prader-Willi Research,
About DCCR (Diazoxide Choline) Extended-Release Tablets
DCCR is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide and is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of individuals in a few rare diseases in neonates, infants, children and adults, but is not approved for use in PWS. Soleno conceived of and established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline and DCCR in individuals with PWS. The DCCR development program is supported by data from five completed Phase 1 clinical studies in healthy volunteers and three completed Phase 2 clinical studies, one of which was in individuals with PWS. In the PWS Phase 3 clinical development program, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and other metabolic parameters.
About Soleno Therapeutics, Inc.
Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. An NDA for its lead candidate, DCCR (diazoxide choline) extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi syndrome (PWS) is currently under review by the FDA and was granted Priority Review. For more information, please visit www.soleno.life.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the timing of any regulatory process or ultimate approvals and determining a path forward for DCCR for the treatment of PWS. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including the risks and uncertainties associated with the FDA’s review of our NDA, market conditions, as well as risks and uncertainties inherent in Soleno’s business, including those described in the company's prior press releases and in the periodic reports it files with the SEC. The events and circumstances reflected in the company's forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, the company does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
Corporate Contact:
Brian Ritchie
LifeSci Advisors, LLC
212-915-2578
FAQ
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