Soleno Therapeutics Announces FDA Extension of Review Period for DCCR (Diazoxide Choline) Extended-Release Tablets in Prader-Willi Syndrome
Soleno Therapeutics announced that the FDA has extended the review period for DCCR (diazoxide choline) extended-release tablets for treating Prader-Willi syndrome (PWS) in patients four years and older with hyperphagia. The new PDUFA target action date is pushed back by three months to March 27, 2025. The extension follows the FDA's determination that responses to recent information requests constituted a major amendment to the NDA. The FDA did not cite any safety, efficacy, or manufacturing concerns. The drug has received Breakthrough, Fast Track, and Orphan Drug Designations.
Soleno Therapeutics ha annunciato che la FDA ha esteso il periodo di revisione per le compresse a rilascio prolungato di DCCR (choline diazoxide) per il trattamento della sindrome di Prader-Willi (PWS) in pazienti di età pari o superiore a quattro anni con iperfagia. La nuova data obiettivo di azione PDUFA è stata posticipata di tre mesi al 27 marzo 2025. L'estensione segue la determinazione della FDA secondo cui le risposte alle recenti richieste di informazioni costituivano una modifica significativa alla NDA. La FDA non ha citato preoccupazioni riguardanti sicurezza, efficacia o produzione. Il farmaco ha ricevuto le designazioni di Breakthrough, Fast Track e Orphan Drug.
Soleno Therapeutics anunció que la FDA ha extendido el período de revisión para las tabletas de liberación prolongada de DCCR (colina diazóxido) para tratar el síndrome de Prader-Willi (PWS) en pacientes de cuatro años o más con hiperfagia. La nueva fecha objetivo de acción PDUFA se ha pospuesto tres meses hasta el 27 de marzo de 2025. La extensión sigue la determinación de la FDA de que las respuestas a las recientes solicitudes de información constituyeron una enmienda importante a la NDA. La FDA no citó preocupaciones sobre seguridad, eficacia ni fabricación. El fármaco ha recibido las designaciones de Breakthrough, Fast Track y Orphan Drug.
솔레노 테라퓨틱스(Soleno Therapeutics)는 FDA가 4세 이상이고 과식증을 앓고 있는 프래더-윌리 증후군(PWS) 환자를 위한 DCCR(디아조옥사이드 콜린) 장기 방출 정제의 심사 기간을 연장했다고 발표했습니다. 새로운 PDUFA 목표 조치 날짜는 2025년 3월 27일로 3개월 연기되었습니다. 이번 연장은 FDA가 최근 정보 요청에 대한 응답이 NDA의 주요 수정 사항을 구성한다고 판단한 후 이루어졌습니다. FDA는 안전성, 효능 또는 제조에 대한 우려를 언급하지 않았습니다. 이 약물은 Breakthrough, Fast Track 및 Orphan Drug 지정받았습니다.
Soleno Therapeutics a annoncé que la FDA a prolongé le délai d'examen pour les comprimés à libération prolongée de DCCR (choline diazoxide) pour le traitement du syndrome de Prader-Willi (PWS) chez les patients de quatre ans et plus présentant une hyperphagie. La nouvelle date cible d'action PDUFA est repoussée de trois mois au 27 mars 2025. Cette prolongation fait suite à la décision de la FDA selon laquelle les réponses aux demandes d'informations récentes constituaient une modification majeure de la NDA. La FDA n'a pas cité de préoccupations concernant la sécurité, l'efficacité ou la fabrication. Le médicament a reçu les désignations Breakthrough, Fast Track et Orphan Drug.
Soleno Therapeutics gab bekannt, dass die FDA den Prüfungszeitraum für DCCR (Diazoxid-Cholin) zur Behandlung des Prader-Willi-Syndroms (PWS) bei Patienten ab vier Jahren mit Hyperphagie verlängert hat. Das neue PDUFA-Zieltermin wurde um drei Monate auf den 27. März 2025 verschoben. Die Verlängerung folgt der Feststellung der FDA, dass die Antworten auf die neuesten Informationsanforderungen eine wesentliche Änderung des NDA darstellten. Die FDA nannte keine Sicherheits-, Wirksamkeits- oder Herstellungsbedenken. Das Medikament erhielt die Auszeichnungen Breakthrough, Fast Track und Orphan Drug.
- FDA did not raise any safety, efficacy, or manufacturing concerns
- Drug maintains Priority Review status
- Product holds multiple valuable FDA designations (Breakthrough, Fast Track, Orphan Drug)
- Three-month delay in potential FDA approval timeline
- Additional information requests from FDA requiring major amendment to NDA
Insights
The FDA's 3-month review extension for DCCR in Prader-Willi syndrome represents a procedural delay rather than a substantive concern. The extension was triggered by Soleno's responses to information requests, which the FDA classified as a "major amendment." Critically, the FDA did not raise any safety, efficacy, or manufacturing issues.
The drug maintains its Priority Review status and previous Breakthrough, Fast Track and Orphan Drug designations, indicating the FDA's recognition of DCCR's potential significance in treating PWS. With no approved treatments for hyperphagia in PWS, DCCR could capture a valuable orphan drug market if approved. The new March 27, 2025 PDUFA date provides a clear timeline for potential market entry.
PDUFA target action date extended by three months to March 27, 2025
REDWOOD CITY, Calif., Nov. 26, 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 extended the review period for the New Drug Application (NDA) for DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia. The new Prescription Drug User Fee Act (PDUFA) target action date is March 27, 2025.
The FDA determined that responses to recent information requests constituted a major amendment to the NDA, resulting in the extension of the PDUFA goal date by three months. The extension allows the FDA time to complete their review, including that of the recently submitted information. The FDA did not cite any safety, efficacy or manufacturing concerns in their correspondence.
Soleno submitted the NDA to the FDA on June 27, 2024. The FDA accepted the NDA and granted Priority Review in August 2024. Diazoxide choline has been granted Breakthrough and Fast Track Designations in the U.S., as well as Orphan Drug Designation in the U.S. and E.U. for the treatment of patients with PWS.
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
When is the new PDUFA date for Soleno's DCCR (SLNO) after FDA extension?
Why did the FDA extend the review period for Soleno's DCCR (SLNO)?
What designations has DCCR (SLNO) received from the FDA for PWS treatment?
Did the FDA raise any safety or efficacy concerns for Soleno's DCCR (SLNO)?
