Soleno Therapeutics Announces Submission and EMA Validation of Marketing Authorization Application for Diazoxide Choline Prolonged-Release Tablets for the Treatment of Hyperphagia in Patients with Prader-Willi Syndrome
Soleno Therapeutics (NASDAQ: SLNO) announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for Diazoxide Choline Prolonged-Release Tablets (DCCR) to treat hyperphagia in Prader-Willi syndrome (PWS) patients aged four and older. The treatment, recently approved by the FDA under the brand name VYKAT™ XR in March 2025, targets approximately 9,500 PWS patients across the UK, France, Germany, Italy, and Spain.
The company has received Orphan Drug Designation in the EU, potentially granting up to 10 years of market exclusivity if approved, along with additional regulatory and financial benefits. This validation marks a crucial step in Soleno's mission to provide treatment for PWS patients in the EU.
Soleno Therapeutics (NASDAQ: SLNO) ha annunciato che l'Agenzia Europea per i Medicinali (EMA) ha convalidato la sua Domanda di Autorizzazione all'Immissione in Commercio (MAA) per le Compresse a Rilascio Prolungato di Diazossido Colina (DCCR) per il trattamento dell'iperfagia nei pazienti con sindrome di Prader-Willi (PWS) di età pari o superiore a quattro anni. Il trattamento, recentemente approvato dalla FDA con il nome commerciale VYKAT™ XR a marzo 2025, è destinato a circa 9.500 pazienti PWS nel Regno Unito, Francia, Germania, Italia e Spagna.
L'azienda ha ottenuto la Designazione di Farmaco Orfano nell'UE, che potrebbe garantire fino a 10 anni di esclusiva di mercato in caso di approvazione, oltre a ulteriori vantaggi regolatori e finanziari. Questa convalida rappresenta un passo fondamentale nella missione di Soleno di offrire un trattamento per i pazienti PWS nell'UE.
Soleno Therapeutics (NASDAQ: SLNO) anunció que la Agencia Europea de Medicamentos (EMA) ha validado su Solicitud de Autorización de Comercialización (MAA) para las Tabletas de Diazóxido Colina de Liberación Prolongada (DCCR) para tratar la hiperfagia en pacientes con síndrome de Prader-Willi (PWS) de cuatro años en adelante. El tratamiento, aprobado recientemente por la FDA bajo el nombre comercial VYKAT™ XR en marzo de 2025, está dirigido a aproximadamente 9,500 pacientes con PWS en Reino Unido, Francia, Alemania, Italia y España.
La compañía ha recibido la Designación de Medicamento Huérfano en la UE, lo que podría otorgar hasta 10 años de exclusividad en el mercado si se aprueba, junto con beneficios regulatorios y financieros adicionales. Esta validación representa un paso crucial en la misión de Soleno de proporcionar tratamiento a pacientes con PWS en la UE.
Soleno Therapeutics (NASDAQ: SLNO)는 유럽 의약품청(EMA)이 4세 이상 프래더-윌리 증후군(PWS) 환자의 과식증 치료를 위한 디아조사이드 콜린 서방정(DCCR)에 대한 판매 허가 신청서(MAA)를 승인했다고 발표했습니다. 이 치료제는 2025년 3월 FDA에서 VYKAT™ XR라는 브랜드명으로 최근 승인되었으며, 영국, 프랑스, 독일, 이탈리아, 스페인에서 약 9,500명의 PWS 환자를 대상으로 합니다.
회사는 EU에서 희귀 의약품 지정(Orphan Drug Designation)을 받아 승인 시 최대 10년간 시장 독점권과 추가적인 규제 및 재정 혜택을 받을 수 있습니다. 이번 승인 검증은 EU 내 PWS 환자 치료를 위한 Soleno의 중요한 진전입니다.
Soleno Therapeutics (NASDAQ: SLNO) a annoncé que l'Agence européenne des médicaments (EMA) a validé sa demande d'autorisation de mise sur le marché (MAA) pour les comprimés à libération prolongée de Diazoxide Choline (DCCR) destinés au traitement de l'hyperphagie chez les patients atteints du syndrome de Prader-Willi (PWS) âgés de quatre ans et plus. Ce traitement, récemment approuvé par la FDA sous le nom commercial VYKAT™ XR en mars 2025, cible environ 9 500 patients PWS au Royaume-Uni, en France, en Allemagne, en Italie et en Espagne.
L'entreprise a obtenu la désignation de médicament orphelin dans l'UE, ce qui pourrait lui accorder jusqu'à 10 ans d'exclusivité commerciale en cas d'approbation, ainsi que des avantages réglementaires et financiers supplémentaires. Cette validation constitue une étape clé dans la mission de Soleno visant à fournir un traitement aux patients PWS dans l'UE.
Soleno Therapeutics (NASDAQ: SLNO) gab bekannt, dass die Europäische Arzneimittel-Agentur (EMA) den Antrag auf Marktzulassung (MAA) für die Diazoxid-Cholin Retardtabletten (DCCR) zur Behandlung der Hyperphagie bei Patienten mit Prader-Willi-Syndrom (PWS) ab vier Jahren validiert hat. Die Behandlung, die im März 2025 von der FDA unter dem Markennamen VYKAT™ XR zugelassen wurde, richtet sich an etwa 9.500 PWS-Patienten in Großbritannien, Frankreich, Deutschland, Italien und Spanien.
Das Unternehmen hat in der EU die Orphan-Drug-Designation erhalten, die im Falle einer Zulassung bis zu 10 Jahre Marktexklusivität sowie weitere regulatorische und finanzielle Vorteile gewähren kann. Diese Validierung stellt einen wichtigen Schritt in Solenos Mission dar, PWS-Patienten in der EU eine Behandlung zu bieten.
- FDA approval already secured in March 2025 under brand name VYKAT™ XR
- Orphan Drug Designation in EU could provide 10 years of market exclusivity
- Large potential market with 9,500 PWS patients in key European countries
- Treatment addresses hyperphagia, the most life-limiting aspect of PWS
- None.
REDWOOD CITY, Calif., May 22, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that Soleno’s Marketing Authorization Application (MAA) seeking regulatory approval of Diazoxide Choline Prolonged-Release Tablets (previously referred to as DCCR) for the treatment of adults and children four years and older with Prader-Willi syndrome (PWS) who have hyperphagia had been validated by the European Medicines Agency (EMA).
“The validation of our MAA represents the next significant milestone in our mission to deliver this important therapy to the broad PWS community, including those in the EU,” said Anish Bhatnagar, M.D., Chairman and Chief Executive Officer of Soleno Therapeutics. “Based on the data generated, DCCR has the potential to help treat hyperphagia, which is the most life-limiting aspect of PWS. We look forward to working closely with European regulators during the review process and intend to make DCCR available to patients in the EU as expeditiously as possible, if approved.”
Soleno estimates that there are approximately 9,500 patients with PWS in the United Kingdom, France, Germany, Italy and Spain combined1. The Company has been granted Orphan Drug Designation for diazoxide choline in the EU for the treatment of PWS, which could provide Soleno with up to 10 years of market exclusivity in the EU, if approved, in addition to certain other regulatory and financial incentives.
DCCR was approved by the U.S. Food and Drug Administration (FDA) under the brand name VYKAT™ XR on March 26, 2025.
About PWS
Prader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder caused by an abnormality in the gene expression on chromosome 15.
The Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births. The defining symptom of PWS is hyperphagia, a chronic and life-threatening condition characterized by an intense persistent sensation of hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behavior problems, and a lack of normal satiety, which can severely diminish the quality of life for individuals with PWS and their families. 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.
U.S. INDICATION
VYKAT XR is indicated in the U.S. for the treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PWS).
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1 Based on mid-point of Orphanet Birth Prevalence rate of 1 in 15,000 to 30,000
IMPORTANT SAFETY INFORMATION
Contraindications
Use of VYKAT XR is contraindicated in patients who have a known hypersensitivity to diazoxide, other components of VYKAT XR, or to thiazides.
Warnings and Precautions
Hyperglycemia
Hyperglycemia, including diabetic ketoacidosis, has been reported. Before initiating VYKAT XR, test fasting plasma glucose (FPG) and HbA1c; optimize blood glucose in patients who have hyperglycemia. During treatment, regularly monitor fasting glucose (FPG or fasting blood glucose) and HbA1c. Monitor fasting glucose more frequently during the first few weeks of treatment in patients with risk factors for hyperglycemia.
Risk of Fluid Overload
Edema, including severe reactions associated with fluid overload, has been reported. Monitor for signs or symptoms of edema or fluid overload. VYKAT XR has not been studied in patients with compromised cardiac reserve and should be used with caution in these patients.
Adverse Reactions
The most common adverse reactions (incidence ≥
Please see the full Prescribing Information, including Medication Guide.
About Soleno Therapeutics, Inc.
Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company’s first commercial product, VYKAT XR (diazoxide choline) extended-release tablets, formerly known as DCCR, is a once-daily oral treatment for hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome and was approved by the U.S. Food and Drug Administration (FDA) on March 26, 2025. 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 regulatory approval process for Diazoxide Choline Prolonged-Release Tablets in the EU, the timing of commercialization of Diazoxide Choline Prolonged-Release Tablets in the EU, the potential market opportunity for Diazoxide Choline Prolonged-Release Tablets and the ability of Diazoxide Choline Prolonged-Release Tablets to address the unmet needs of the PWS community. 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 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
Media Contact:
media@soleno.life
FAQ
What is the status of SLNO's Marketing Authorization Application for DCCR in Europe?
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How many potential patients could benefit from SLNO's DCCR treatment in Europe?
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