Soleno Therapeutics Announces U.S. FDA Approval of VYKATTM XR to Treat Hyperphagia in Prader-Willi Syndrome
Soleno Therapeutics (NASDAQ: SLNO) has received FDA approval for VYKAT XR (diazoxide choline), the first approved therapy for treating hyperphagia in adults and children aged 4 and older with Prader-Willi syndrome (PWS). The drug will be available in the U.S. starting April 2025.
The approval was based on a Phase 3 multi-center, randomized, double-blind, placebo-controlled trial where patients who switched to placebo showed significant worsening of hyperphagia compared to those remaining on VYKAT XR. The safety profile was established through four years of data across multiple studies.
The most common adverse reactions (≥10% and 2% greater than placebo) included hypertrichosis, edema, hyperglycemia, and rash. To support patient access, Soleno has launched Soleno One, a comprehensive patient support program accessible through VykatXR.com.
Soleno Therapeutics (NASDAQ: SLNO) ha ricevuto l'approvazione della FDA per VYKAT XR (diazoxide colina), la prima terapia approvata per il trattamento dell'iperfagia negli adulti e nei bambini di età pari o superiore a 4 anni affetti da sindrome di Prader-Willi (PWS). Il farmaco sarà disponibile negli Stati Uniti a partire da aprile 2025.
L'approvazione si basa su uno studio di fase 3 multicentrico, randomizzato, in doppio cieco e controllato con placebo, in cui i pazienti che sono passati al placebo hanno mostrato un significativo peggioramento dell'iperfagia rispetto a quelli che sono rimasti in trattamento con VYKAT XR. Il profilo di sicurezza è stato stabilito attraverso quattro anni di dati provenienti da diversi studi.
Le reazioni avverse più comuni (≥10% e 2% in più rispetto al placebo) includevano ipertricosi, edema, iperglicemia e rash. Per supportare l'accesso dei pazienti, Soleno ha lanciato Soleno One, un programma di supporto completo per i pazienti accessibile tramite VykatXR.com.
Soleno Therapeutics (NASDAQ: SLNO) ha recibido la aprobación de la FDA para VYKAT XR (diazóxido colina), la primera terapia aprobada para tratar la hiperfagia en adultos y niños de 4 años o más con síndrome de Prader-Willi (PWS). El medicamento estará disponible en EE. UU. a partir de abril de 2025.
La aprobación se basó en un ensayo multicéntrico de fase 3, aleatorizado, doble ciego y controlado con placebo, donde los pacientes que cambiaron a placebo mostraron un empeoramiento significativo de la hiperfagia en comparación con aquellos que permanecieron en VYKAT XR. El perfil de seguridad se estableció a través de cuatro años de datos en múltiples estudios.
Las reacciones adversas más comunes (≥10% y 2% más que el placebo) incluyeron hipertricosis, edema, hiperglucemia y erupción cutánea. Para apoyar el acceso de los pacientes, Soleno ha lanzado Soleno One, un programa integral de apoyo al paciente accesible a través de VykatXR.com.
솔레노 테라퓨틱스 (NASDAQ: SLNO)가 프라더-윌리 증후군(PWS)으로 진단받은 4세 이상의 성인 및 아동의 과식증 치료를 위한 최초의 승인된 치료제인 VYKAT XR (디아조폭스 콜린)에 대해 FDA 승인을 받았습니다. 이 약물은 2025년 4월부터 미국에서 사용할 수 있습니다.
이번 승인은 다기관, 무작위, 이중 맹검, 위약 대조 시험인 3상 시험에 기반하며, 위약으로 전환한 환자들은 VYKAT XR을 계속 복용한 환자들에 비해 과식증이 유의미하게 악화되었습니다. 안전성 프로필은 여러 연구를 통해 4년간의 데이터를 통해 설정되었습니다.
가장 흔한 부작용(≥10% 및 위약보다 2% 더 많음)으로는 다모증, 부종, 고혈당 및 발진이 포함되었습니다. 환자 접근을 지원하기 위해, 솔레노는 VykatXR.com을 통해 접근할 수 있는 포괄적인 환자 지원 프로그램인 Soleno One을 시작했습니다.
Soleno Therapeutics (NASDAQ: SLNO) a reçu l'approbation de la FDA pour VYKAT XR (diazoxide choline), la première thérapie approuvée pour traiter l'hyperphagie chez les adultes et les enfants âgés de 4 ans et plus atteints du syndrome de Prader-Willi (PWS). Le médicament sera disponible aux États-Unis à partir d'avril 2025.
L'approbation était basée sur un essai de phase 3 multicentrique, randomisé, en double aveugle et contrôlé par placebo, où les patients ayant changé pour le placebo ont montré une aggravation significative de l'hyperphagie par rapport à ceux restant sous VYKAT XR. Le profil de sécurité a été établi grâce à quatre années de données provenant de plusieurs études.
Les effets indésirables les plus courants (≥10% et 2% de plus que le placebo) comprenaient hypertrichose, œdème, hyperglycémie et éruption cutanée. Pour soutenir l'accès des patients, Soleno a lancé Soleno One, un programme complet de soutien aux patients accessible via VykatXR.com.
Soleno Therapeutics (NASDAQ: SLNO) hat die FDA-Zulassung für VYKAT XR (Diazoxid-Cholin) erhalten, die erste genehmigte Therapie zur Behandlung von Hyperphagie bei Erwachsenen und Kindern ab 4 Jahren mit Prader-Willi-Syndrom (PWS). Das Medikament wird ab April 2025 in den USA erhältlich sein.
Die Zulassung basierte auf einer multizentrischen, randomisierten, doppelblinden, placebo-kontrollierten Phase-3-Studie, in der Patienten, die auf Placebo umschalteten, eine signifikante Verschlechterung der Hyperphagie im Vergleich zu denen zeigten, die weiterhin VYKAT XR erhielten. Das Sicherheitsprofil wurde über vier Jahre Daten aus mehreren Studien etabliert.
Die häufigsten Nebenwirkungen (≥10% und 2% höher als bei Placebo) umfassten Hypertrichose, Ödeme, Hyperglykämie und Hautausschlag. Um den Zugang für Patienten zu unterstützen, hat Soleno Soleno One gestartet, ein umfassendes Patientenunterstützungsprogramm, das über VykatXR.com zugänglich ist.
- First FDA-approved therapy for hyperphagia in PWS patients
- Clear efficacy demonstrated in Phase 3 clinical trials
- Comprehensive patient support program launched for market access
- Well-established safety profile with 4+ years of clinical data
- Multiple adverse reactions reported in >10% of patients
- to patients 4 years and older only
Insights
The FDA approval of VYKAT XR represents a significant regulatory achievement for Soleno Therapeutics. This is the first approved therapy specifically targeting hyperphagia in Prader-Willi syndrome (PWS), addressing a critical unmet need in this rare disease population.
The approval was supported by robust clinical evidence from a Phase 3 randomized withdrawal study demonstrating statistically significant efficacy. Patients who switched to placebo showed worsening hyperphagia compared to those maintaining VYKAT XR treatment. The safety profile is well-characterized with over four years of accumulated data across multiple studies.
Notably, the FDA label covers a broad patient population – adults and children 4 years and older – maximizing the therapeutic's potential market. The April 2025 commercial availability indicates Soleno has manufacturing and distribution channels prepared for rapid market entry.
The launch of the Soleno One patient support program demonstrates the company's commitment to ensuring patient access, which is crucial for rare disease therapies where reimbursement can be complex. This proactive approach to commercialization suggests Soleno has strategically prepared for this approval.
For a company focused on rare diseases, securing this first FDA approval represents a transformation from a clinical-stage to commercial-stage organization, validating their development capabilities and regulatory strategy execution.
Soleno's VYKAT XR approval marks a watershed moment for the PWS therapeutic landscape. PWS affects approximately 1 in 15,000 births, creating a small but defined addressable market with virtually no competition for hyperphagia treatment.
This approval addresses the most debilitating aspect of PWS – the insatiable hunger (hyperphagia) that leads to life-threatening obesity and requires constant supervision. The testimonials from both clinical experts and patient advocacy organizations underline the significant functional impact this treatment could have on patients' daily lives.
From a market perspective, orphan drugs for rare diseases typically command premium pricing, and with no competing approved therapies, Soleno faces little pricing pressure. The broad indication covering patients 4 years and older enables treatment across most of the patient lifespan, maximizing potential revenue per patient.
The endorsements from major PWS advocacy groups (FPWR and PWSA | USA) are particularly valuable as these organizations often influence prescription decisions and payer policies in rare disease markets. Their public support signals potential strong adoption within the community.
With immediate commercial plans for April availability, Soleno demonstrates readiness to monetize this approval. For rare disease therapeutics, the transition from approval to meaningful revenue can be rapid compared to mass-market drugs, as the patient population is well-defined and often already connected to specialized treatment centers.
First approved therapy to address hyperphagia in individuals with Prader-Willi syndrome
Management to host conference call and webcast today, March 26th, at 5:30pm ET
REDWOOD CITY, Calif., March 26, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (NASDAQ: SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved VYKAT XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, for the treatment of hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome (PWS). Soleno expects VYKAT XR to be available in the U.S. beginning in April 2025.
“The approval of VYKAT XR is a significant milestone for Soleno and, most importantly, for the PWS community who have had no options to treat the most disruptive aspect of this disease,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno. “We are deeply grateful to the many individuals with PWS, their caregivers and clinical sites who participated in our trials, the advocacy groups, including FPWR and PWSA | USA, the advocates who have tirelessly supported the approval of VYKAT XR, the FDA for a collaborative review process, and our employees who have been committed to delivering VYKAT XR to those with PWS.”
“The FDA approval of VYKAT XR is an incredible achievement for the entire PWS community,” said Jennifer Miller, M.D., Professor of Pediatric Endocrinology at the University of Florida, Gainesville, who specializes in treating children and adults with PWS and is a principal investigator in the VYKAT XR clinical development program. “I am excited to have VYKAT XR available to help treat hyperphagia, which is the most life-limiting aspect of PWS. Families of people with PWS have been prisoners in their own homes because of the need to provide constant, eyes-on supervision 24/7 with access to food being completely restricted.”
“Today marks a historic day for the PWS community. The FDA’s approval of VYKAT XR represents a monumental step forward in addressing the longstanding unmet needs of individuals living with PWS and their families,” said Stacy Ward, Chief Executive Officer of the Prader-Willi Syndrome Association | USA. “Our families experience the constant and disruptive challenges of hyperphagia, and VYKAT XR offers hope to so many.”
"This approval is a testament to the power of persistence, science, and advocacy," said Susan Hedstrom, Executive Director of the Foundation for Prader-Willi Research. "For years, families and researchers have worked towards a treatment option that truly addresses the complexities of PWS. Today, we take a major step forward in changing the future for individuals navigating hyperphagia associated with PWS."
The FDA approval of VYKAT XR was based on an adequate and well-controlled study and safety data from the comprehensive clinical development program. Efficacy was established during the 16-week randomized withdrawal study period of Study 2-RWP (Study C602-RWP), a Phase 3 multi-center, randomized, double-blind, placebo-controlled trial. Individuals randomized to switch to placebo demonstrated a statistically significant worsening of hyperphagia compared with individuals who remained on VYKAT XR. Prior to participating in the randomized withdrawal period, all individuals received double-blind and/or open-label VYKAT XR for a mean duration of 3.3 years.
VYKAT XR has a well-established safety profile with over four years of data across four double-blind and/or open label studies. The primary safety analyses are based on Study 1 (Study C601) and the most common adverse reactions occurring in greater than or equal to
Patient Support for Accessing VYKAT XR
Today, Soleno has launched Soleno OneTM, a comprehensive patient support program. Information about the program is available at VykatXR.com. The Soleno One team can be reached toll-free at 1-(833)-SOLENO-1 (1-833-765-3661) from 8 a.m. to 8 p.m. ET Monday through Friday. Healthcare providers who want to submit prescriptions can visit VykatXRHCP.com to complete the Start Form that initiates the process for accessing treatment.
Conference Call and Webcast Information
Soleno management will host an investor conference call and webcast with slides to discuss the FDA’s approval of VYKAT XR (diazoxide choline) extended-release tablets today, March 26th, at 5.30pm ET. Details can be found below:
| Conference call details: | Toll-free: 1-877-423-9813 |
| International: 1-201-689-8573 | |
| Conference ID: 13752679 | |
| Call me™ (avoids waiting for an operator): | Click here |
| Webcast: | Click here |
A replay of the call will be available following the call on the Investors section of the Soleno website.
About Prader-Willi Syndrome
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.
INDICATION
VYKAT XR is indicated for the treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PWS).
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. 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 commercialization of VYKAT XR for the treatment of hyperphagia in individuals with PWS, the potential market opportunity for VYKAT XR and the ability of VYKAT XR 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.
Investor Contact:
Brian Ritchie
LifeSci Advisors, LLC
212-915-2578
Media Contact:
media@soleno.life
FAQ
When will VYKAT XR (SLNO) be available for Prader-Willi syndrome patients?
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