Vykat XR (diazoxide choline controlled release) / Soleno Therap, PANTHERx Rare, Neurocrine Biosciences - LARVOL DELTA

THE COST OF SATIETY: PRADER-WILLI SYNDROME, VYKAT XR, AND ACUTE PULMONARY HYPERTENSION (CHEST 2026) - No abstract available

Cardiovascular • Prader–Willi syndrome • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Standardizing hyperphagia outcomes: addressing the need for rigorous causality assessment. (PubMed, Ann Med Surg (Lond)) - No abstract available

Journal • Prader–Willi syndrome

Impact of Long-Term Diazoxide Choline Extended Release (DCCR) Treatment and the Prader-Willi Syndrome Profile Questionnaire (PWSP) (ENDO 2026) - "DCCR administration significantly improved PWS-related behaviors for up to 3 years as demonstrated by improved scores across all domains of the PWSP questionnaire as compared to natural history controls."

CNS Disorders • Depression • Mood Disorders • Prader–Willi syndrome

Long-Term Reductions of Hyperphagia with Diazoxide Choline Extended Release (DCCR) in Participants with Prader-Willi Syndrome (ENDO 2026) - "DCCR administration resulted in significant, sustained improvements up to 3 years in hyperphagia as demonstrated by reduction in HQ-CT total score compared to natural history controls who did not spontaneously improve. DCCR represents an effective, long-term intervention for hyperphagia in patients with PWS."

Prader–Willi syndrome

Efficacy and Safety of Resuming Diazoxide Choline Extended-Release (DCCR) after 16-week Randomized Withdrawal in Prader-Willi Syndrome (PWS) (Study C614) (ENDO 2026) - "In study C614, resuming DCCR for 2 years after 16-week randomized withdrawal was associated with improvements in both hyperphagia and behavioral symptoms characteristic of PWS. BMI remained relatively stable. Long-term DCCR treatment was generally well-tolerated with an AE profile consistent with that observed in previous clinical trials of DCCR in PWS."

Clinical • Late-breaking abstract • CNS Disorders • Depression • Diabetes • Metabolic Disorders • Mood Disorders • Prader–Willi syndrome

Impact of Long-Term Diazoxide Choline Extended Release (DCCR) Treatment and the Prader-Willi Syndrome Profile Questionnaire (PWSP) (ENDO 2026) - "DCCR administration significantly improved PWS-related behaviors for up to 3 years as demonstrated by improved scores across all domains of the PWSP questionnaire as compared to natural history controls."

CNS Disorders • Depression • Mood Disorders • Prader–Willi syndrome

Long-term Administration of Diazoxide Choline Prolonged-Release (DCCR) Tablets in People with Prader-Willi Syndrome: Changes in Lean Body Mass and Lean Mass Index (ECO 2026) - P3 | "Oral semaglutide 25 mg effects on (A) weight loss and (B) physical function score in participants with poor PF and in the overall study population... In people with PWS, DCCR administration was associated with progressive increases in lean body mass through 3 years, with greater increases realized in those who had overweight or obesity at baseline. DCCR administration was also associated with increases in LMI compared to the published natural history of the syndrome, particularly in pre-pubertal children, but also in adults where no further increases in LMI are expected. These increases are expected to be beneficial to people with PWS."

Metabolic Disorders • Obesity • Prader–Willi syndrome

Diazoxide Choline Prolonged-Release for Hyperphagia of Patients with Prader-Willi Syndrome Who Have Both Obesity/ Overweight and Healthy Body Weight (ECO 2026) - "DCCR significantly improves hyperphagia in patients with PWS, regardless of obesity/overweight status. These findings suggest that DCCR offers a novel therapeutic approach beyond strict environmental controls, benefiting patients with PWS who have both overweight/obesity and normal body weight. Conflicts of Interest & Funding: Research relating to this abstract was funded by Soleno Therapeutics, Inc."

Clinical • Genetic Disorders • Obesity • Prader–Willi syndrome

Vykat XR (diazoxide choline-extended release): a new FDA-approved treatment for hyperphagia in Prader-Willi syndrome. (PubMed, Ann Med Surg (Lond)) - "On March 27, 2025, the U.S. Food and Drug Administration (FDA) approved Vykat XR (diazoxide choline-extended release tablets) for the treatment of hyperphagia in individuals aged 4 years and older with Prader-Willi Syndrome (PWS). This marks the first FDA-approved pharmacologic therapy targeting hyperphagia in this population, addressing a long-standing unmet need and offering new hope for patients, caregivers, and clinicians."

FDA event • Journal • Prader–Willi syndrome

Open-Label Study of DCCR in Patients with Prader-Willi Syndrome (clinicaltrialsregister.eu) - P3 | N=83 | Sponsor: Soleno Therapeutics UK LTD

New P3 trial • Developmental Disorders • Genetic Disorders • Prader–Willi syndrome

Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome (clinicaltrials.gov) - P=N/A | N=200 | Enrolling by invitation | Sponsor: Soleno Therapeutics, Inc.

New trial • Prader–Willi syndrome

Diazoxide Choline Extended-Release Tablets in Prader-Willi Syndrome: A Randomized, Double-Blind, Withdrawal Period Study. (PubMed, J Clin Endocrinol Metab) - "Continued DCCR treatment was superior to placebo for hyperphagia. DCCR appears to offer meaningful therapeutic benefits for people with Prader-Willi syndrome."

Clinical • Journal • Metabolic Disorders • Prader–Willi syndrome

Soleno Product Theater - Hyperphagia in Prader-Willi Syndrome: VYKAT XR(diazoxide choline) - The First and Only FDA-Approved Treatment for Patients 4+ (OBESITY WEEK 2025) - "Description:Prader-Willi Syndrome (PWS) is a rare genetic disorder associated with the onset of hyperphagia in early childhood that continues into adulthood. This persistent, intense hunger affects nearly all individuals with PWS.This session will cover:The burden and impact of hyperphagia in PWSClinical efficacy and safety data for VYKAT™ XR (diazoxide choline)Treatment initiation, dosing, and monitoring with VYKAT™ XR (diazoxide choline)"

Clinical • Genetic Disorders • Prader–Willi syndrome

SI03-03. Introducing VYKAT™ XR (diazoxide choline) extended-release: The First and Only FDA-Approved Treatment for Hyperphagia in Adults and Children (≥4 Years) With Prader-Willi Syndrome (PWS) (ENDO 2025) - "Join us for a peer-led session covering: • The impact of hyperphagia in PWS • Clinical efficacy and safety data for VYKAT XR • Dosing, administration, and how to initiate treatment • A powerful firsthand account from a caregiver whose loved one is using VYKAT XR Don't miss this opportunity to learn from experts and gain a firsthand perspective on managing hyperphagia in PWS. Important Safety Information, full Prescribing Information, and Medication Guide for VYKAT XR will be available at the Science & Innovation Theater or online at VYKATXRHCP.com."

Clinical • Genetic Disorders • Pediatrics • Prader–Willi syndrome

The Glycemic Outcomes of Diazoxide Choline Extended-Release (DCCR) Tablets for Administered for Hyperphagia in Individuals with Prader-Willi Syndrome Over 4 Years (ENDO 2025) - "Administration of DCCR for the treatment of hyperphagia in individuals with PWS was generally well tolerated and associated with non-clinically significant increases in mean HbA1c over 3 years of use. Hyperglycemic adverse events were readily monitorable and generally manageable with standard of care concomitant medications allowing for continuation of DCCR therapy. Overall, the safety profile of DCCR in patients with PWS remains favorable."

Clinical • Diabetes • Metabolic Disorders • Prader–Willi syndrome • Type 2 Diabetes Mellitus

Characterization of Peripheral Edema in Individuals with Prader-Willi Syndrome During Long-term Administration of Diazoxide Choline Extended-Release Tablet (DCCR) Over 4.5 Years (ENDO 2025) - "Administration of DCCR to patients with PWS was associated with peripheral edema in a minority of C601 + C602-OLE participants. When present, peripheral edema tended to occur early in treatment, was usually Grade 1 or 2, and infrequently resulted in study medication interruption or discontinuation. Overall, DCCR was well tolerated in the intended population."

Clinical • Metabolic Disorders • Prader–Willi syndrome

Soleno Therapeutics, Inc. (SLNO) Secures EMA Validation for PWS Treatment VYKAT XR (Insider Monkey) - "Soleno Therapeutics, Inc...has achieved a major regulatory milestone as the European Medicines Agency (EMA) validated its Marketing Authorization Application for Diazoxide Choline Prolonged-Release Tablets (formerly DCCR) to treat hyperphagia in Prader-Willi syndrome (PWS) for patients aged four and older."

EMA filing • Prader–Willi syndrome

Expected vs. Observed Mortality Rates, Expressed As Number Needed to Treat, From a Phase 3 Clinical Trial Program of Patients With Hyperphagia and Prader-Willi Syndrome Treated With Diazoxide Choline Extended Release (DCCR) (ISPOR 2025) - "PWS is a rare condition with many unmet needs and associated with an elevated mortality rate. Patients treated with DCCR did not experience a mortality event through up to 4.5 years of continued exposure. Given the epidemiologically expected mortality rate, the resulting NNT suggests that treating patients with DCCR may also reduce mortality events."

Clinical • P3 data • Genetic Disorders • Prader–Willi syndrome

Soleno Therapeutics Announces Presentations Featuring VYKAT(TM) XR in Prader-Willi Syndrome at ISPOR 2025 (GlobeNewswire) - "Soleno Therapeutics, Inc...today announced that it will present data on the burden of Prader-Willi syndrome (PWS) and findings from its clinical development program of VYKAT XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, for the treatment of hyperphagia associated PWS at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Meeting 2025, which is being held May 13-16, in Montreal, Canada."

Clinical data • Prader–Willi syndrome

Long-Term Results for Diazoxide Choline Extended-Release (DCCR) Tablets in Patients with Prader-Willi Syndrome: Developmental Behaviour Checklist 2 Response and Relationship to Hyperphagia Reductions (ESPE-ESE 2025) - "Behavioural improvements appeared to be independent of hyperphagia improvements. These hyperphagia and behavioural improvements should benefit patients with PWS and their families."

Clinical • Developmental Disorders • Metabolic Disorders • Mood Disorders • Prader–Willi syndrome • Psychiatry

LogiCare3PL Supports Commercial Launch of Soleno Therapeutics' VYKAT XR Following FDA Approval (PRNewswire) - "LogiCare3PL is proud to support the commercial launch of VYKAT XR, Soleno Therapeutics' newly FDA-approved therapy and the first and only treatment indicated for hyperphagia in patients with Prader-Willi syndrome (PWS). This milestone marks a significant advancement for the PWS community."

Launch US • Prader–Willi syndrome

Diazoxide choline (Vykat XR) for Prader-Willi syndrome-associated hyperphagia. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Prader–Willi syndrome

Soleno Therapeutics Announces VYKAT XR Launch (GlobeNewswire) - "Soleno Therapeutics, Inc...announced the U.S. commercial availability of VYKA XR (diazoxide choline) extended-release tablets, the company’s treatment for hyperphagia in patients four years of age and older with Prader-Willi syndrome (PWS), which was approved by the U.S. Food and Drug Administration (FDA) on March 26, 2025. Prescriptions of VYKAT XR have now been delivered to the first individuals living with PWS who have been prescribed the medication."

Launch US • Prader–Willi syndrome

PANTHERx Rare Selected by Soleno Therapeutics for the Distribution of VYKAT XR (PRNewswire) - "PANTHERx Rare...is pleased to announce that it was selected by Soleno Therapeutics as the specialty pharmacy for the distribution of VYKAT XR (diazoxide choline extended-release tablets), a once daily oral tablet indicated for the treatment of hyperphagia in individuals four years of age and older with Prader-Willi syndrome (PWS). VYKAT XR is the first and only FDA-approved treatment for hyperphagia in individuals with PWS."

Commercial • Genetic Disorders • Prader–Willi syndrome

Soleno Therapeutics Announces U.S. FDA Approval of VYKAT XR to Treat Hyperphagia in Prader-Willi Syndrome (GlobeNewswire) - "Soleno Therapeutics...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 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)....The primary safety analyses are based on Study 1 (Study C601) and the most common adverse reactions occurring in greater than or equal to 10% of individuals receiving VYKAT XR and at 2% greater than placebo included hypertrichosis, edema, hyperglycemia and rash."

FDA approval • Launch US • Genetic Disorders • Prader–Willi syndrome