Breakthrough Clinical Results
Soleno Therapeutics announced that two presentations featuring data on VYKAT™ XR (diazoxide choline) extended-release tablets for the treatment of hyperphagia in Prader-Willi Syndrome (PWS) will be presented at ENDO 2025. One presentation will be an oral presentation focusing on glycemic outcomes over four years, and the other will be a poster presentation characterizing peripheral edema over 4.5 years. VYKAT XR, approved by the FDA on March 26, 2025, is a once-daily oral treatment for hyperphagia in adults and children aged 4 and older with PWS. The presentations highlight the long-term efficacy and safety data of VYKAT XR in managing hyperphagia and associated complications in PWS patients.
Key Highlights
- Two abstracts featuring data on VYKAT™ XR (diazoxide choline) extended-release tablets for Prader-Willi Syndrome (PWS) will be presented at ENDO 2025.
- One presentation will be an oral presentation on glycemic outcomes over four years, and the other a poster presentation on peripheral edema over 4.5 years.
- VYKAT™ XR was approved by the FDA on March 26, 2025, and is now commercially available.
- The presentations showcase long-term efficacy and safety data of VYKAT XR in managing hyperphagia in PWS patients.
Incidence and Prevalence
Global Epidemiology of Prader-Willi Syndrome
Incidence and Prevalence
Based on the available information, Prader-Willi syndrome (PWS) is a complex neuroendocrine disorder affecting approximately 1/15,000-1/30,000 people. This represents the only epidemiological estimate provided in the context.
The literature does not provide comprehensive global epidemiological metrics from recent systematic reviews or meta-analyses. While some specific prevalence data exists for conditions within PWS, such as epilepsy prevalence of 0.11 and febrile seizures prevalence of 0.09, there is no comprehensive global prevalence data available.
Most of the available literature focuses more on clinical features, treatment approaches, genetic mechanisms, and specific complications rather than global epidemiological metrics.
Epidemiological Patterns Across Populations
The available information does not contain specific data about how the epidemiological patterns of Prader-Willi syndrome vary across different geographical regions, ethnicities, and demographic groups.
While some studies mention specific patient populations, such as: - 96 individuals with PWS recruited from genetic centers and the PWS association - 8 patients (11.9%) with Prader-Willi syndrome among 67 patients with central apnea
These studies do not provide geographical, ethnic, or demographic comparisons that would allow for analysis of epidemiological patterns across different populations.
The literature contains information about clinical characteristics, diagnostic methods, genetic subtypes, and treatment approaches for PWS, but lacks comparative epidemiological data across different populations or regions.
Risk Factors and Comorbidities
Risk Factors and Comorbidities of Prader-Willi Syndrome
Genetic and Chromosomal Risk Factors
Prader-Willi syndrome (PWS) is a genetic disorder of imprinting with almost all cases occurring spontaneously. The syndrome is caused by the absence of expression of the paternally active genes in the PWS critical region on 15q11-q13. The primary genetic mechanisms include:
- In approximately 70% of cases, PWS results from a deletion of the 15q11-q13 region from the paternal chromosome 15
- In approximately 28% of cases, PWS is attributable to maternal uniparental disomy (UPD), which involves inheritance of 2 copies of chromosome 15 from the mother and no copies from the father
- In less than 2% of cases, PWS results from a mutation, deletion, or other defect in the imprinting center
PWS involves genomic imprinting, which is the epigenetic differential marking of maternally and paternally inherited alleles. This imprinting process implies the existence of a reversible imprinting signal that is erased in the gonads to be reset according to the sex of the individual. Importantly, mutations in imprinted genes are not inherited in a regular Mendelian fashion.
Medical Comorbidities and Secondary Conditions
Patients with Prader-Willi syndrome experience numerous medical comorbidities:
Metabolic and Endocrine Complications
- Non-insulin dependent diabetes mellitus (NIDDM) with a prevalence rate of 25% in adults, with mean age at onset of 20 years
- Type 2 diabetes mellitus (T2DM) with prevalence approximately 7-24%
- Growth hormone (GH) deficiency and other hormone deficiencies due to hypothalamic dysfunction
- Hypogonadism/hypogenitalism
- Metabolic syndrome
Musculoskeletal Issues
- High rates of fractures (29%)
- Severe scoliosis (15% in childhood)
- Small hands and feet
Respiratory and Sleep Disorders
- Recurrent respiratory infections reported in nearly 50% across age groups
- Sleep disorders (20%)
Other Physical Complications
- Severe infantile hypotonia
- Obesity and hyperphagia
- Short stature
- Leg ulceration (22% in adults)
- Abnormal body composition with increased body fat and deficit of lean body mass
- Dysmorphic facial features (almond-shaped eyes, narrow bifrontal diameter, thin upper lip)
- Strabismus
- Diabetic nephropathy (rare in childhood)
Neurological and Behavioral Issues
- Intellectual disabilities
- Behavioral/psychiatric problems
- Irritability and social dysfunction
Maternal Risk Factors
The context indicates that maternal disomy 15 in PWS may contribute to disturbances in gestational age and delivery. This is the only maternal factor mentioned in the provided information.
Drug used in other indications
Alternative Indications for VYKAT™ XR (Diazoxide Choline)
Based on the provided information, there is no evidence of VYKAT™ XR (diazoxide choline) being trialed for indications other than Prader-Willi syndrome. The available data only discusses diazoxide choline controlled-release tablets (DCCR) in relation to the treatment of Prader-Willi syndrome (PWS).
The studies mentioned in the context focus exclusively on DCCR's effects on PWS-related symptoms such as hyperphagia, aggressive behaviors, and body composition changes.
No information is available regarding: - Alternative therapeutic indications - Orphan disease targets beyond PWS - Intervention models for trials in other conditions - Specific dosing regimens for other conditions - Clinical trial designs for non-PWS applications
The research appears to be concentrated solely on the application of this medication for managing symptoms associated with Prader-Willi syndrome.