April 28, 2025
The first medicine for the treatment of genetically determined overeating
The American company Soleno Therapeutics has developed the first drug for the treatment of a rare hereditary disease, Prader-Willi syndrome (PWS), called Vykat XR (diazoxide choline), which recently received FDA approval.
The genetic syndrome that causes hunger, short stature, and other physical and behavioral disorders was discovered back in 1956 by Swiss scientists Andrea Prader and Heinrich Willi. The cause of this disease is a change in the work of the 15th chromosome, as a result of which there are a number of systemic disorders in the body that can manifest themselves even before birth (incorrect fetal position, low fetal mobility). However, one of the most serious manifestations of this disease is hyperphagia.
Pathology occurs in one of the 10 000 — 30 000 newborns with the same frequency among men and women. About 50 thousand Americans suffer from the disease. The diagnosis of PWS in developed countries is established at the age of 3-4 years.
Clinical studies have shown the effectiveness of Vykat XR in the treatment of hyperphagia. As part of the third phase of clinical trials, the drug has been studied for more than three years. The drug affects certain pathways in the brain, suppressing the production of a peptide that regulates appetite, which can help patients control their nutritional needs. At the same time, Vykat XR showed a positive safety profile. Hypertrichosis, edema, hyperglycemia, and rash were among the most common adverse reactions.
The drug is registered in the form of extended-release tablets and is indicated for adults and children over the age of 4 years.
The start of sales of the Vykat XR is scheduled for April 2025. The cost of the annual course will be 466 thousand dollars for this drug.
The approval received from the American regulator gives hope to patients with Prader–Willi disease to improve their quality of life.
- Pravoslavnaya O.V., Kantutis S.S., Shakirova A.A., Demyanova T.G., Sarkisyan E.A., Chugunova O.L., Cherkasova S.V., Levchenko L.A., Shumilov P.V. Modern features of managing children with Prader–Willi syndrome. Issues of children's dietetics. 2023; 21(4): 59–64. DOI: 10.20953/1727-5784-2023-4-59-64
- Butler M.G., Miller J.L., Forster J.L. Prader-Willi Syndrome — Clinical Genetics, Diagnosis and Treatment Approaches: An Update. Current Pediatric Reviews 2019;15(4):207-244.
