The US Food and Drug Administration has given the green light to Soleno Therapeutics’ medication for a rare genetic disorder, making it the first treatment available for patients who experience feelings of intense and persistent hunger.
The drug, to be called Vykat XR, treats hyperphagia, the hallmark symptom of the Prader-Willi syndrome, a genetic disorder caused by full or partial deletions on chromosome 15 that affect the regulation of gene expression, or how genes turn on and off.
Reuters reports that the debilitating condition, which affects about 50 000 people in the United States, affects everyday aspects of life, such as eating, behaviour and mood.
From this month, Soleno will be available in the US for patients aged four and over who have PWS with hyperphagia. The drug will have an annual average cost of $466 200 and will be dosed according to the patient’s weight.
The syndrome’s symptoms appear immediately, with infants born with low muscle tone and an inability to breastfeed. As they grow older, children develop hyperphagia, a feeling of insatiable hunger persisting even after meals. This could occur as early as age four but, on average, starts at about eight and then continues into adulthood.
The increased appetite can lead to rapid weight gain and, if not controlled, obesity and related problems like respiratory difficulties and heart disease. The company estimates the average lifespan of people with the disease is between 21 and 29 years.
The drug is a once-daily oral pill designed to target a specific pathway in the brain to help reduce hyperphagia by decreasing the secretion of a peptide known to regulate appetite.
Long-awaited approval
The long-awaited approval comes after the FDA’s extended review of the drug and mixed results from a late-stage trial on its efficacy. The approval was based on data from the study and a randomised withdrawal study.
Anthony Holland, president of the International Prader-Willi Syndrome Organisation, said the IPWSO will work closely with the company as well as other global health bodies to expand access to the drug worldwide. “One of the tasks of our organisation will be to campaign for its use more widely.”
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