- PMLiVE
The US Food and Drug Administration (FDA) has approved Soleno Therapeutics’ Vykat XR (diazoxide choline) extended-release tablets as the first treatment to address hyperphagia in patients with the rare neurodevelopmental disorder Prader-Willi syndrome (PWS).
Affecting an estimated one in every 15,000 newborns in the US, PWS is caused by an abnormality in the gene expression on chromosome 15.
The disorder results in a wide range of physical, mental and behavioural problems, but its defining symptom is hyperphagia, an intense and persistent sensation of hunger that can lead to longer term co-morbidities such as diabetes, obesity and cardiovascular disease.
Vykat XR, which is administered orally once daily, has been specifically authorised to treat hyperphagia in adults and paediatric PWS patients aged four years and older.
Among the evidence supporting the FDA’s decision on the drug was positive results from the 16-week withdrawal period of the phase 3 2-RWP study.
After receiving Vykat XR for a mean duration of 3.3 years, patients randomised to switch to placebo demonstrated a statistically significant worsening of hyperphagia compared to those who remained on Soleno’s drug.
Vykat XR also has a well-established safety profile with over four years of data across four double-blind and/or open label studies, the company outlined.
Soleno’s chief executive officer, Anish Bhatnagar, described the US approval as a “significant milestone for [the company] and, most importantly, for the PWS community who have had no options to treat the most disruptive aspect of this disease”.
Sharing a similar sentiment, Stacy Ward, chief executive officer of the Prader-Willi Syndrome Association USA, said: “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.
“Our families experience the constant and disruptive challenges of hyperphagia, and Vykat XR offers hope to so many.”
Soleno said it expects Vykat XR to be available in the US from April this year.