Prader-Willi syndrome is a leading cause of obesity in children. The rare disease was discovered over seventy years ago, yet this is the first therapy to be approved by the FDA.
Although there are many drivers causing obesity, in the case of Prader Willi, the primary driver is genetic.
A recent article in MedCity News explains that patients experience fierce hunger which cannot be satisfied causing families to lock pantries, refrigerators and other food storage areas.
Prader-Willi syndrome had been mentioned in medical books for almost seventy years. However, until Soleno Therapeutics of Redwood, California discovered the drug Vykat, all attempts to develop a treatment had failed.
The recently announced regulatory decision granted first approval to treat patients aged 4 or older for excessive hunger (hyperphagia).
The first paper on the disorder was published in 1956. The disease results from the absence of genes that regulate fullness and hunger. Patients face an increased risk of issues that affect metabolism and the heart (cardiometabolic).
Estimates of between 300,000 and 400,000 individuals worldwide have Prader Willi. According to Anish Bhatnagar, Soleno CEO, the earliest symptoms are evident about age 7.
About Vykat and its Competition
The main ingredient in Vykat is the 50-year-old drug diazoxide choline which is used today in the management of hyperinsulinism.
Currently, Teva Pharmaceutical is marketing its generic medicine known as Proglycem. In comparison, due to its extended-release, Soleno’s drug results in lower blood concentrations than Proglycem.
Soleno’s annual report stated that diazoxide choline is responsible for activation of potassium channels which are central to the regulation of several physiological processes.
It further states that these processes may increase appetite and other symptoms of Prader-Willi while targeting potassium channels establishes the potential to reduce food seeking and appetite.
Although Vykat failed the initial pivotal test, a second analysis provided statistical significance for the primary goal and all the secondary goals.
Vykat is scheduled to launch in April. The drug, with a list price of $5.92 per mg., is dosed in three strengths in accordance with a patient’s weight. Vykat’s label cautions against substitution with generic medications stating the different effects of pharmacokinetic profiles.
