Hyperphagia treatment Vykat XR for PWS now available in US
Soleno: First eligible patients have received prescriptions for therapy
The first eligible Prader-Willi syndrome (PWS) patients in the U.S. have received their prescriptions for Vykat XR (diazoxide choline) — the once-daily, extended-release tablets now approved as a treatment for hyperphagia, or insatiable hunger, in people with PWS, ages 4 and older.
Soleno Therapeutics’ Vykat XR became available on the market less than a month after it was approved in March by the U.S. Food and Drug Administration (FDA), according to a company press release. The treatment is the first to be approved for hyperphagia, a hallmark PWS symptom characterized by persistent thoughts about food and excessive hunger.
“We are extremely pleased to begin delivering Vykat XR, the only FDA-approved treatment for hyperphagia, to individuals living with PWS,” said Anish Bhatnagar, MD, Soleno’s CEO, adding that, “since FDA approval, we have seen strong interest in Vykat XR.”
Extended-release PWS treatment designed to ease hyperphagia in patients
The developer is offering a support program called Soleno ONE to help patients, caregivers, and doctors access Vykat XR. The toll-free number for those interested is 833-SOLENO-1 — or 833-765-3661. Doctors who want to prescribe the medication can download and fill out the start form.
“Our team is fully prepared to meet demand and is well-positioned to execute a successful commercial launch of this first-to-market treatment,” Bhatnagar said. “These first shipments mark an important step in our efforts to deliver this new treatment to the PWS community and are an exciting milestone reflecting our dedication to ensuring timely access to treatment.”
In infancy, symptoms of PWS include weak muscle tone, slow growth, and failure to thrive. Hyperphagia usually begins in early childhood, often leading to excessive weight gain and even obesity at a young age. The condition is known to trigger challenging behaviors, such as getting angry and engaging in risky actions to obtain food, or becoming frustrated when food isn’t available.
In addition, as “the hallmark condition of PWS,” hyperphagia “is the leading cause of mortality in the PWS population and creates a significant burden for caregivers,” Bhatnagar said.
These first shipments mark an important step in our efforts to deliver this new treatment to the PWS community and are an exciting milestone reflecting our dedication to ensuring timely access to treatment.
Vykat XR is designed to activate ATP-sensitive potassium channels — gate-like proteins that open and close based on the amount of glucose, or blood sugar, present in the bloodstream. By keeping these channels open, Vykat XR reduces the release of appetite-signaling molecules, which is expected to ease hyperphagia.
In the pancreas, the therapy’s effects suppress the release of insulin, a hormone that helps move glucose from the bloodstream into cells. With less insulin, more glucose stays in the bloodstream, increasing the feeling of satiety and helping prevent excess fat from building up in tissues.
Taken once daily with or without food, Vykat XR is available as extended-release oral tablets that come in three dose strengths: 25, 75, and 150 mg. The starting dose, which is increased every two weeks for a total of six weeks to reach the target maintenance dose, depends on each patient’s body weight.
The therapy’s approval in the U.S. was based on data from DESTINY PWS (NCT03440814) and its open-label extension (NCT03714373).
In DESTINY PWS, a Phase 3 clinical study involving 127 patients ages 4 and older, Vykat XR did not significantly outperform a placebo in reducing hyperphagia. However, most patients chose to continue treatment in the extension study, where one year of Vykat XR led to reduced hyperphagia and fewer challenging behaviors, per study results.
In a four-month withdrawal period that followed the extension study, patients who switched from Vykat XR to a placebo experienced worsening hyperphagia compared with those who remained on treatment.
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