NEW Approved Treatment for Prader-Willi syndrome

The FDA has recently approved Vykat XR (diazoxide choline) extended-release tablets for the treatment of hyperphagia, or persistent hunger, in individuals with Prader-Willi syndrome (PWS) aged 4 and older.  This is the first FDA-approved treatment for this diagnosis. It is administered via a once-daily, extended-release tablet . The drug is approved for individuals with PWS aged 4 years and older who experience hyperphagia.

Vykat XR works by targeting a specific pathway in the brain to help reduce hyperphagia. It does this by decreasing the secretion of a peptide that regulates appetite.  Vykat XR can cause issues with blood sugar. Before it is started, the person should have the following bloodwork completed: Fasting blood sugar and HbA1c.  During treatment the person should have regularly monitored fasting glucose and HbA1c.  Support staff need to monitor fasting glucose more frequently during the first few weeks of treatment in patients with risk factors for hyperglycemia.

Edema has also been identified as a side effect. Supports will want to watch for signs and symptoms.  Vykat ER has not been studied in patients with compromised cardiac reserve. It should be used with caution in these people.