The U.S. Food and Drug Administration (FDA) this week approved Vykat XR, developed by Soleno Therapeutics, as the first treatment for Prader-Willi Syndrome (PWS). This once-daily oral medication addresses hyperphagia, the insatiable hunger that is a hallmark of PWS.
PWS is a complex genetic disorder that affects approximately 50,000 individuals in the United States. It manifests from birth with symptoms such as low muscle tone and feeding difficulties, progressing to relentless hunger in early childhood. The persistent hunger often leads to rapid weight gain, obesity, and associated health complications, significantly impacting the quality and length of life.
The approval sent Soleno Therapeutics' shares up by 40% after the announcement. The San Francisco area company was founded in 2017, and is traded on Nasdaq. In an interview with Federal Newswire last year, the company’s founder, Dr. Anish Bhatnagar, said the company's mission is to develop novel therapeutics to treat rare diseases, with a particular focus on PWS.
"We are trying to … make a difference in the lives of these 10 to 20,000 families,” Dr. Bhatnagar told Federal Newswire in a March 2024 interview. He described challenges faced by families dealing with PWS, noting "these families live with locked kitchens, locked pantries, alarms, and motion activated cameras so these kids don't get access to food.”
The FDA approval allows the company to move closer to providing relief to PWS patients, and brings the company closer to a financial payoff for its considerable research investment. “We and many other companies like ours do a lot of the high-risk work ... treatments that have a low likelihood of success but can make a big difference,” Dr. Bhatnagar said in the 2024 interview.
He went on to say, “if at the end of the day a product is successful, if we can actually make a difference to these 10-20,000 lives, it’s all going to be worth it.”
The drug should be available for patients in April, according to the company.
