US FDA Approves First-Ever Treatment for Prader-Willi Syndrome

It is a rare disease treatment milestone that the US FDA has approved Soleno Therapeutics' medication Vykat XR as the first-ever approved treatment for Prader-Willi syndrome (PWS). The disorder, which is characterized by excessive hunger (hyperphagia), had previously not had any specific treatment options.

Key Highlights:

First Therapy for Hyperphagia in PWS

• Vykat XR addresses hyperphagia, the characteristic symptom of Prader-Willi syndrome.

• The daily oral pill suppresses appetite through the modulation of a brain peptide that plays a role in hunger signaling.

• Vykat XR will be launched in the US from April 2025 for patients aged 4 years and older.

A Rare Neurogenetic Disorder with Devastating Consequences

• PWS affects around 50,000 individuals in the US, leading to extreme hunger, behavioral abnormalities, and developmental delays.

• Untreated, hyperphagia can result in extreme obesity, cardiovascular disease, and shortened life expectancy (21–29 years on average).

• Symptoms may start at an early age, usually getting worse by age 8.

Pricing and Access

• Treatment cost per year: $466,200, dosed based on patient weight.

• Soleno Therapeutics aims for global expansion with the help of international health organizations.

Trial Results and FDA Approval

• In a 127-patient study, Vykat XR had benefit in severe patients, though it did not beat placebo in the overall group.

• Approval was based on both this trial and a randomized withdrawal study, which showed long-term benefit.

• Follow-up results after one year demonstrated significant reduction in hyperphagia.

Global Reaction:

• Patients and families have welcomed the news as a long-awaited breakthrough.

• Justice Faith, a 21-year-old patient, shared: “My experience with hyperphagia, like everyone with PWS, is different from others… I’m very excited a treatment may be available.”

Statements from Leaders or Officials
"Think about being hungrier than you've ever been. That's what I am constantly like. I never ever get that satisfied feeling from food."
— Quote by patient and contributed by Dr. Jennifer Miller, Pediatric Endocrinologist, University of Florida"We will closely cooperate with the firm and worldwide health organizations in a push to bring the medication globally."
— Anthony Holland, President, International Prader-Willi Syndrome Organisation (IPWSO)The FDA approval of Vykat XR is a landmark step in treating a debilitating and life-shortening illness. India's similar issues of access to rare diseases mean that this approval may set off global debates around affordability and access, particularly for emerging nations. Stakeholders now turn their attention to rolling out this innovation globally beyond the US, making hope turn into assistance for patients globally.