US FDA approves first treatment for hyperphagia in Prader-Willi syndrome

The United States Food and Drug Administration (US FDA) has approved VYKAT™ XR, a significant milestone as the first approved treatment for hyperphagia in Prader-Willi syndrome (PWS). This breakthrough follows the US FDA's approval of Growth Hormone Treatment (GHT) nearly 25 years ago. The approval comes after an extensive US FDA evaluation, supported by data from clinical trials including a randomized withdrawal study.

IPWSO hopes the US FDA approval is the first step towards ensuring that all individuals with PWS who need access to these novel therapeutics will soon find them available, and accessible, in their own countries. As part of its global mission, IPWSO continues to advocate for equitable access to life-changing treatments, working with regulatory agencies, policymakers, and the rare disease community to remove barriers and accelerate availability worldwide.

Acknowledging the support of advocates, researchers, volunteers and families who refused to lose hope, IPWSO President, Anthony Holland, said,

"The approval of VYKAT™ XR (DCCR) for treating hyperphagia in people with PWS is a major and very welcome development. This is the first approved treatment for hyperphagia and the first new medical treatment for people with PWS for over 20 years. Congratulations to Soleno Therapeutics and our thanks to them for their work and determination. IPWSO will liaise with Soleno and work with national PWS Associations to highlight the urgent need for, and importance of, such treatments becoming available globally as soon as possible".

Thank you to Soleno Therapeutics for their dedication and commitment to developing VYKAT™ XR (DCCR) and getting approval from the US FDA. Thanks also to The Foundation for Prader-Willi Research, the Prader-Willi Syndrome Association | USA and IPWSO for their advocacy and support in making this happen, and to all the families who participated in trials to better the future for all with PWS. The news that this new treatment has been approved for use in the United States has taken the PWS world by storm and is celebrated far and wide by families with loved ones living with PWS, including in South Africa where I woke up to celebratory WhatsApp messages from parents. We do hope for and are looking forward to accessing this treatment for those living with PWS in South Africa and the rest of the world".

Karin Clarke, IPWSO Board Trustee and Chairperson for the PWS Support Group in South Africa