Starting in 2024, I began serving on the Board of Directors for the Foundation for Prader-Willi Research (“FPWR”). Why you ask? Schaaf-Yang Syndrome (“SYS”) is an incredibly rare disease that was only discovered in 2013 and doesn’t have its own research foundation. Also, SYS is defined by a mutation of the MAGEL2 gene, which is in the “Prader-Willi Region” of Chromosome 15. As a result, Prader-Willi Syndrome (“PWS”) and SYS research are closely aligned and benefit from each other.
Earlier this month, there was a breakthrough drug that received FDA approval to treat the hallmark sign of PWS, hyperphagia (“excessive hunger”). This is a milestone moment for PWS and SYS research. This drug was developed because of research funded by PWS over 10 years ago in 2014! This is proof that your donations are working and progress is being made. The results from the clinical trials for this drug are amazing and will make the lives of those living with PWS and SYS so much improved.
Help celebrate with us the progress that is being made!
Cheers!
Matt
FPWR Blog Post: https://www.fpwr.org/blog/dccr-approved-for-treating-hyperphagia-in-prader-willi-syndrome
We are thrilled to share that the FDA has approved the first treatment for hyperphagia (excessive hunger) in individuals with Prader-Willi syndrome (PWS). VYKAT XR (previously referred to as DCCR) is now approved for adults and children 4 years of age and older with Prader-Willi syndrome. Soleno Therapeutics expects VYKAT XR to be available in the U.S. beginning in April 2025. This groundbreaking achievement marks a pivotal moment in the journey to provide life-changing therapies for those affected by PWS, and we are proud to have been a part of it.
In 2014, your donations made it possible for FPWR to make a critical investment in the Phase 2 study of DCCR, which helped lay the foundation for its success. We want to extend our deepest gratitude to our generous donors who made this pivotal moment possible. Your support was crucial in driving this research forward and advancing us to this exciting milestone.
We would also like to thank the families who participated in the PATH for PWS study within the Global PWS Registry. The data you provided was essential in showing that individuals with PWS who had not taken DCCR had higher hyperphagia scores compared to those who were part of the Phase 3 study and received DCCR. This critical insight supported Soleno’s conversations with the FDA, helping to pave the way for this approval.
A special thank you to all of the families who participated in the Phase 2 and Phase 3 clinical trials of DCCR. Without your commitment to participate in these clinical trials for PWS, our community would not be able to celebrate this moment.
Finally, our deep gratitude to the team at Soleno Therapeutics for their dedication and perseverance in developing DCCR as a treatment for hyperphagia in PWS. Their commitment to advancing this therapy has been instrumental in reaching this remarkable milestone, and we are deeply grateful for their partnership in improving the lives of those with PWS.