If you agree with the information described in the community petition to FDA (below) and would like to see FDA review the New Drug Application for DCCR, please consider signing on to show your support. You can do so by completing the form on the next page.
Petition to request that the FDA file, grant priority review, and review the application for DCCR for people living with Prader-Willi syndrome (PWS).
We, the Prader Will syndrome (PWS) community, are at a point where our efforts to build a ‘research ready’ community, including the development of tools for drug development (such as our patient registry, natural history studies, cell and animal models, and clinical network), and investments in research are translating into a pipeline of new potential treatments for PWS. Diazoxide choline (DCCR) is an investigational drug being developed by Soleno Therapeutics, which has been shown to improve symptoms in people with PWS in well-controlled clinical trials. DCCR has the potential to improve hyperphagia, decrease fat mass, and improve challenging PWS-associated behaviors as rated both clinicians and caregivers.
Why are we sharing this, and why now? It is because we come to you with a call to action. The voice of the patient is critical to the drug development process. The 21st Century Cures Act requires drug developers to include, and FDA to consider, the patient perspective in making approval decisions for new drugs. The PWS community has been active in documenting the patient perspective and sharing the PWS patient voice with FDA over the last decade. Surveys of parents of those with PWS and individuals with PWS themselves have documented the profound impact of PWS on health and well-being, demonstrating the tremendous unmet need in this population. Treating PWS-associated symptoms including hyperphagia, anxiousness, and temper outbursts are the highest priority for the community. These symptoms make it so patients cannot live alone without significant supervision, cannot have jobs, and have limited friendships. Not surprisingly, there is high tolerance of risk for treatments that would reduce hyperphagia in PWS. These insights into PWS have been shared with FDA through on-site meetings with advocates, a Patient Listening Session (June 2021), and, most recently, through an externally-led Patient Focused Drug Development meeting (June 2023). This meeting showed that hyperphagia, anxiousness, temper outbursts, sleep disorders, obsessive-compulsive behaviors, and intellectual disability have tremendous negative impacts. We described to FDA that these symptoms greatly reduce the ability of the person with PWS to participate in community activities, gain independence, and reach their life goals. Further, we shared the reality that PWS is a life-threatening disease, whether from choking, accidents (e.g., running across a busy street to get food), stomach ruptures, or weight-related health events. This brings us to where we are today. With no approved treatments for any of these aspects of PWS, patients are left with no relief from hyperphagia or the other serious symptoms.