On Tuesday, April 22, PWSA | USA and FPWR offered a PWS Community Town Hall for caregivers and guardians to hear more about people's lived experiences with VYKAT XR. Experiences were shared from individuals who participated in the phase 3 clinical trial of VYKAT XR, and attendees were able to ask questions of each other.
During the town hall, we heard from several parents who have experienced an overall positive impact of VYKAT XR in the Phase 3 clinical trial. Common benefits shared included improved behavior, fewer outbursts related to food, improved metabolism, and less worrying or talking about food. It was noted that it took time for these benefits to be observed; parents suggested that you shouldn’t expect immediate results.
Some side effects of the medication, which are consistent with the VYKAT XR label, were discussed by the trial participants, including high blood sugar levels (hyperglycemia), edema (excessive fluid accumulation in the body), and increased hair growth throughout the body. The general consensus was that hair growth was ‘not a big deal’, but some did use hair removal techniques to manage the growth. Those who experienced increased blood sugar levels shared that this could be a medically important concern for some individuals and suggested that glucose should be closely monitored when using VYKAT. Consistent with this, the VYKAT XR Medication Guide recommends checking blood sugar levels before you start VYKAT XR, at least 1 time every week for the first 2 weeks of treatment, then at least 1 time every 4 weeks and as needed during treatment.
Several townhall participants shared their experience with Panther One, the specialty pharmacy for VYKAT XR, has been positive, and several families have already received their first shipment of medication.
One of the most common questions raised during the town hall was: What percentage of the PWS community is likely to benefit from taking VYKAT XR? We are not yet able to provide a definitive answer. To help us track real-world outcomes, we will be adding a new survey to the Global PWS Registry. This will allow us to begin identifying trends over time. We encourage everyone who starts taking VYKAT XR to watch for this survey and complete it—your participation will be essential in helping the community better understand the impact of this treatment.
Soleno Therapeutics has published several resources on their website, vykatkr.com, for families who are interested in learning more. These include:
Additional data on safety and efficacy from the phase 3 clinical trial of VYKAT XR can be found in these peer-reviewed publications:
- Long-term efficacy and safety profile of Phase 3 / Open Label Extension: Diazoxide choline extended-release tablet in people with Prader-Willi syndrome: results from a long-term open-label study
- Phase 3 study results (prior to randomized withdrawal): Diazoxide Choline Extended-Release Tablet in People With Prader-Willi Syndrome: A Double-Blind, Placebo-Controlled Trial
- Comparison of Phase 3 Open Label Extension with PATH for PWS natural history study: Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study
VYKAT XR is FDA approved for the treatment of hyperphagia in individuals with PWS ages 4 years and older. Ultimately, the decision to try VYKAT XR is one that should be made in consultation with your loved one’s doctor. Your doctor will review the patient’s history, answer your questions, and advise if VYKAT XR may be right for you.
