FPWR is dedicated to advancing research and accelerating the development of safe and effective treatments for people with Prader-Willi syndrome (PWS). While significant unmet needs remain, there are several therapies—both established and emerging—that can positively impact health and quality of life for individuals with PWS.
Below are five important treatments available for PWS that families, caregivers, and clinicians should know about and consider.
Please note that this is not an exhaustive list of every possible treatment. As always, the information FPWR provides is intended to serve as a starting point for an informed discussion with a qualified healthcare provider who is familiar with the person with PWS, allowing you to work together to determine the best treatments for that individual.
Growth Hormone (GH)
How does growth hormone help people with Prader-Willi syndrome?
Growth hormone (GH) therapy is considered the cornerstone of PWS medical management. It not only supports healthy growth in children, but also:
- Improves body composition by increasing lean muscle and reducing fat
- Increases strength and mobility, and bone health
- Benefits development, behavior and cognition
Importantly, GH is also beneficial for adults with PWS, helping maintain strength, energy levels, and body composition, even when started later in life.
Learn more about growth hormone therapy in PWS.
Sex Hormones
Why are sex hormones important in PWS treatment?
Hypogonadism (low sex hormone levels) is common in both males and females with PWS. Hormone replacement therapy with testosterone (males) or estrogen/progesterone (females):
- Supports healthy bone mineral density
- Improves body composition
- Contributes to emotional well-being and psychosocial health
Parents of children with PWS should begin asking their endocrinologist about hypogonadism and sex hormones around the normal time of puberty.
Learn more about hormone replacement therapy in PWS.
Guanfacine for Behavior
Can guanfacine help with behavior and anxiety in PWS?
Yes. Behavioral challenges—such as aggression, anxiety, irritability, and hyperactivity—are common in PWS. Guanfacine extended release (GXR), a medication originally approved for ADHD, was evaluated for people with PWS in a randomized, placebo-controlled clinical trial that showed that guanfacine can provide meaningful benefits:
- Reduced aggression compared to placebo
- Decreased skin-picking behaviors
- Lower levels of hyperactivity
Learn more about Guanfacine as a treatment for people with PWS.
Modafinil for Daytime Sleepiness
What treatments are available for sleep problems in PWS?
Daytime sleepiness is common in people with PWS and can impact daily functioning and quality of life. Modafinil (Provigil), is a wakefulness-promoting medication that has been used off-label with some success in individuals with PWS and is a good option to consider to improve sleepiness and overall functioning in PWS. Read our blog on modafinil here.
Trial Note: Pitolisant is currently being evaluated in a phase 3 clinical trial for people with PWS who struggle with daytime sleepiness. The drug pitolisant has also shown promise in improving wakefulness and reducing daytime sleepiness in PWS. This drug works through a different mechanism than modafinil, affecting the histamine H3 receptor. Harmony Biosciences is evaluating the safety and efficacy of the drug for PWS in a clinical trial that is currently enrolling. Learn more about the clinical trial opportunity.
Vykat™ XR for Hyperphagia
What is Vykat™ XR and how does it treat hyperphagia in PWS?
Vykat™ XR (diazoxide choline extended-release) is the first FDA-approved treatment specifically for hyperphagia in PWS. Hyperphagia—a constant, insatiable hunger—is one of the most challenging and life-limiting symptoms of PWS. In clinical trials, Vykat reduced hyperphagia-related behaviors, providing families with new hope and marking a historic milestone for PWS care.
Learn more about VYKAT for hyperphagia.
Clinical Trials in PWS
Clinical trials play a critical role in developing new treatments and community participation is essential to their completion! When trials fill quickly, researchers can gather data faster, accelerating the path to safe and effective therapies. Families and individuals interested in learning more or exploring participation options can find details on current PWS clinical trials here.
Moving Forward
While PWS remains a complex condition, progress in therapeutics is accelerating. Every study funded, every trial advanced, and every approval achieved brings us closer to a future where individuals with PWS can live longer, healthier, and more independent lives. At FPWR, we remain committed to advancing the research that makes this progress possible.
