Beyond the Symptom: Lessons from the United in Hope PWS Conference
Sugata Biswas, BA, MA, MBA
July 2025
At the recent United in Hope: International Prader-Willi Syndrome Conference, scientific posters showcased the latest research and lived experiences in this rare disease. Some explored genetic pathways and neuronal development. Others detailed caregiver burdens, behavioral challenges, and health system gaps. Yet amid this diversity, hyperphagia (an unrelenting hunger that resists satiety) dominated the conversation.
From new scales to assess food-related behaviors to updates on emerging treatments, the focus on hyperphagia was clear. And for good reason: for most, it is the relentless hunger that defines daily life with PWS, shaping not only physical health but family routines, psychological well-being, and safety itself.
There is genuine excitement in the air with good reason. The biotechnology company, Soleno Therapeutics, recently launched Vykat XR. This is the first FDA approved therapy to specifically target hyperphagia in PWS. In addition, two other products are in the pipeline:
- ACP-101 (Acadia Pharmaceuticals): a carbetocin nasal spray in its pivotal Phase 3 COMPASS PWS trial. Unlike traditional systemic treatments, ACP-101 is an intranasal formulation designed to deliver carbetocin directly to the brain, targeting the oxytocin deficiency believed to underlie hyperphagia in PWS.
- ARD-101 (Aardvark Therapeutics): a novel therapy engaging the CCK pathway, now entering Phase III trials.
It is an exciting moment to be sure but reviewing the posters revealed a deeper truth: treating a single symptom, no matter how impactful, is still one piece of a complex puzzle that impacts the daily quality of life of patients and their families.
In the case of hyperphagia, the reality is more demanding. Years of managing hyperphagia leave a mark beyond physical hunger. Families implement locks, alarms, and rigid routines to keep their loved ones safe, and these behavioral adaptations do not disappear overnight, even with effective treatment. Helping families navigate these transitions requires more than prescribing a drug; it requires psychological support and careful planning.
Moreover, hyperphagia is rarely experienced in isolation. It is deeply intertwined with anxiety, obsessive-compulsive behaviors, and distress. These comorbidities persist regardless of hunger improvements. Treating hyperphagia does not erase mental health challenges.
Caregiver expectations also complicate the path to real-world impact. For many, a new treatment brings hope of a “cure.” Yet posters addressing caregiver burden reminded us that while symptoms may improve, the emotional, logistical, and financial stresses of care often remain. Effective treatment is only part of the support families need.
“The hunger is a big part of our lives but it’s not all about that. Even if the hunger eases, I still worry every day about meltdowns, his anxiety… what happens if we can’t keep it all under control.”
— PWS Caregiver
And finally, the health system itself must be ready. Access to treatments depends not only on FDA approval but on payer coverage, clinician adoption, and integration with behavioral health and support programs. Without these elements in place, the promise of new therapies risks falling short of their transformative potential.
This dynamic is not unique to PWS. In Rett Syndrome, improving motor symptoms does not eliminate communication barriers. In Spinal Muscular Atrophy (SMA), gene therapies have changed survival trajectories, but daily physical therapy and adaptive care remain critical for meaningful function. In Angelman Syndrome, controlling seizures is essential, but cognitive and behavioral challenges continue to shape everyday life. Across rare diseases, treating one measurable symptom is often only half the battle.
Real progress requires addressing the lived experiences behind the data. It means combining pharmacologic breakthroughs with behavioral interventions, caregiver education, and holistic care models that reflect the complexity of these conditions. It means asking deeper questions: What else needs to change for patients and families to thrive?
For manufacturers preparing to launch therapies targeting specific symptoms, especially in rare diseases, one takeaway is clear: plan beyond the symptom. Consider building integrated launch strategies that combine pharmacologic data with behavioral health resources, caregiver education, and stakeholder alignment. Engage caregivers and patients early to understand how symptom improvement will affect daily routines, mental health, and expectations. Anticipate what additional support systems, whether behavioral interventions, provider training, or policy changes, will be needed to translate clinical efficacy into real-world impact.
At Cadence, we’ve worked across numerous rare diseases, partnering with healthcare professionals, patients, and caregivers to understand what truly matters. Whether it’s supporting medical meetings and advisory boards, conducting deep primary market research, or building tailored respondent panels, we help teams see beyond the symptom to the lived experience.
Because in rare diseases, that’s where the real impact, and the real insight, begins.
If you’re looking to better understand your stakeholders, uncover what drives decision-making, or engage the right experts to guide your strategy, we’d love to explore how Cadence can support your next initiative.
