The Prader-Willi Syndrome (PWS) market, projected to be valued at USD 0.98 Billion in 2025, is anticipated to grow steadily, reaching USD 1.59 Billion by 2032 at a CAGR of 7.2% during the forecast period. This growth is fueled by increasing awareness and early diagnosis of PWS, coupled with rising investment in rare disease research. Advancements in targeted therapies and supportive care treatments are also contributing to improved patient outcomes, further driving the market growth. Moreover, the expansion of patient registries and advocacy initiatives is facilitating clinical trials and regulatory approvals. The growing focus on novel gene and hormone-based therapies, along with supportive reimbursement policies in developed markets, is expected to enhance the market growth over the coming years.

Market Takeaways

• By Growth Hormone Therapy, GENOTROPIN (Pfizer) is expected to lead with a 38.5% share in 2025, owing to its established clinical efficacy and widespread use in treating growth hormone deficiency, which is highly prevalent among Prader-Willi syndrome patients.
• By Hyperphagia Treatment, DCCR (Soleno Therapeutics) is expected to lead with a 45.6% share in 2025, owing to its effectiveness as an oral, once-daily therapy targeting chronic hyperphagia and related behavioral symptoms in PWS patients.
• By Behavioral and Sleep Management, WAKIX (Harmony Biosciences) is a key therapeutic option addressing excessive daytime sleepiness and behavioral issues in PWS patients, contributing to improved patient quality of life.
• Regionally, North America is anticipated to lead the global Prader-Willi syndrome (PWS) market in 2025, driven by early diagnosis, higher treatment adoption rates, robust healthcare infrastructure, and the presence of leading pharmaceutical companies focused on rare diseases.

Prader-Willi Syndrome (PWS) Market Report Coverage

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Market Dynamics

The Prader-Willi Syndrome (PWS) market is experiencing steady growth, driven by increased awareness, early diagnosis, and rising prevalence of genetic disorders requiring long-term management. Growth hormone therapy remains a cornerstone of treatment, addressing the common issue of growth hormone deficiency among PWS patients, with established products like GENOTROPIN (Pfizer) maintaining strong market presence. The need for chronic management of hyperphagia and behavioral symptoms is accelerating the adoption of innovative therapies such as DCCR (Soleno Therapeutics), which targets appetite regulation and associated behaviors.

Advancements in clinical research, particularly in the areas of gene therapy, hormonal treatments, and neuropeptide regulation, are fostering the development of more targeted and effective therapies. Additionally, the growing support from patient advocacy groups and rare disease initiatives is improving clinical trial enrollment and access to treatment in both developed and emerging markets.

In April 2024, Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, announced that the U.S. Food and Drug Administration (FDA) had granted Breakthrough Therapy Designation to diazoxide choline for the treatment of adults and children ages 4 years and older with genetically confirmed Prader-Willi syndrome (PWS) who have hyperphagia. The designation reflects the Agency’s determination that based on an assessment of the preliminary data from the Phase 3 clinical development program, diazoxide choline may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies.

Furthermore, the introduction of therapies such as WAKIX (Harmony Biosciences) for behavioral and sleep-related issues demonstrates the market's evolution toward comprehensive, multidisciplinary treatment approaches. Regional market growth, especially in North America and Europe, is supported by strong healthcare infrastructure, reimbursement coverage, and regulatory incentives for orphan drugs. These dynamics, combined with ongoing innovation and supportive policy frameworks, are expected to propel the Prader-Willi syndrome (PWS) market over the forecast period.

Market Trends

• Integration of Multidisciplinary Treatment Approaches

The evolving understanding of Prader-Willi syndrome as a multisystem disorder is driving demand for holistic treatment models that integrate hormonal, behavioral, metabolic, and sleep management strategies. Drug combinations and adjunct therapies are gaining traction as part of comprehensive care protocols. WAKIX (pitolisant), approved for narcolepsy, is being increasingly explored for use in PWS patients with excessive daytime sleepiness, suggesting a trend toward the repurposing of neurologically active agents to address behavioral and cognitive symptoms in syndromic conditions.

In May 2023, Harmony Biosciences presented safety and efficacy data for a late‑breaking Phase 2 proof‑of‑concept study evaluating pitolisant (WAKIX) to treat excessive daytime sleepiness (EDS) in PWS at the APSS “SLEEP 2023” conference—marking the first formal data assessing pitolisant in PWS patients.

• Growing Investment in Rare Disease Research and Advocacy

Rising government and private sector funding for rare disease research is stimulating innovation and improving diagnosis and patient access to emerging therapies. Advocacy groups are playing a critical role in trial recruitment, public awareness, and patient support, significantly influencing regulatory and commercial strategies.

In May 2022, Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO) announced that Running for Research - Prader-Willi Syndrome (“RFR”), an organization committed to raising private donations in support of advancing the science around Prader-Willi syndrome (“PWS”), will fund an investigator-sponsored clinical study to evaluate Diazoxide Choline Extended-Release tablets (“DCCR”) in patients with early phase PWS.

March Opportunities

• Emergence of Early-Phase Interventions to Delay Hyperphagia Onset

Recent research is shifting focus toward early-stage treatment in younger PWS patients to delay or prevent the onset of hyperphagia, rather than managing it after behavioral symptoms emerge. This approach represents a significant opportunity for developers to capture untapped pediatric segments with high unmet need.

In March 2022, the patient advocacy group Running for Research – Prader‑Willi Syndrome funded an investigator‑sponsored multi-center clinical study to evaluate DCCR (diazoxide choline extended‑release tablets) in pre-hyperphagic, early-phase PWS individuals. The goal is to assess whether DCCR can delay or prevent progression into the hyperphagia phase by intervening before behavioral symptoms emerge.

• Expansion into Behavioral and Cognitive Therapy Segments

The neurological and behavioral aspects of PWS—such as anxiety, rigidity, and obsessive behaviors—are under-addressed by current treatments, representing a key area for therapeutic expansion. Drug repurposing and new CNS-targeted agents can provide behavioral relief and cognitive stabilization.

In October 2023, Harmony Biosciences Holdings, Inc. announced the presentation of new secondary endpoint data, including improvements in behavioral disturbances, from its Phase 2 signal-detection study evaluating pitolisant for the treatment of excessive daytime sleepiness (EDS) in Prader-Willi syndrome (PWS) at the 2023 Foundation for Prader-Willi Research (FPWR) Symposium and Family Conference. The company also announced that it anticipates initiating its Phase 3 registrational TEMPO study in Q4 2023.

Analyst View

• The Prader-Willi Syndrome (PWS) market is undergoing a paradigm shift, driven by the emergence of targeted, disease-modifying therapies such as VYKAT XR (diazoxide choline) and increased recognition of PWS as a complex, multisystem disorder requiring integrated care. The FDA’s approval of VYKAT XR in March 2025 validates a long-standing clinical need in hyperphagia treatment, significantly boosting the competitive intensity in this segment and opening up new revenue streams for rare disease-focused biopharma players.
• Growth hormone therapy continues to anchor the market, led by Pfizer’s GENOTROPIN, but its dominance is gradually being challenged by new entrants in the behavioral and sleep management space. Therapies like pitolisant (WAKIX) are paving the way for a broader therapeutic toolkit, especially for managing cognitive, sleep, and behavioral challenges that are increasingly prioritized by clinicians and caregivers.
• The growing momentum around early-phase intervention strategies—especially in pediatric segments before the onset of hyperphagia—presents a significant opportunity for market expansion. Investigator-sponsored trials backed by advocacy organizations such as Running for Research – PWS are accelerating innovation pipelines in this space, with potential to redefine standard-of-care approaches.
• Investment in rare disease R&D and favorable regulatory designations (Breakthrough, Orphan Drug) are streamlining commercialization timelines. The North American and European markets are expected to remain dominant due to supportive reimbursement ecosystems, higher diagnostic rates, and active patient advocacy infrastructure. Meanwhile, emerging markets offer untapped potential as awareness and access to specialist care improve.
• Over the forecast period, multidisciplinary treatment models incorporating hormone, neurobehavioral, and metabolic therapies will become the standard, with drug repurposing and CNS-targeted agents poised to address long-overlooked behavioral symptoms. Pharma companies entering the PWS landscape are advised to adopt patient-centric commercialization strategies, engage with advocacy stakeholders early, and explore combination or adjunctive therapy approaches to enhance therapeutic value.

Key Developments

• In May 2025, Soleno Therapeutics, Inc. announced that Soleno’s Marketing Authorization Application (MAA) seeking regulatory approval of Diazoxide Choline Prolonged-Release Tablets (previously referred to as DCCR) for the treatment of adults and children four years and older with Prader-Willi syndrome (PWS) who have hyperphagia had been validated by the European Medicines Agency (EMA).
• In July 2023, Harmony Biosciences Holdings, Inc., a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, has successfully completed an End-of-Phase 2 meeting with FDA regarding its clinical development plan evaluating pitolisant as a potential treatment for excessive daytime sleepiness (EDS) in patients ages six and older with Prader-Willi syndrome (PWS). Harmony plans to initiate a Phase 3 registrational study in the fourth quarter of 2023.

Competitive Landscape

• Soleno Therapeutics
• Harmony Biosciences
• Pfizer
• Novo Nordisk
• Sandoz
• ACADIA Pharmaceuticals
• Aardvark Therapeutics
• Gedeon Richter
• Palobiofarma
• ConSynance Therapeutics

Prader-Willi Syndrome (PWS) Market Segmentation

• By Growth Hormone Therapy
• GENOTROPIN (Pfizer)
• NORDITROPIN (Novo Nordisk)
• OMNITROPE (Sandoz)
• By Hyperphagia Treatment
• DCCR (Soleno Therapeutics)
• Carbetocin (ACADIA Pharmaceuticals)
• ARD-101
• By Behavioral and Sleep Management
• WAKIX (Harmony Biosciences)

Regional Insights

• North America
  • U.S.
  • Canada
• Latin America
  • Mexico
  • Brazil
  • Argentina
  • Rest of Latin America
• Europe
  • Italy
  • Germany
  • U.K.
  • Spain
  • France
  • Russia
  • Rest of Europe
• Asia Pacific
  • South Korea
  • India
  • Japan
  • China
  • Australia
  • ASEAN
  • Rest of Asia Pacific
• Middle East
  • GCC Countries
  • Israel
  • Rest of Middle East
• Africa
  • South Africa
  • North Africa
  • Central Africa