DelveInsight’s “Prader–Willi Syndrome Market Insights, Epidemiology, and Market Forecast – 2034” report delivers an in-depth understanding of PWS, historical and forecasted epidemiology as well as the PWS market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

 

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Key Takeaways from the Prader-Willi Syndrome Market Report

• In November 2024:- Harmony Biosciences LLC- This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome. The primary objective of this study is to evaluate the efficacy of pitolisant in treating excessive daytime sleepiness (EDS) in patients ≥6 years of age with Prader-Willi syndrome.
• In November 2024:- ACADIA Pharmaceuticals- A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel-group Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome. This is a 12-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group study comparing carbetocin nasal spray 3.2 mg TID with placebo (matched placebo nasal spray TID) in subjects with PWS.
• Among the 7MM, the US accounted for the highest prevalent cases of PWS in 2023, with around 22,600 cases; these cases are expected to increase during the forecast period.
• Amongst EU4 and the UK, the total prevalent cases of PWS were highest in Germany, while the lowest number of cases were in Spain in 2023.
• According to the estimates, in Japan, it is observed that PWS was most prevalent in the 18-40 years age group, accounting for over 38% of total cases in 2023.
• In 2023, among genetic subtype-specific cases of PWS in the US, the paternal microdeletion subtype accounted for the highest proportion, approximately 70% of cases, while the translocation subtype was the least common.
• The leading Prader-Willi Syndrome Companies such as Pfizer, Soleno Therapeutics, Neuren Pharmaceuticals, Harmony Biosciences, Gedeon Richter Plc, Montefiore Medical Center, Aardvark Therapeutics, ACADIA Pharmaceuticals, and others
• Promising Prader-Willi Syndrome Therapies such as GENOTROPIN (somatropin), DCCR, NNZ-2591, Pitolisant tablet, RGH-706, ARD-101, Carbetocin, and others

 

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Prader-Willi Syndrome Epidemiology Segmentation in the 7MM

• Total Prevalence of Prader-Willi Syndrome
• Prevalent Cases of Prader-Willi Syndrome by severity
• Gender-specific Prevalence of Prader-Willi Syndrome
• Diagnosed Cases of Episodic and Chronic Prader-Willi Syndrome

 

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Prader-Willi Syndrome Marketed Drugs

• NORDITROPIN: Novo Nordisk

NORDITROPIN (somatropin) is a recombinant human growth hormone and is developed by Novo Nordisk. NORDITROPIN is a polypeptide hormone of recombinant DNA origin. The hormone is synthesized by a special strain of E. coli bacteria that has been modified by the addition of a plasmid that carries the gene for human growth hormone. NORDITROPIN contains the identical sequence of 191 amino acids constituting the naturally occurring pituitary human growth hormone with a molecular weight of about 22,000 Daltons. In February 2018, the US FDA approved NORDITROPIN for the treatment of growth failure due to PWS.

 

Prader-Willi Syndrome Emerging Drugs

• WAKIX (pitolisant): Harmony Biosciences

Pitolisant is a selective histamine 3 (H3) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H3 receptors, thereby increasing the synthesis and release of histamine, a wake-promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture, and commercialize pitolisant in the United States. WAKIX is FDA-approved to treat EDS or cataplexy in adult patients with narcolepsy. Pitolisant is not approved for use in patients with PWS and is currently being evaluated in PWS. The US FDA granted Orphan Drug designation to pitolisant for the treatment of PWS in February 2024. Harmony Biosciences is also developing HBS-102 for PWS.

 

• Diazoxide Choline Controlled-Release (DCCR): Soleno Therapeutics

DCCR is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide, and is administered once daily. The parent molecule, diazoxide, has been used for decades in thousands of individuals in a few rare diseases in neonates, infants, children, and adults but is not approved for use in PWS. Soleno conceived of and established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline, and DCCR in individuals with PWS. The US FDA has granted breakthrough therapy designation to diazoxide choline for the treatment of adults and children ages 4 years and older with genetically confirmed PWS who have hyperphagia in April 2024.

 

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Prader–Willi Syndrome Market Outlook

Current treatment options for PWS are limited, with management primarily focusing on lifestyle modifications to prevent obesity-related deaths. Nearly half of the deaths in PWS patients under 18 are linked to food-seeking behaviors such as choking and accidents. The clinical management of PWS spans many therapeutic domains, including nutritional, developmental, educational, hormonal, and behavioral support, with each stage of development requiring unique management strategies. Growth hormone treatment has been shown to increase growth velocity and height, improve body composition, and, with proper dietary management, prevent obesity. It also enhances physical and respiratory performance, thereby improving quality of life and potentially preventing long-term cardiovascular and metabolic issues such as hypercholesterolemia and diabetes.

 

Prader-Willi Syndrome Therapies and Companies

• GENOTROPIN (somatropin): Pfizer
• DCCR: Soleno Therapeutics
• NNZ-2591: Neuren Pharmaceuticals
• Pitolisant tablet: Harmony Biosciences
• RGH-706: Gedeon Richter Plc
• ARD-101: Aardvark Therapeutics
• Carbetocin: ACADIA Pharmaceuticals

 

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Scope of the Prader-Willi Syndrome Market Report

• Study Period: 2020-2034
• Coverage: 7MM
• Key Prader-Willi Syndrome Companies: Pfizer, Soleno Therapeutics, Neuren Pharmaceuticals, Harmony Biosciences, Gedeon Richter Plc, Montefiore Medical Center, Aardvark Therapeutics, ACADIA Pharmaceuticals, and others
• Key Prader-Willi Syndrome Therapies: GENOTROPIN (somatropin), DCCR, NNZ-2591, Pitolisant tablet, RGH-706, ARD-101, Carbetocin, and others
• Prader-Willi Syndrome Therapeutic Assessment: Prader-Willi Syndrome current marketed and Prader-Willi Syndrome emerging therapies
• Prader-Willi Syndrome Market Dynamics: Prader-Willi Syndrome market drivers and Prader-Willi Syndrome market barriers

 

Table of Contents

1. Prader-Willi Syndrome Market Report Introduction

2. Executive Summary for Prader-Willi Syndrome

3. SWOT analysis of Prader-Willi Syndrome

4. Prader-Willi Syndrome Patient Share (%) Overview at a Glance

5. Prader-Willi Syndrome Market Overview at a Glance

6. Prader-Willi Syndrome Disease Background and Overview

7. Prader-Willi Syndrome Epidemiology and Patient Population

8. Country-Specific Patient Population of Prader-Willi Syndrome

9. Prader-Willi Syndrome Current Treatment and Medical Practices

10. Prader-Willi Syndrome Unmet Needs

11. Prader-Willi Syndrome Emerging Therapies

12. Prader-Willi Syndrome Market Outlook

13. Country-Wise Prader-Willi Syndrome Market Analysis (2020-2034)

14. Prader-Willi Syndrome Market Access and Reimbursement of Therapies

15. Prader-Willi Syndrome Market Drivers

16. Prader-Willi Syndrome Market Barriers

17. Prader-Willi Syndrome Appendix

18. Prader-Willi Syndrome Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

 

About Us

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