Prader-Willi Syndrome Market

Market Overview:

The 7 major Prader-Willi syndrome markets are expected to exhibit a CAGR of 3.65% during 2024-2034.

The Prader-Willi syndrome market has been comprehensively analyzed in IMARC's new report titled "Prader-Willi Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Prader-Willi syndrome refers to a rare genetic disorder that occurs due to the loss of specific genes on chromosome 15, which are typically inherited from the father. This condition is characterized by a distinctive pattern of symptoms, such as a chronic feeling of hunger, obesity, intellectual disabilities, behavioral problems, etc. Numerous other common indications of the disease include underdeveloped sex organs, poor growth and physical development, stubbornness, short stature, cognitive impairment, delayed motor development, speech problems, temper tantrums, reduced hormone production, lazy eye, etc. Individuals suffering from Prader-Willi syndrome may also experience psychiatric conditions like anxiety and mood disorders. The diagnosis of this ailment is mainly based on evaluating the patient's reported symptoms, medical history, and a generic blood test. The healthcare provider may also perform DNA-based methylation testing to identify abnormalities or changes in the chromosome that indicate the syndrome. Additionally, various diagnostic procedures, such as fluorescence in-situ hybridization and chromosomal microarray analysis, are utilized to rule out other possible causes of the underlying symptoms.

The rising cases of genetic disorders on account of an error in one or more genes in a particular region of chromosome 15 are primarily driving the Prader-Willi syndrome market. Furthermore, the widespread adoption of growth hormone therapy, which promotes development, increases muscle mass, reduces body fat, etc., for managing the growth hormone deficiency associated with the ailment is also propelling the market growth. In addition to this, the inflating utilization of behavioral and psychiatric interventions for addressing a patient's emotional challenges and boosting social skills is further acting as another significant growth-inducing factor. Moreover, the escalating demand for lifestyle modifications, such as the consumption of a well-balanced, calorie-restricted diet, on account of its several associated benefits, including maintaining a healthy weight and minimizing the likelihood of other complications related to the ailment, is also creating a positive outlook for the market. Apart from this, the emerging popularity of intranasal selective oxytocin receptors, since they can aid in appetite regulation and improve social functioning, reduce repetitive behaviors, as well as address hyperphagia in patients, is expected to drive the Prader-Willi syndrome market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Prader-Willi syndrome market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for Prader-Willi syndrome and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Prader-Willi syndrome market in any manner.

Recent Developments:

• In July 2024, ConSynance Therapeutics announced that its investigational medication CSTI-500 had been given Rare Pediatric Disease Designation (RPDD) by the US FDA for the treatment of Prader-Willi Syndrome in children and adolescents.
• In April 2024, Soleno Therapeutics reported that diazoxide choline had been granted Breakthrough Therapy Designation by the US FDA for the treatment of adults and children aged 4 and up with genetically proven Prader-Willi syndrome and hyperphagia. The designation represents the Agency's decision that, based on an evaluation of preliminary data from the Phase 3 clinical development program, diazoxide choline may outperform existing treatments on a clinically relevant endpoint(s).
• In June 2023, Acadia Pharmaceuticals announced that it had added a new Phase 3 research candidate to its rare illness portfolio, ACP-101 (intranasal carbetocin), for the treatment of hyperphagia (a false and relentless state of starving) in Prader-Willi syndrome.
• In June 2023, Aardvark Therapeutics announced that the FDA had designated ARD-101, an oral small molecule bitter taste receptor (TAS2R) agonist, as an orphan drug. Early clinical study results point to a promising future for a new class of pharmaceuticals that could benefit people suffering from insatiable hunger (hyperphagia) and aggressive food-seeking behaviors, as well as obesity and metabolic conditions, all of which are common in the rare genetic condition PWS.

Key Highlights:

• The prevalence of Prader-Willi syndrome is one in every 20,000 to 30,000 births.
• Prader-Willi syndrome affects around 400,000 people worldwide, 20,000 of whom live in the United States.
• It remains the most common hereditary cause of life-threatening obesity.
• The reported prevalence rates for Prader-Willi syndrome range from one per 8,000 people in rural Sweden to one per 16,000 people in western Japan.
• The annual mortality rate of PWS ranges between 1% and 4%.

Drugs:

GENOTROPIN (somatropin) is a prescription drug licensed in the United States for children with Prader-Willi syndrome. GENOTROPIN is injected just beneath the skin and is available in a variety of devices to meet specific dose requirements. GENOTROPIN is similar to the body's natural growth hormone and has a well-established safety profile.

DCCR is a novel, proprietary, extended-release dosage form that contains diazoxide choline, the crystalline salt of diazoxide, and is given once a day. Soleno developed and obtained significant patent protection for the therapeutic use of diazoxide, diazoxide choline, and DCCR in patients with PWS. The DCCR development program is supported by data from five completed Phase 1 clinical studies in healthy volunteers and three completed Phase 2 clinical studies, one of which included PWS patients.

RGH-706 is an experimental treatment being developed by Gedeon Richter Plc for weight management in PWS. This is an oral melanin-concentrating hormone receptor 1 (MCHR1) antagonist that is being tested in a phase 2 clinical trial (NCT05322096) to investigate its efficacy, safety, and tolerability in patients with PWS. The binding of MCH to brain receptors increases the desire to eat. RGH-706 is intended to inhibit this action and reduce appetite.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2024-2034
   

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the Prader-Willi syndrome market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the Prader-Willi syndrome market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current Prader-Willi syndrome marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the Prader-Willi syndrome market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2034?
• What was the country-wise size of the Prader-Willi syndrome market across the seven major markets in 2024 and what will it look like in 2034?
• What is the growth rate of the Prader-Willi syndrome market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (2018-2034) of Prader-Willi syndrome across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Prader-Willi syndrome by age across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Prader-Willi syndrome by gender across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Prader-Willi syndrome by type across the seven major markets?
• How many patients are diagnosed (2018-2034) with Prader-Willi syndrome across the seven major markets?
• What is the size of the Prader-Willi syndrome patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2024-2034) across the seven major markets?
• What are the key factors driving the epidemiological trend of Prader-Willi syndrome?
• What will be the growth rate of patients across the seven major markets?
   

Prader-Willi Syndrome: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for Prader-Willi syndrome drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Prader-Willi syndrome market?
• What are the key regulatory events related to the Prader-Willi syndrome market?
• What is the structure of clinical trial landscape by status related to the Prader-Willi syndrome market?
• What is the structure of clinical trial landscape by phase related to the Prader-Willi syndrome market?
• What is the structure of clinical trial landscape by route of administration related to the Prader-Willi syndrome market?