Global Phenylketonuria (PKU) Markets to 2032: Market Share Distribution, Impact of Emerging Therapies, Pricing Variations Among Geographies, Drug Competition, and Future Opportunities

DUBLIN, July 28, 2023 /PRNewswire/ -- The "Phenylketonuria (PKU) Market Forecasts to 2032" report has been added to ResearchAndMarkets.com's offering.

This report provides a detailed understanding of PKU, its epidemiology, and market trends in the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan.

Epidemiology Insights: The report presents historical and forecasted epidemiological data of PKU, segmented by total diagnosed prevalent cases, mutation type-specific cases, age-specific cases, and severity-specific cases in the 7MM. The United States contributed to the largest diagnosed prevalent population of PKU in 2022. Germany accounted for the highest number of PKU cases in the EU4 and the UK. Missense mutations formed the highest number of PKU cases, while others formed the least number of cases in the 7MM.

Current Treatment Scenario and Emerging Therapies: Current treatment options for PKU include dietary supplements, a low-protein diet, and approved drugs like KUVAN (sapropterin hydrochloride) and PALYNZIQ (pegvaliase-pqpz). The report highlights emerging therapies like PTC923 and SYNB1934, which have shown promising safety and efficacy in Phase II trials. These new treatments could potentially change the PKU treatment landscape.

Market Insights: The PKU market was valued at ~610 million in 2022 and is projected to grow during the forecast period (2023-2032). Factors contributing to this growth include the expected launch of upcoming therapies and increased awareness and integration of newborn screening in various countries.

Patient Perspectives and Unmet Needs: The report examines patient perspectives, treatment preferences, and barriers to accessibility. It identifies unmet needs, such as limited treatment options, resulting in untreated PKU patients experiencing intellectual disability and other complications. The development of more effective and personalized treatment options is crucial to address these unmet needs.

Market Access and Reimbursement: The report discusses the reimbursement landscape for PKU therapies, the challenges of high drug prices, and limited evidence for evaluating rare disease drugs. Patient assistance programs, like those offered by NORD, provide financial aid for eligible individuals diagnosed with PKU to help them access treatment.

Key Questions Answered: The report addresses key questions related to the PKU market, such as market share distribution, impact of emerging therapies, pricing variations among geographies, drug competition, and future opportunities. It also covers disease risk, patient burdens, growth opportunities, and treatment guidelines.

Conclusion: The report provides a comprehensive analysis of PKU, its epidemiology, and market trends in different regions. With the emergence of promising pipeline candidates, the PKU treatment landscape is expected to change, offering new hope for patients. Stakeholders can use this report to make informed decisions, identify growth opportunities, and address unmet needs in the PKU market. Effective disease management strategies incorporating personalized treatment approaches are essential to improve the quality of life for PKU patients and their families.

Key Topics Covered

1. Key Insights
2. Report Introduction
3. Phenylketonuria (PKU) Market Overview at a Glance
4. Executive Summary of Phenylketonuria (PKU)
5. Key Events
6. Disease Background and Overview
7. Methodology
8. Epidemiology and Patient Population
9. Patient Journey
10. Marketed Drugs
11. Emerging Drugs
12. Phenylketonuria (PKU): The 7MM Analysis
13. KOL Views
14. SWOT Analysis
15. Unmet Needs
16. Market Access and Reimbursement
17. Appendix

A selection of companies mentioned in this report includes:

• PTC Therapeutics
• Synlogic

For more information about this report visit https://www.researchandmarkets.com/r/xabjx2

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