The Phenylketonuria market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Phenylketonuria: An Overview
Phenylketonuria (PKU) is an inborn error of metabolism that is detectable during the first days of life via routine newborn screening. PKU is characterized by the absence or deficiency of an enzyme called phenylalanine hydroxylase (PAH), responsible for processing the amino acid phenylalanine.
PKU is inherited in families in an autosomal recessive pattern. Autosomal recessive inheritance means that a person has two copies of the gene that is altered. Usually, each parent of an individual who has PKU carries one copy of the altered gene. Since each parent also has a normal gene, they do not show signs or symptoms of PKU
Newborn blood testing identifies almost all cases of phenylketonuria. All 50 states in the United States require newborns to be screened for PKU. Many other countries also routinely screen infants for PKU. If a person has PKU or a family history of it, the doctor may recommend screening tests before pregnancy or birth. It’s possible to identify PKU carriers through a blood test.
Phenylketonuria Market Key Facts
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The total diagnosed prevalent cases of PKU in the 7MM was 49,800+ in 2020. The majority of the cases of PKU are diagnosed in infants due to the high rate of newborn screening.
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In 2020, there were a total of 24,100+ Classic PKU (cPKU), 14,700+ Mild PKU (mPKU), and 10,900+ Mild hyperphenylalaninemia (MHP) cases in the 7MM.
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In EU-5, Germany has the highest number of cases, i.e., 8,300+, followed by France with 7,500+ cases and the UK with 6,500+ cases. While Spain has the least number of cases with 2,400+ in 2020.
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In 2020, the diagnosed prevalent cases of PKU in Japan were 1,000+.
Phenylketonuria (PKU) market size is expected to increase during the forecast period owing to the launch of emerging therapies. Extensive research and development activities of pharmaceutical companies and the rising prevalence will also fuel the market growth.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Phenylketonuria market size by analyzing the impact of current and emerging therapies in the market. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies.
The report gives complete detail of the Phenylketonuria market trend for each marketed drug and late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, mechanism of action (MoA), competition with other therapies, brand value, and their impact on the market.
Phenylketonuria Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of diagnosed patient pool and future trends.
Phenylketonuria (PKU) Epidemiology Segmentation –
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Diagnosed prevalent Cases
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Age-specific diagnosed prevalent cases
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Severity-specific Diagnosed Prevalent Cases
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Diagnosed Prevalent cases of Phenylketonuria by mutation type
Phenylketonuria Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Phenylketonuria market or expected to get launched during the study period. The analysis covers Phenylketonuria market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Phenylketonuria Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Phenylketonuria Therapeutics Analysis
Companies across the globe are diligently working toward the development of novel treatment therapies with a considerable amount of success over the years.
Some of the key companies in the Phenylketonuria (PKU) Market include:
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Codexis
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Synlogic
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Censa Pharmaceuticals
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Homology Medicines
And many others.
Phenylketonuria (PKU) Therapies covered in the report include:
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SYNB1618
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CNSA-001
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HMI-102
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BMN 307
And many more.
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Phenylketonuria Competitive Intelligence Analysis
4. Phenylketonuria Market Overview at a Glance
5. Phenylketonuria Disease Background and Overview
6. Phenylketonuria Patient Journey
7. Phenylketonuria Epidemiology and Patient Population
8. Phenylketonuria Treatment Algorithm, Current Treatment, and Medical Practices
9. Phenylketonuria Unmet Needs
10. Key Endpoints of Phenylketonuria Treatment
11. Phenylketonuria Marketed Products
12. Phenylketonuria Emerging Therapies
13. Phenylketonuria Seven Major Market Analysis
14. Attribute Analysis
15. Phenylketonuria Market Outlook (7 major markets)
16. Phenylketonuria Access and Reimbursement Overview
17. KOL Views on the Phenylketonuria Market.
18. Phenylketonuria Market Drivers
19. Phenylketonuria Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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