The 7 major phenylketonuria markets reached a value of US$ 956.5 Million in 2023. Looking forward, the publisher expects the 7MM to reach US$ 4,734.0 Million by 2034, exhibiting a growth rate (CAGR) of 19.45% during 2023-2034.
The phenylketonuria market has been comprehensively analyzed in this report titled "Phenylketonuria Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Phenylketonuria is a rare hereditary condition that results in an accumulation of the amino acid phenylalanine in the body. It is caused by an alteration in the phenylalanine hydroxylase (PAH) gene, which aids in the production of the enzyme required to metabolize phenylalanine. In infants, the symptoms of this ailment can vary from mild to severe and may include a musty odor in the breath, urine, or skin, an unusually small head size, hyperactivity, irritability, delayed development, seizures, skin rashes, etc. Additionally, in older children and adults, the disease manifests as intellectual disability, learning difficulties, behavioral problems, such as anxiety and depression, tremors, jerky movements, etc. Phenylketonuria is typically diagnosed through newborn screening, a blood workup, and a laboratory study. In addition to this, genetic testing is utilized to identify gene mutations associated with this condition. The healthcare provider may also perform certain diagnostic tests and procedures, such as a triple-quadrupole mass spectrometer, urinalysis, Guthrie card bacterial inhibition assay, etc., to confirm a diagnosis among patients.
The increasing cases of genetic mutations and the inflating demand for effective medications to treat such conditions are primarily driving the phenylketonuria market. Besides this, the widespread adoption of several drug formulations, including sapropterin dihydrochloride and pegvaliase-pqpz, for reducing blood phenylalanine levels in patients is acting as another significant growth-inducing factor. Furthermore, the introduction of novel diagnostic techniques, such as tandem mass spectrometry and high-performance liquid chromatography, which can accurately measure blood phenylalanine levels and differentiate between the disease and other conditions that can cause elevated phenylalanine levels, is also bolstering the market growth. Additionally, several key players are exploring the use of gene therapy to restore phenylalanine hydroxylase activity and prevent neurological as well as cognitive complications associated with the ailment. This, in turn, is further creating a positive outlook for the market. Moreover, the ongoing development of numerous biomarkers, including brain-derived neurotrophic factor (BDNF) and oxidative stress markers, for enhancing the accuracy of diagnosis and monitoring of the disorder is expected to drive the phenylketonuria market in the coming years.
This report provides an exhaustive analysis of the phenylketonuria market in the United States, EU5 (Germany, Spain, Italy, France, and 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 phenylketonuria and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and 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 phenylketonuria market in any manner.
The phenylketonuria market has been comprehensively analyzed in this report titled "Phenylketonuria Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Phenylketonuria is a rare hereditary condition that results in an accumulation of the amino acid phenylalanine in the body. It is caused by an alteration in the phenylalanine hydroxylase (PAH) gene, which aids in the production of the enzyme required to metabolize phenylalanine. In infants, the symptoms of this ailment can vary from mild to severe and may include a musty odor in the breath, urine, or skin, an unusually small head size, hyperactivity, irritability, delayed development, seizures, skin rashes, etc. Additionally, in older children and adults, the disease manifests as intellectual disability, learning difficulties, behavioral problems, such as anxiety and depression, tremors, jerky movements, etc. Phenylketonuria is typically diagnosed through newborn screening, a blood workup, and a laboratory study. In addition to this, genetic testing is utilized to identify gene mutations associated with this condition. The healthcare provider may also perform certain diagnostic tests and procedures, such as a triple-quadrupole mass spectrometer, urinalysis, Guthrie card bacterial inhibition assay, etc., to confirm a diagnosis among patients.
The increasing cases of genetic mutations and the inflating demand for effective medications to treat such conditions are primarily driving the phenylketonuria market. Besides this, the widespread adoption of several drug formulations, including sapropterin dihydrochloride and pegvaliase-pqpz, for reducing blood phenylalanine levels in patients is acting as another significant growth-inducing factor. Furthermore, the introduction of novel diagnostic techniques, such as tandem mass spectrometry and high-performance liquid chromatography, which can accurately measure blood phenylalanine levels and differentiate between the disease and other conditions that can cause elevated phenylalanine levels, is also bolstering the market growth. Additionally, several key players are exploring the use of gene therapy to restore phenylalanine hydroxylase activity and prevent neurological as well as cognitive complications associated with the ailment. This, in turn, is further creating a positive outlook for the market. Moreover, the ongoing development of numerous biomarkers, including brain-derived neurotrophic factor (BDNF) and oxidative stress markers, for enhancing the accuracy of diagnosis and monitoring of the disorder is expected to drive the phenylketonuria market in the coming years.
This report provides an exhaustive analysis of the phenylketonuria market in the United States, EU5 (Germany, Spain, Italy, France, and 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 phenylketonuria and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and 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 phenylketonuria market in any manner.
Time Period of the Study
- Base Year: 2023
- Historical Period: 2018-2023
- 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 phenylketonuria market
- Historical, current, and future performance of various therapeutic categories in the market
- Sales of various drugs across the phenylketonuria market
Competitive Landscape:
This report also provides a detailed analysis of the current phenylketonuria 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
Key Questions Answered in this Report:
Market Insights
- How has the phenylketonuria market performed so far and how will it perform in the coming years?
- What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
- What was the country-wise size of the phenylketonuria market across the seven major markets in 2023 and what will it look like in 2034?
- What is the growth rate of the phenylketonuria 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 phenylketonuria across the seven major markets?
- What is the number of prevalent cases (2018-2034) of phenylketonuria by age across the seven major markets?
- What is the number of prevalent cases (2018-2034) of phenylketonuria by gender across the seven major markets?
- What is the number of prevalent cases (2018-2034) of phenylketonuria by type across the seven major markets?
- How many patients are diagnosed (2018-2034) with phenylketonuria across the seven major markets?
- What is the size of the phenylketonuria patient pool (2018-2023) 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 phenylketonuria?
- What will be the growth rate of patients across the seven major markets?
Phenylketonuria: 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 phenylketonuria 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 phenylketonuria market?
- What are the key regulatory events related to the phenylketonuria market?
- What is the structure of clinical trial landscape by status related to the phenylketonuria market?
- What is the structure of clinical trial landscape by phase related to the phenylketonuria market?
- What is the structure of clinical trial landscape by route of administration related to the phenylketonuria market?
Table of Contents
?1 Preface3 Executive Summary6 Patient Journey9 Phenylketonuria - Unmet Needs10 Phenylketonuria - Key Endpoints of Treatment13. Phenylketonuria - Attribute Analysis of Key Marketed and Pipeline Drugs16 Phenylketonuria - Recent Events and Inputs From Key Opinion Leaders18 Phenylketonuria Market - Strategic Recommendations19 Appendix
2 Scope and Methodology
4 Phenylketonuria - Introduction
5 Phenylketonuria - Disease Overview
7 Phenylketonuria - Epidemiology and Patient Population
8 Phenylketonuria - Treatment Algorithm, Guidelines, and Medical Practices
11 Phenylketonuria - Marketed Products
12 Phenylketonuria - Pipeline Drugs
14. Phenylketonuria - Clinical Trial Landscape
15 Phenylketonuria - Market Scenario
17 Phenylketonuria Market - SWOT Analysis
Methodology
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Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 130 |
| Published | May 2024 |
| Forecast Period | 2023 - 2034 |
| Estimated Market Value ( USD | $ 956.5 Million |
| Forecasted Market Value ( USD | $ 4734 Million |
| Compound Annual Growth Rate | 15.6% |
| Regions Covered | Global |
