The Phenylketonuria market growth is driven by factors like increase in the prevalence of Phenylketonuria, investments in research and development, entry of emerging therapies during the study period 2019-2032.
The Phenylketonuria market report also offers comprehensive insights into the Phenylketonuria market size, share, Phenylketonuria epidemiology, emerging therapies, market drivers and barriers, ongoing clinical trials, key collaboration in the space, market uptake by key therapies and companies actively pushing Phenylketonuria market size growth forward.
Some of the key highlights from the Phenylketonuria Market Insights Report:
- Several key pharmaceutical companies, including PTC Therapeutics, Synlogic, and others, are developing novel products to improve the Phenylketonuria treatment outlook.
- The total Phenylketonuria market size will include the market size of the potential upcoming therapies and current treatment regimens in the seven major markets.
- As per DelveInsight analysis, the Phenylketonuria market is anticipated to witness growth at a considerable CAGR
- The pipeline for PKU consists of promising drugs that are anticipated to change the treatment landscape of PKU. The current market anticipates the emergence of PTC923 (PTC Therapeutics) and SYNB1934 (Synlogic) products.
- In 2022, the market size of Phenylketonuria (PKU) was found to be ~USD 610 million in the 7MM. The market size is estimated to increase during the forecast period (2023–2032).
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Phenylketonuria 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. Amino acids are the chemical building blocks of proteins and are essential for proper growth and development. With normal PAH activity, phenylalanine is converted to another amino acid, tyrosine. However, when PAH is absent or deficient, phenylalanine accumulates and is toxic to the brain. Without treatment, most people with PKU would develop a severe intellectual disability. To prevent intellectual disability, treatment consists of a carefully controlled, phenylalanine-restricted diet beginning during the first days or weeks of life.
Do you know the treatment paradigms for different countries? Download our Phenylketonuria Market Sample Report
Recent Breakthroughs in the Phenylketonuria Market:
FDA approves a new treatment for PKU, a rare and serious genetic disease: The U.S. Food and Drug Administration today approved Palynziq (pegvaliase-pqpz) for adults with a rare and serious genetic disease known as phenylketonuria (PKU). Patients with PKU are born with an inability to break down phenylalanine (Phe), an amino acid present in protein-containing foods and high-intensity sweeteners used in a variety of foods and beverages. Palynziq is a novel enzyme therapy for adult PKU patients who have uncontrolled blood Phe concentrations on current treatment.
Phenylketonuria Epidemiology Insights
- The United States contributed to the largest diagnosed prevalent population of Phenylketonuria (PKU), acquiring ~36% of the 7MM in 2022.
- Among EU4 and the UK, Germany accounted for the highest number of Phenylketonuria (PKU) cases, followed by France, whereas Spain had the lowest number of cases in 2022.
- The mutation-specific cases of PKU were further divided into missense mutations, nonsense mutations, mutations at splice sites, deletions, and others. It was found that missense mutations formed the highest number of cases of PKU, whereas the others category formed the least number of cases of PKU, throughout the 7MM, in 2022.
- It was found that missense mutations formed the highest number of cases of PKU, i.e., ~11,300 cases, whereas the others category formed the least number of cases of PKU, i.e., ~850 cases in the US, in 2022.
DelveInsight’s Phenylketonuria market report is prepared on the basis of epidemiology model. It offers comprehensive insights to the Phenylketonuria historical patient pools and forecasted Phenylketonuria patients. The report provides in-depth data of various subtypes and for the same epidemiology is segmented further. The Phenylketonuria Market report proffers epidemiological analysis for the study period 2019-32 in the 7MM segmented into:
- Phenylketonuria Prevalence
- Age-Specific Phenylketonuria Prevalence
- Gender-Specific Phenylketonuria Prevalence
- Diagnosed and Treatable Cases of Phenylketonuria
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Phenylketonuria Treatment Market
The Phenylketonuria market outlook of the report helps to build a detailed comprehension of the historic, current, and forecasted Phenylketonuria market trends by analyzing the impact of current Phenylketonuria therapies on the market, unmet needs, drivers and barriers, and demand for better technology.
This segment gives a thorough detail of Phenylketonuria market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Phenylketonuria market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
According to DelveInsight, the Phenylketonuria market in 7MM is expected to witness a major change in the study period 2019-2032.
Phenylketonuria Key Companies
- PTC Therapeutics
- Synlogic
For more information, visit Phenylketonuria Market Analysis, Patient Pool, and Emerging Therapies
Scope of the Phenylketonuria Market Report:
- 11 Years Forecast
- 7MM Coverage
- Descriptive overview of Phenylketonuria, causes, signs and symptoms, diagnosis, treatment
- Comprehensive insight into Phenylketonuria epidemiology in the 7MM
- Phenylketonuria marketed and emerging therapies
- Phenylketonuria companies
- Phenylketonuria market drivers and barriers
Key Questions Answered in the Phenylketonuria Market Report 2032:
- What was the Phenylketonuria market share distribution in 2019, and how would it appear in 2032?
- What is the total Phenylketonuria market size and the market size by therapy across the 7MM for the study period (2019-32)?
- What are the important findings from 7MM, and which country will have the greatest Phenylketonuria market size from 2019-32?
- During the study period (2019-2032), at what CAGR is the Phenylketonuria market projected to expand at 7MM?
Table of Contents:
1 Phenylketonuria Market Key Comprehensive Insights
2 Phenylketonuria Market Report Introduction
3 Competitive Intelligence Analysis for Phenylketonuria
4 Phenylketonuria Market Analysis Overview at a Glance
5 Executive Summary of Phenylketonuria
6 Phenylketonuria Epidemiology and Market Methodology
7 Phenylketonuria Epidemiology and Patient Population
8 Phenylketonuria Patient Journey
9 Phenylketonuria Treatment Algorithm, Phenylketonuria Current Treatment, and Medical Practices
10 Key Endpoints in Phenylketonuria Clinical Trials
11 Phenylketonuria Marketed Therapies
12 Phenylketonuria Emerging Therapies
13 Phenylketonuria: 7 Major Market Analysis
14 Attribute analysis
15 Access and Reimbursement Overview of Phenylketonuria
16 Phenylketonuria Market Key Opinion Leaders Reviews
18 Phenylketonuria Market Drivers
19 Phenylketonuria Market Barriers
20 SWOT Analysis
21 Disclaimer
22 DelveInsight Capabilities
23 About DelveInsight
Related Reports:
Phenylketonuria Epidemiology 2032
DelveInsight’s “Phenylketonuria – Epidemiology Forecast to 2032” report delivers an in-depth understanding of the disease, historical and forecasted Phenylketonuria epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
Phenylketonuria Pipeline 2023
“Phenylketonuria Pipeline Insights, 2023” report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Phenylketonuria market. A detailed picture of the Phenylketonuria pipeline landscape is provided, which includes the disease overview and Phenylketonuria treatment guidelines.
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