Market Overview
- The Global Phenylketonuria Treatment Market is expected to grow at a CAGR of 9.56% during the forecasting period (2020-2027).
- Phenylketonuria (PKU) is a rare genetic disorder that causes an amino acid called phenylalanine to build up in the body. Phenylalanine is an amino acid (a building block protein), which is obtained from food and diet. It varies from mild to a severe form, classic PKU is considered as most severe, and variant PKU is a less severe form of the disease.
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Market Dynamics
- The global phenylketonuria (PKU) treatment market is growing due to factors such as the rising prevalence of PKU globally, increasing the development of new drugs for the treatment of phenylketonuria, and rising initiatives to increase awareness towards the treatment phenylketonuria globally.
- The rising incidences of PKU have resulted in the high demand for its treatment globally. For instance, according to the Genetic Home Reference, 1 in 10,000 to 15,000 cases of PKU occur in newborns in the U.S. In Europe, some countries have a high incidence rate of PKU, such as 1 in every 4500 babies in Ireland and 1 in 4,000 of individuals in Turkey, are suffering from the target disease.
- Also, rising development and commercialization of new drugs are expected to drive market growth. For instance, In 2018, BioMarin Pharmaceuticals, Inc. launched enzyme therapy drug-Palynziq (penalize), for the treatment of PKU in the U.S. Also, the company received the European Medicines Agency (EMA) approval for the Marketing Authorization Application (MAA) for the same drug in 2018 and launched the drug in May 2019.
- The increasing number of pipeline drugs, such as RTX-134, SYNB1618, and CNSA-001, are also anticipated to boost the market growth over the forecast period. Synlogic, Inc.’s SYNB1618 drug is undergoing Phase 1/2a clinical trials and also received fast-track designation from FDA. Also, other companies, such as Rubius Therapeutics, Inc., Retrophin, Inc., and Homology Medicines, Inc. are researching to develop novel therapies, such as oral, enzyme therapy, gene therapy.
- Also, the rising merger, acquisition, and collaboration activities among the major key players are contributing to the growth of the phenylketonuria treatment market. For instance, in January 2018, Retrophin, Inc. entered into a joint agreement with Cena Pharmaceuticals to manufacture CNSA-001. Under this agreement, Cena will perform clinical trial programs for CNSA-001 and after drug development, Retrophin will acquire exclusive rights from Cena.
- Further, in May 2017, Synlogic, Inc. entered into a merger agreement with Mirna Therapeutics, Inc. The merged company operates under Synlogic name and is engaged in the discovery and development of new drugs for Synthetic Biotic medicines. These medicines are designed using synthetic biology to reprogram microbes genetically to treat inflammatory and metabolic diseases, such as PKU and cancer.
Market Segmentation Analysis
- By product type, the phenylketonuria treatment market is segmented into drugs and dietary supplements. Based on drugs, the market is further segmented into Kuvan, Palynziq, and others. Kuvan (sapropterin dihydrochloride) is an oral drug manufactured by BioMarin Pharmaceutical, Inc. which was approved in 2007 in the U.S. and 2008 in Europe. Kuvan is an oral pharmaceutical formulation of BH4, the natural cofactor for the PAH enzyme, which stimulates the activity of the residual PAH enzyme to metabolize phenylalanine into tyrosine. Kuvan is to be used in conjunction with a phenylalanine restricted diet. In April 2017, BioMarin Pharmaceutical Inc. entered into a settlement agreement with Par Pharmaceutical that resolves patent litigation in the United States (U.S.) related to BioMarin's Kuvan® (sapropterin dihydrochloride) 100mg oral tablets and powder for oral solution in 100mg packets.
- On the other hand, in 2018, Palynziq (penalize-pqpz) was approved by the FDA for adults with PKU. Palynziq is an injectable enzyme therapy for patients who have uncontrolled blood phenylalanine concentrations on current treatment. Palynziq is manufactured by BioMarin Pharmaceutical Inc. The drug was approved in 2018 in the U.S. and received the EMA approval for the Marketing Authorization Application (MAA) in May 2019.
Market Geographical Analysis
- North America is dominating the global phenylketonuria treatment market accounting for the largest market share in 2018, owing to favorable regulations and rising government initiatives for the treatment of PKU. For instance, according to the FDA, in 2018, phenylketonuria affected about 1 in 10,000 to 15,000 people in the U.S. This incidence is greater for Caucasian and Native American populations and less for African American, Hispanic, and Asian populations. In the United States, PKU is usually detected at birth by newborn screening tests and dietary therapy started in consultation with a dietitian and geneticist/metabolism specialist. Also, increasing awareness programs conducted by private and public organizations are fuelling the growth of the market. Some of the organizations conducting such programs are SickKids, Children’s Hospital of Wisconsin, CHOC Children’s, and Boston Children’s Hospital. These programs aim to promote optimal emotional, developmental, and physical health PKU patients.
- On the other hand, Europe is expected to grow at the fastest CAGR during the forecast period (2020-2027), owing to rising incidences of PKU in this region, and the development of a generic version of Kuvan, and introduction of palynziq drug in this region. According to the Orphanet, it is estimated that 1 in 10,000 newborns in Europe is suffering from PKU. The target disease is highly prevalent in European countries, such as Italy, Ireland, and Turkey. It is estimated that around 1 in 4000 individuals in Turkey were diagnosed with PKU. Also, the availability of reimbursement policies for high-cost treatments of PKU is expected to boost the growth of the market in this region. For instance, Kuvan is currently approved for use and reimbursed in many European countries, such as Germany, Belgium, France, Italy, and Austria.
Market Competitive Trends
- The phenylketonuria treatment market is competitive with players focusing on the development of new drugs to gain a significant market share. Some of the major players include BioMarin Pharmaceutical Inc., Daiichi Sankyo Company, Limited, SOM Innovation Biotech, S.L., Retrophin, Inc., Censa Pharmaceutical, Inc., Erytech Pharma, Synlogic, Inc., Homology Medicines, Inc., Codexis, Inc., Rubius Therapeutics, Inc., among others.
- The key players are adopting various growth strategies such as product launches, mergers & acquisitions, partnerships, and collaborations which are contributing to the growth of the phenylketonuria treatment market globally.
- In July 2019, Synlogic, Inc, a clinical-stage company developing a novel class of Synthetic BioticTM therapeutics, announced positive top-line clinical data from patient cohorts of a randomized, double-blind, placebo-controlled Phase 1/2a study of SYNB1618, which is being developed for the treatment of phenylketonuria (PKU).
- In March 2019, BioMarin Pharmaceutical Inc. announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion for the company's Marketing Authorization Application (MAA) for Palynziq® (penalize) Injection to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options.
- In May 2019, Retrophin, Inc. provided initial funds to the Rare Caregiver Respite Program. The program was launched by the National Organization for Rare Disorders (NORD), which aims to provide various services and financial assistance to the patients.
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This report studies Phenylketonuria (PKU) in Global market, especially in North America, China, Europe, Southeast Asia, Japan and India, with production, revenue, consumption, import and export in these regions, from 2013 to 2018, and forecast to 2025This report focuses on top manufacturers in global market, with production, price, revenue and market share for each manufacturer, coveringRequest Free Sample Report: https://www.qymarketinsights.com/report-detail/37110/request-sampleBiomarin(US)Vitaflo(UK)Mead Johnson(US)Nutricia(US)Abbott(US)Prominmetabolics(US)Cambrooke(US)Juvela(UK)Firstplay Dietary(UK)On the basis of product, this report displays the production, revenue, price, market share and growth rate of each type, primarily split intoMedicationsSupplementsBy Application, the market can be split intoHouseholdHospitalBy Regions, this report covers (we can add the regions/countries as you want)North AmericaChinaEuropeSoutheast AsiaJapanIndiaAccess Full Report: https://www.qymarketinsights.com/report/global-phenylketonuria-pku-market-professional-survey-report-2018-37110.htmlIf you have any special requirements, please let us know and we will offer you the report as you want.Table of ContentsGlobal Phenylketonuria (PKU) Market Professional Survey Report 20181 Industry Overview of Phenylketonuria (PKU)1.1 Definition and Specifications of Phenylketonuria (PKU)1.1.1 Definition of Phenylketonuria (PKU)1.1.2 Specifications of Phenylketonuria (PKU)1.2 Classification of Phenylketonuria (PKU)1.2.1 Medications1.2.2 Supplements1.3 Applications of Phenylketonuria (PKU)1.3.1 Household1.3.2 Hospital1.3.3 Application 31.4 Market Segment by Regions1.4.1 North America1.4.2 China1.4.3 Europe1.4.4 Southeast Asia1.4.5 Japan1.4.6 India2 Manufacturing Cost Structure Analysis of Phenylketonuria (PKU)2.1 Raw Material and Suppliers2.2 Manufacturing Cost Structure Analysis of Phenylketonuria (PKU)2.3 Manufacturing Process Analysis of Phenylketonuria (PKU)2.4 Industry Chain Structure of Phenylketonuria (PKU)Contact UsMark StoneSales ManagerPhone: (201) 465-4211Email: [email protected]: https://www.qymarketinsights.com/
The latest trending report Global Phenylketonuria (PKU) Market 2020 by Manufacturers, Regions, Type and Application, Forecast to 2025 offered by DecisionDatabases.com is an informative study covering the market with detailed analysis.
The report will assist reader with better understanding and decision making.The global Phenylketonuria (PKU) market size is expected to gain market growth in the forecast period of 2020 to 2025, with a CAGR of 7.2% in the forecast period of 2020 to 2025 and will expected to reach USD 1990.1 million by 2025, from USD 1507.1 million in 2019.The Phenylketonuria (PKU) market report provides a detailed analysis of global market size, regional and country-level market size, segmentation market growth, market share, competitive Landscape, sales analysis, impact of domestic and global market players, value chain optimization, trade regulations, recent developments, opportunities analysis, strategic market growth analysis, product launches, area marketplace expanding, and technological innovations.Browse the complete report and table of contents @ https://www.decisiondatabases.com/ip/18340-phenylketonuria-pku-market-analysis-reportMarket segmentationPhenylketonuria (PKU) market is split by Type and by Application.
For the period 2015-2025, the growth among segments provide accurate calculations and forecasts for sales by Type and by Application in terms of volume and value.
This analysis can help you expand your business by targeting qualified niche markets.By Type, Phenylketonuria (PKU) market has been segmented into Medications, Supplements, Others, etc.By Application, Phenylketonuria (PKU) has been segmented into Household, Hospital, Others, etc.Regions and Countries Level AnalysisRegional analysis is another highly comprehensive part of the research and analysis study of the global Phenylketonuria (PKU) market presented in the report.
For the historical and forecast period 2015 to 2025, it provides detailed and accurate country-wise volume analysis and region-wise market size analysis of the global Phenylketonuria (PKU) market.The report offers in-depth assessment of the growth and other aspects of the Phenylketonuria (PKU) market in important countries (regions), including United States, Canada, Mexico, Germany, France, United Kingdom, Russia, Italy, China, Japan, Korea, India, Southeast Asia, Australia, Brazil and Saudi Arabia, etc.
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The World Phenylketonuria (PKU) Market Research Report 2025 (covering USA, Europe, China, Japan, India, South East Asia and etc), is an informative market report offered by DecisionDatabases.com.Phenylketonuria (PKU) market research report provides the newest industry data and industry future trends, allowing you to identify the products and end users driving Revenue growth and profitability.The industry report lists the leading competitors and provides the insights strategic industry Analysis of the key factors influencing the market.The report includes the forecasts, Analysis and discussion of important industry trends, market size, market share estimates and profiles of the leading industry Players.Final Report will cover the impact of COVID-19 on this industry.Access Full Report with Table of Contents @ https://www.decisiondatabases.com/ip/50719-world-phenylketonuria-pku-market-reportThe Players mentioned in our reportBiomarinVitafloNutriciaCambrookeDr.
Sch?rProminmetabolicsPKU PerspectivesMead JohnsonGlobal Phenylketonuria (PKU) Market: Product Segment AnalysisMedicationsSupplementsGlobal Phenylketonuria (PKU) Market: Application Segment AnalysisHouseholdHospitalGlobal Phenylketonuria (PKU) Market: Regional Segment AnalysisUSAEuropeJapanChinaIndiaSouth East AsiaDownload Free Sample Report of World Phenylketonuria (PKU) Market @ https://www.decisiondatabases.com/contact/download-sample-50719There are 10 Chapters to Deeply Display the World Phenylketonuria (PKU) Market.Chapter 1 About the Phenylketonuria (PKU) IndustryChapter 2 World Market Competition LandscapeChapter 3 World Phenylketonuria (PKU) Market shareChapter 4 Supply Chain AnalysisChapter 5 Company ProfilesChapter 6 Globalisation & TradeChapter 7 Distributors and CustomersChapter 8 Import, Export, Consumption and Consumption Value by Major CountriesChapter 9 World Phenylketonuria (PKU) Market Forecast through 2025Chapter 10 Key success factors and Market OverviewPurchase full World Phenylketonuria (PKU) Market Research Report @ https://www.decisiondatabases.com/contact/buy-now-50719Other Reports by DecisionDatabases.com:World M-phenylene Diamine (MPD) (CAS 108-45-2) Market Research Report 2024 (covering USA, Europe, China, Japan, India, South East Asia and etc)World Diphenylamine Market Research Report 2022 (covering USA, Europe, China, Japan, India, South East Asia and etc)About-Us:DecisionDatabases.com is a global business research reports provider, enriching decision makers and strategists with qualitative statistics.
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The Report entitled 2019-2026 Global Phenylketonuria Treatment Market Report explores the essential factors of the Phenylketonuria Treatment market considering such as industry situations, market demands, market players adopted business strategies and their growth scenario.
The Global Phenylketonuria Treatment market has been separated by this report based on the key players profiles, Type, Application and Regions.Phenylketonuria, also known as PKU, is a genetic disorder in which, the levels of phenylalanine increases in blood.
Phenylketonuria might lead to seizures, behavioral problems, psychiatric disorder, and delayed development is not treated.
Moreover, the increased level of phenylalanine can cause intellectual disability and other health related problems.Download Exclusive Free Sample Of This Report @ https://dataintelo.com/request-sample/?reportId=719Children suffering from phenylketonuria might have the musty odor due to the excess phenylalanine in the body.
Similarly, the patients tend to have light skin and hair color; and other skin disorders such as eczema.
Phenylketonuria vary from mild to severe from, classic PKU is considered as most severe, and variant PKU is a less severe form of the diseases.
