Phenylketonuria Treatment Market Application 2020 – Industry Overview, Global Trends, Market Analysis, CAGR Values and Country Level Demand To Forecast by 2026
SEATTLE, March 29, 2021, (PHARMIWEB) — Global Phenylketonuria Treatment Market
Phenylketonuria (PKU) is a rare genetic disorder caused due to the increased levels of phenylalanine in the blood. The excess amount of phenylalanine can cause intellectual disability and other serious health issues. The most severe form of this disorder is termed as ‘classic PKU’ and its general symptoms include seizures, tremors, or trembling and shaking, stunted growth, hyperactivity, skin conditions such as eczema, and musty odor of the breath, skin, or urine. PKU can be managed by maintaining a phenylalanine-free diet. Therefore, KUVAN (sapropterin dihydrochloride) is a U.S. FDA-approved drug, which is majorly used in the treatment of Phenylketonuria.
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Global Phenylketonuria Treatment Market Analysis
Frequent research for development of novel drugs for the treatment of phenylketonuria is expected to drive growth of global phenylketonuria treatment market in the near future. For instance, in 2017, BioMarin Pharmaceutical Inc. submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its new Pegvaliase, indicated for the treatment of patients with Phenylketonuria (PKU). In 2018, European Medicines Agency (EMA) accepted BioMarin’s submission of a Marketing Authorization Application (MAA) for pegvaliase for the treatment of adults with phenylketonuria (PKU) who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l). In 2017, Synlogic, Inc. received Orphan Drug Designation for its SYNB1618 from U.S. Food and Drug Administration. SYNB1618, an orally administered synthetic biotic medicine indicated for the treatment of PKU. Furthermore, the rising number of acquisitions and collaborations between the key market players is further expected to create a conducive environment for the growth of the global phenylketonuria treatment market. In 2015, BioMarin Pharmaceutical Inc. acquired all global rights of Kuvan (sapropterin dihydrochloride) and pegvaliase from Merck Serono (Merck). Kuvan, for the record, is an oral therapy indicated for the treatment of hyperphenylalaninemia (HPA) caused due to phenylketonuria (PKU). Pegvaliase (PEGylated recombinant phenylalanine ammonia lyase) is an investigational enzyme substitution therapy for the treatment of phenylketonuria (PKU). In 2016, Dimension Therapeutics, Inc. entered into a gene therapy research collaboration with the University of Pennsylvania, for R&D of a novel treatment option for phenylketonuria.
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Global Phenylketonuria Treatment Market – Regional Analysis
North America is expected to hold a dominant position in the global phenylketonuria treatment market, owing to several government initiatives aimed at the appropriate diagnosis and treatment of phenylketonuria in the region. For instance, in 2017, The National PKU Alliance and Baby Genes launched a program to allow easy access to genetic testing for people with phenylketonuria, and their families. The program aims to help in diagnosis and assist the people for treatment of phenylketonuria. Furthermore, new product launches by numerous U.S.-based key players is also expected to drive market growth in North America. For instance, in 2017, Vitaflo USA, LLC launched its new PKU Sphere, a low phenylalanine medical food used in dietary management of Phenylketonuria (PKU).
Furthermore, Europe is projected to exhibit significant market growth in the near future, owing to increasing new drug approvals in the region. For instance in 2015, Merck Group, a Germany-based company, received EU-approval to extend the use of Kuvan in children below four years of age suffering from Phenylketonuria.
Global Phenylketonuria Treatment Market – Key Competitors
The well-known players operating in the global phenylketonuria treatment market include BioMarin Pharmaceutical Inc., Dimension Therapeutics, Inc., American Gene Technologies International Inc., Synthetic Biologics, Inc., Codexis, Inc., SOM Innovation Biotech SL, Daiichi Sankyo Company, Limited, and Erytech Pharma SA.
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These market players are currently focusing on strategic collaborations for the development of novel therapies for phenylketonuria treatment. For instance, in January 2018, Retrophin, Inc. and Censa Pharmaceuticals collaborated for the development and evaluation of CNSA-001 for the treatment of phenylketonuria (PKU). CNSA-001, is an orally bioavailable proprietary form of sepiapterin, serves as a natural precursor of tetrahydrobiopterin (BH4) that is converted by an endogenous enzymatic pathway to BH4.
By Treatment Type,
- Dietary Supplement
By Route of Administration,
By Distribution Channel,
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
- North America
- Latin America
- Asia Pacific
- Middle East
Table of Content
Global Phenylketonuria Treatment Market Research Report
Section 1: Global Phenylketonuria Treatment Industry Overview
Section 2: Global Economic Impact on Phenylketonuria Treatment Industry
Section 3: Global Market Competition by Industry Producers
Section 4: Global Productions, Revenue (Value), according to Regions
Section 5: Global Supplies (Production), Consumption, Export, Import, geographically
Section 6: Global Productions, Revenue (Value), Price Trend, Product Type
Section 7: Global Market Analysis, on the basis of Application
Section 8: Phenylketonuria Treatment Market Pricing Analysis
Section 9: Market Chain, Sourcing Strategy and Downstream Buyers
Section 10: Strategies and key policies by Distributors/Suppliers/Traders
Section 11: Key Marketing Strategy Analysis, by Market Vendors
Section 12: Market Effect Factors Analysis
Section 13: Global Phenylketonuria Treatment Market Forecast
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