Phenylketonuria (PKU) is a rare genetic disorder caused due to increased level of phenylalanine in the blood. The excess amount of phenylalanine can cause intellectual disability and other serious health problems. The most severe condition of this disorder is termed as classic PKU and symptoms related to it are seizures, tremors, or trembling and shaking, stunted growth, hyperactivity, skin conditions such as eczema, and musty odor of their breath, skin, or urine. PKU can be managed by maintaining phenylalanine free diet. KUVAN (sapropterin dihydrochloride) is a U.S. Food and Drug Administration approved drug which is majorly used in the treatment of Phenylketonuria.
Phenylketonuria Treatment Market Drivers
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, increasing acquisitions and collaborations between key players is also expected to create a lucrative environment for growth of global phenylketonuria treatment market. For instance, in 2015, BioMarin Pharmaceutical Inc. acquired all global rights of Kuvan (sapropterin dihydrochloride) and pegvaliase from Merck Serono (Merck). Kuvan is an oral therapy indicated for the treatment of hyperphenylalaninemia (HPA) 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 research and development of novel treatment for phenylketonuria.
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Phenylketonuria Treatment Market Regional Analysis
North America is expected to hold a dominant position in global phenylketonuria treatment market, owing to initiatives taken by governments for the 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, launches of novel products by U.S.-based key players is also expected to drive growth of North America phenylketonuria treatment market. For instance, in 2017, Vitaflo USA, LLC launched its new PKU Sphere, a low phenylalanine medical food used in dietary management of Phenylketonuria (PKU). Europe is also expected to exhibit significant growth in the near future, owing to increasing approval of new drugs 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.
Phenylketonuria Treatment Market Restraint
According to data published by The ZB Foundation in 2017, Phenylketonuria is a rare inherited disease that affects only about 1 in 10,000 to 15,000 newborn children. The lack of awareness about the rare disease is a major factor, which is expected to restrain growth of the market in the near future.
Phenylketonuria Treatment Market Key Players
Key players operating in 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. Key Players in global phenylketonuria treatment market are focusing on strategic collaborations for the development of novel therapies for the treatment of phenylketonuria. 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.
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