Phenylketonuria Treatment Market To Witness A Pronounce Growth By 2026 With Market Dominanats BioMarin Pharmaceutical Inc, Dimension Therapeutics Inc, American Gene Technologies International Inc

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.

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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.

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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The global Phenylketonuria treatment market is segmented on the basis of treatment type, route of administration, distribution channel, and region:

By Treatment Type-
Drugs
Kuvan
Pegvaliase
Others
Dietary Supplement

By Route of Administration-
Oral
Parenteral

By Distribution Channel-
Hospital Pharmacies
Retail Pharmacies
Online Pharmacies

By Region-
North America
Latin America
Europe
Asia Pacific
Middle East
Africa

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