Phenylketonuria (PKU) is an inborn error of amino acid metabolism. It is a rare genetic disorder that increases the level of phenylalanine in the blood and causes problems throughout the body. Some of them includes seizures, developmental delays, mental or behavioural health problems. PKU is caused by deficiency of Phenylalanine hydroxylase (PAH) enzyme that converts phenlyalanine to tyrosine. However, in some cases, defects in co-factor (BH4) of PAH, also results in PKU. This disease can be treated by medication such as Kuvan, Playnziq, and gene therapy.
Global phenylketonuria treatment market is expected to witness lucrative growth over the forecast period. The increase in research & development of novel drugs is estimated to project the market growth. For instance, in 2018, BioMarin Pharmaceuticals, Inc. launched Palynziq (pegvaliase) drug for the treatment of PKU. Furthermore, in 2018, the company received the European Medicines Agency (EMA) approval for the Marketing Authorization Application (MAA) for pegvaliase. Moreover, in April 2018, Synlogic received Fast Track designation approval for its SYNB1618 by FDA.
In addition, presence of pipeline drugs such as CNSA-001, SYNB1618, and RTX-134 is anticipated to boost the market growth. CNSA-001 is under Phase 2 while SYNB1618 is undergoing Phase 1 clinical trial. Moreover, rising collaborations, mergers & acquisition among the key market players is expected to fuel the market growth. For instance, in January 2018, Retrophin, Inc. collaborated with Censa Pharmaceuticals to develop CNSA-001 for the treatment of PKU.
In addition, in May 2017, Synlogic, Inc. entered into a merger agreement with Mirna Therapeutics, Inc. The merged company operates under Synlogic name and focuses on 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.
Furthermore, introduction of new therapy such as gene therapy, for the treatment of PKU is expected to drive the market. Homology Medicines, Inc., is focusing on the development of gene therapy products such as HMI-102 & HMI-103 for treatment of PKU in adults and pediatrics respectively. In addition, rising prevalence of PKU across the globe is estimated to further enhance the market growth. According to the Genetic Home Reference, it was estimated that 1 in 10,000 to 15,000 cases of PKU occurs in newborns in the U.S. In Europe, some countries have high incidence rate of PKU such as 1 in 4,000 of incidence in Turkey.
Geographically, North America dominated the market in 2018 owing to presence of established healthcare facilities, high healthcare expenditure, and high awareness regarding early diagnosis and treatment of diseases. Furthermore, rising government initiatives for the treatment of PKU is expected to drive the market in this region.
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