Phenylketonuria (PKU) is the rare genetic condition caused due to increased phenylalanine levels in the blood. Excess phenylalanine amounts can lead to intellectual disability as well as other severe health issues. This disorder’s most serious condition is called classic PKU, its symptoms include seizures, tremors, hyperactivity, stunted growth, or shaking and trembling, skin conditions including eczema, as well as musty odor of the urine, breath, or skin. PKU could be managed through maintaining diet that is phenylalanine free. The KUVAN (sapropterin dihydrochloride) drug is approved by the U.S. Food and Drug Administration that is used majorly for the Phenylketonuria treatment.
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Growing R&D of new drugs to treatment phenylketonuria is expected to propel the global phenylketonuria treatment market growth over the forecast period. For instance, BioMarin Pharmaceutical Inc. in 2017, submitted to the U.S. Food and Drug Administration (FDA), a Biologics License Application (BLA) for its novel Pegvaliase, prescribed for Phenylketonuria (PKU) treatment in patients. European Medicines Agency (EMA), in 2018, accepted the submission of BioMarin to Marketing Authorization Application (MAA) for pegvaliase drug to treat adults suffering from phenylketonuria (PKU), having improper blood phenylalanine control (levels of blood phenylalanine above 600 micromol/l). Synlogic, Inc., in 2017, received the designation of Orphan Drug from the U.S. Food and Drug Administration (FDA) for the SYNB1618. The SYNB1618, is a synthetic biotic drug administered orally indicated to treat PKU.
Moreover, increasing collaborations and acquisitions among key companies is expected to drive growth of the global phenylketonuria treatment market over the forecast period. For instance, BioMarin Pharmaceutical Inc., in 2015, acquired the entire global rights of the Kuvan (sapropterin dihydrochloride) and Merck Serono’s (Merck) pegvaliase. Kuvan is the oral therapy prescribed to treat hyperphenylalaninemia (HPA) owing to phenylketonuria (PKU). The Pegvaliase (PEGylated recombinant phenylalanine ammonia lyase) is investigational enzyme substitution therapy to treat phenylketonuria (PKU). Dimension Therapeutics, Inc. in 2016, entered in a research collaboration of gene therapy with the University of Pennsylvania for R&D phenylketonuria’s novel treatment.
North America is expected to account for the highest share in the global phenylketonuria treatment market, due government initiatives for the treatment and diagnosis of phenylketonuria in this region. For instance, The National PKU Alliance and Baby Genes, in 2017, introduced a program for allowing easy access for genetic testing to people having phenylketonuria, along with their families. This program aims at helping in diagnosis along with assisting people for the phenylketonuria treatment.
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Major players functioning in the global phenylketonuria treatment market are BioMarin Pharmaceutical Inc., American Gene Technologies International Inc., Dimension Therapeutics, Inc., Synthetic Biologics, Inc., SOM Innovation Biotech SL, Codexis, Inc., Daiichi Sankyo Company, Limited, Erytech Pharma SA, and others. Major market players are focused on strategic collaborations to develop novel therapies in order to treat phenylketonuria. For instance, Retrophin, Inc., collaborated with Censa Pharmaceuticals, in January 2018, to develop and evaluate CNSA-001 for phenylketonuria (PKU) treatment. CNSA-001, is the orally bioavailable sepiapterin proprietary form, which serves as the natural tetrahydrobiopterin (BH4) precursor which is converted to BH4 by endogenous enzymatic pathway.
Global Phenylketonuria Treatment Market Taxonomy:
Based on Treatment Type-
- Drugs
- Pegvaliase
- Kuvan
- Others
- Dietary Supplement
Based on Route of Administration-
- Parenteral
- Oral
Based on Distribution Channel-
- Retail Pharmacies
- Hospital Pharmacies
- Online Pharmacies
Based on Region
- North America
- Europe
- Latin America
- Asia Pacific
- Africa
- Middle East
Reasons to Purchase this Report
Current and future of global Phenylketonuria Treatment market outlook in the developed and emerging markets
The segment that is expected to dominate the market as well as the segment which holds highest CAGR in the forecast period.
Regions/countries that are expected to witness the fastest growth rates during the forecast period
The latest developments, market shares, and strategies that are employed by the major market players
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Major Point Answered in Phenylketonuria Treatment Market Research Study are:
What will be the progress rate of the Phenylketonuria Treatment Market for the conjecture period, 2020-2027?
What are the prominent factors driving the Phenylketonuria Treatment Market across different regions?
Who are the major vendors dominating the Phenylketonuria Treatment industry and what are their winning strategies?
What will be the market scope for the estimated period?
What are the major trends shaping the expansion of the industry in the coming years?
What are the challenges faced by the Phenylketonuria Treatment Market?
Actual Numbers & In-Depth Analysis, Business opportunities, Market Size Estimation Available in Full Report.
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