The phenylketonuria treatment market is expected to register a CAGR of around 11 % over the forecast period. The growing burden of phenylketonuria disease and the novel treatments emerging related to phenylketonuria is expected to boost the phenylketonuria treatment market growth. According to the National PKU Alliance, the chances of the gene carrier is around one in fifty people, and the chances of two carriers mating are expected to be around 25%. The degree of affecting the disease is majorly based upon ethnicity and geographical region. According to the same source, in Ireland, around 1 out of 4,500 population is diagnosed with it. Similarly, in Asia, 1 out of 20,000 is diagnosed with it, while in Turkey the incidence is high, i.e., 1 in 4,000.
Key Market Trends
Kuvan Phenylketonuria Treatment Segment is Expected to Hold a Major Market Share in the Phenylketonuria Treatment Market
Kuvan is the only drug present currently for the efficient treatment of phenylketonuria, which is being manufactured by BioMarin Pharmaceuticals. It adds more BH4 compound, helps in stimulating phenylalanine hydroxylase enzyme, which then breakdown the phenylalanine protein. In 2017, it lost its patent, and BioMarin Pharmaceuticals gave license to Par Pharmaceuticals to develop a generic version of Kuvan.
North America is Expected to Hold a Significant Share in the Market during the Forecast Period
North America expected to hold a major market share in the global phenylketonuria treatment market due to a higher incidence rate of phenylketonuria and the development in treatment type. For instance, Homology Medicines Inc., a US-based genetic company, is developing a gene therapy, HMI-102, for phenylketonuria. Currently, its IND (Investigational New Drug) application has been approved by the USFDA (Food & Drug Administration), and it is undergoing the first phase of clinical trials to test safety and efficacy. According to the National Organization for Rare Disorders, there have been approximately 13,500 to 19,000 newborns diagnosed with phenylketonuria disease in the United States. According to the National Centre for Biotechnology Information published study “Phenylketonuria”, the disease is mostly found in Native Americans, and the incidence rate is around 1 in 15,000 people.
The phenylketonuria treatment market is consolidated and consists of a few major players. Some of the companies currently dominating the market are BioMarin Pharmaceuticals Inc., Censa Pharmaceutical, Synlogic Inc., Erytech Pharma SA, Codexis Inc., SOM Innovation Biotech SL, Homology Medicines Inc., and Ultragenyx (Dimension Therapeutics).
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