Global Phenylketonuria Treatment Market

Phenylketonuria, also known as PKU, is a genetic disorder in which, the levels of phenylalanine increases in blood. Phenylalanine is an amino acid (a building block protein), which is obtained from food and diet. Phenylketonuria might lead to seizures, behavioral problems, psychiatric disorder, and delayed development is not treated. Moreover, the increased level of phenylalanine can cause intellectual disability and other health related problems. Children suffering from phenylketonuria might have the musty odor due to the excess phenylalanine in the body. Similarly, the patients tend to have light skin and hair color; and other skin disorders such as eczema. Phenylketonuria vary from mild to severe from, classic PKU is considered as most severe, and variant PKU is a less severe form of the diseases. Children with classic PKU are at the risk of brain damage and requires treatment. However, patients with mild PKU might not need any treatment. PKU suffering babies have low birth weight and slow body growth. Other associated medical problem includes microcephaly, heart problems, and other behavioral problems

Growing burden of incidence of PKU is major factors driving the growth of global phenylketonuria treatment market. The incidences of PKU varies based on demographics and ethnic groups. In the U.S. PKU affects one among 10,000 to 15,000 newborn babies. Most of these cases are detected by the after the birth screening. The PKU screening test includes bacterial inhibition assay, immunoassay with fluorometric detection, and mass spectrometry for the amino acid measurement.

The global market for phenylketonuria treatment is segmented on basis of product type, route of administration, distribution channel, and geography.

Based on the product of type, the phenylketonuria treatment market is segmented into drugs and nutrition supplements. For the treatment of phenylketonuria, nutrition supplements are widely prescribed by healthcare professionals along with some diet modification. There only few companies which are dealing with the manufacturing of drugs used for the treatment of phenylketonuria. In 2007, Kuvan (sapropterin dihydrochloride), manufactured by BioMarin was approved by Food and Drug Administration (FDA) for the treatment of phenylketonuria in U.S. Kuvan is available in tablet and powder form. The tablet form of drugs is approved in the U.S. European Union, and Canada; and powdered form is approved in U.S. The powdered form of the drug was approved in 2013. Similarly, in 2008, Biopten, manufactured by Daiichi Sankyo Company, Limited was approved in Japan for phenylketonuria treatment.

Based on the route of administration, the market segmented into an oral route and parenteral route. In phenylketonuria treatment market, the oral route is more preferred mainly due to patient’s convenience and ease of administration. However, intravenous injections/ infusions are difficult to administer. To avoid administration difficulties various companies are developing oral drug formulations of drugs to increase ease of administration.

Based on geography, the market is segmented into North America, Latin America, Europe, Asia-pacific, and MEA. The market is majorly driven by developed economies owing to the large patient base suffering from phenylketonuria, high disease awareness and high diagnostic rate of such disease. North America phenylketonuria treatment market is expected to increase over the forecast period due to growing in a number of newborn suffering from phenylketonuria, the rise in awareness among people and improved healthcare infrastructure in the region. Asia Pacific market is projected to grow with significant CAGR over the forecast period due to large pool of population, increase in R & D activities for the development of drugs for phenylketonuria, and growing awareness is contributing the growth of this market Some of the key participating global players in phenylketonuria treatment market are American Gene Technologies International Inc., Dimension Therapeutics, Inc., BioMarin Pharmaceutical Inc., Synthetic Biologics, Inc., Codexis, Inc., SOM Innovation Biotech SL, Daiichi Sankyo Company, Limited and Erytech Pharma SA among others.

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