The phenylketonuria (PKU) treatment market is anticipated to grow at a lucrative growth during the forecast period. PKU is a genetic metabolism defect that results in decreased metabolism of the amino acid called phenylalanine. PKU has no definite cure yet, it can only be managed using additional therapy and diet controls. Unmanaged PKU can lead to various intellectual disability, seizures, behavioral and mental disorders in children. In 2019, the global phenylketonuria treatment market was more than $570 million with a growth of around 22% YoY. Kuvan is a major drug for the treatment of phenylketonuria however by the fourth quarter of 2020, its generic version will be available in the US market. Due to these, a drop in the growth can be witnessed in the near future. The patent expiration offers considerable opportunities to the new market players in the US, Europe, and other regulated market. 

Moreover, the surge in the use of various gene and targeted therapy for the management of PKU is anticipated to significantly drive the phenylketonuria treatment market. In addition, the increasing R&D activities for the development of new effective drugs are also expected to fuel the growth of the phenylketonuria treatment market. In addition to this, pipeline drugs such as SYNB1618 and many more will also present new opportunities for the further expansion of the global phenylketonuria treatment market.

Segmental Outlook 

The global phenylketonuria treatment market is segmented based on drug type. The market is sub-segmented into kuvan, playnziq, and others.  Among these, the kuvan sub-segment is anticipated to hold the largest market share during the forecast period. Kuvan was approved for the treatment of PKU by the US FDA in December 2007 and by the EU in December 2008. However, the drug will start to lose its patent from 2020. Playnziq is expected to show a significant growth rate during the forecast period as it was approved in May 2018 and will not lose any patent until 2025. Other segments will also segment with a lucrative growth with opportunities in new drug discoveries for the treatment of PKU during the forecast period. 

Global Phenylketonuria Treatment Market Share by Drug Type, 2019 (%)

Regional Outlooks

The global phenylketonuria treatment market is further segmented based on geography including North America, Europe, Asia-Pacific, and the Rest of the World. North America is expected to hold the largest market share in the phenylketonuria treatment market during the forecast period. As per the National Organization for Rarer Disorder (NORD), the reported incidence of PKU from newborn screening programs ranges from 1 in 13,500 to 19,000 in the US. The high cost of drug and high healthcare expenditure by the people and government is providing the largest market share to the region. Moreover, the disease is covered by most of the health insurance in the US.

Global Phenylketonuria Treatment Market Growth, by Region 2020-2026

Asia-Pacific will augment with the considerable growth rate in the Phenylketonuria Treatment market 

Asia-Pacific is anticipated to grow with a considerable growth rate in the phenylketonuria treatment market owing to the developing healthcare sector of the region. Additionally, increasing awareness regarding the PKU disorder and surge in healthcare spending by developing economies such as China and India also significantly aid in the growth of the market. 

Market Players Outlook

Phenylketonuria Treatment market is a consolidated market however in the near future the number of active companies can increase in the global market. The key players operating in the market include BioMarin Pharmaceutical Inc., Censa Pharmaceuticals Inc., Daiichi Sankyo Co. Ltd., Pluvia AS, Synlogic Inc., and others. The market players are considerably contributing to the market growth by the adoption of various strategies including merger & acquisition, collaborations with government, funding to the start-ups, and new product launches to stay competitive in the market. 

Recent Activity

• In May 2020, Censa Pharmaceuticals, Inc. was acquired by the PTC Therapeutics, Inc. With the acquisition PTC Therapeutics, Inc. will forward the Phase 3 study of CNSA-001 that will be used in the management of PKU. 
• In May 2020, Generation Bio Co. filed for a $125 million IPO to the advancement of liver-targeted therapies for hemophilia A and phenylketonuria (PKU). The funds will allow the company to finish IND-enabling studies for both programs as well as start a clinical trial for one.
• In February 2019, Codexis, Inc. announced that Nestlé Health Science has exercised its option to obtain an exclusive license for the global development and commercialization of Codexis’ orally delivered enzyme CDX-6114. The enzyme is used for the management of PKU across the globe.
• In May 2018, the US FDA approved a new drug called Palynziq for adults for phenylketonuria (PKU). Palynziqis is manufactured and marketed by BioMarin Pharmaceutical Inc.

The Report Covers

• Market value data analysis of 2019 and forecast to 2026.
• Annualized market revenues ($ million) for each market segment.
• Country-wise analysis of major geographical regions.
• Key companies operating in the global Phenylketonuria Treatment market. Based on the availability of data, information related to new product launches, and relevant news is also available in the report.
• Analysis of business strategies by identifying the key market segments positioned for strong growth in the future.
• Analysis of market-entry and market expansion strategies.
• Competitive strategies by identifying ‘who-stands-where’ in the market.