Phenylketonuria Treatment Market Size, Share, Growth and Trend Analysis Report

by Susan Hill Business Consultant

The global phenylketonuria treatment market size is expected to reach USD 1.02 billion by 2026 registering a CAGR of 11.0%, according to a new report by Grand View Research, Inc. Rising prevalence of PKU across the world and favorable government regulations are the factors expected to spur the market growth over the forecast period.

For instance, according to the FDA, in 2018, phenylketonuria affected about 1 in 10,000 to 15,000 people in U.S. Furthermore, according to the Orphanet, it was estimated that 1 in 10,000 newborns in Europe is suffering from PKU. The target disease is highly prevalent in European countries, such as Italy, Ireland, and Turkey. It was estimated that around 1 in 4000 individuals in Turkey were diagnosed with PKU.

Various awareness programs conducted by private and public organizations are also projected to fuel market growth. Some of the organizations conducting such programs are SickKids, Children’s Hospital of Wisconsin, CHOC Children’s, and Boston Children’s Hospital. These programs aim to promote optimal emotional, developmental, and physical health PKU patients.

Furthermore, continuous R&D for the disease treatment is estimated to enhance the market growth. For instance, Synlogic, Inc.’s SYNB1618 drug is undergoing Phase 1/2a clinical trials and also received fast-track designation from FDA. In addition, other companies, such as Rubius Therapeutics, Inc., Retrophin, Inc., and Homology Medicines, Inc. are also conducting research to develop novel therapies, such as oral, enzyme therapy, gene therapy.

Availability of reimbursement policies for high-cost treatments of PKU is also projected to boost the market growth. Kuvan is currently approved for use and reimbursed in many European countries, such as Germany, Belgium, France, Italy, and Austria. Furthermore, increasing number of funding programs for R&D by various organizations is anticipated to drive the market. For instance, the National PKU Alliance provides funding to research projects that helps in promoting advances in the management and treatment of PKU.

Further key findings from the study suggest:

Kuvan held the largest market share in 2018 as it is the first and only approved drug for PKU treatment since 2007
North America dominated the phenylketonuria treatment market in 2018 owing to the presence of well-established healthcare infrastructure, high healthcare expenditure, and favorable government regulations
Europe region is expected to exhibit the fastest CAGR over the forecast period owing to rising R&D activities for the development of new PKU drugs
Major companies in the market are BioMarin Pharmaceuticals, Inc.; Retrophin, Inc.; Synlogic, Inc.; Censa Phamraceuticals; Homology Medicinces, Inc.; and Rubius Therapeutics, Inc.