Market Challenges And Opportunities

Phenylketonuria Treatment Market Drivers

Frequent research for development of novel drugs for the treatment of phenylketonuria is expected to drive growth of global phenylketonuria treatment market in the near future. For instance, in 2017, BioMarin Pharmaceutical Inc. submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its new Pegvaliase, indicated for the treatment of patients with Phenylketonuria (PKU). In 2018, European Medicines Agency (EMA) accepted BioMarin’s submission of a Marketing Authorization Application (MAA) for pegvaliase for the treatment of adults with phenylketonuria (PKU) who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l). In 2017, Synlogic, Inc. received Orphan Drug Designation for its SYNB1618 from U.S. Food and Drug Administration. SYNB1618, an orally administered synthetic biotic medicine indicated for the treatment of PKU. Furthermore, increasing acquisitions and collaborations between key players is also expected to create a lucrative environment for growth of global phenylketonuria treatment market. For instance, in 2015, BioMarin Pharmaceutical Inc. acquired all global rights of Kuvan (sapropterin dihydrochloride) and pegvaliase from Merck Serono (Merck). Kuvan is an oral therapy indicated for the treatment of hyperphenylalaninemia (HPA) due to phenylketonuria (PKU). Pegvaliase (PEGylated recombinant phenylalanine ammonia lyase) is an investigational enzyme substitution therapy for the treatment of phenylketonuria (PKU). In 2016, Dimension Therapeutics, Inc. entered into a gene therapy research collaboration with the University of Pennsylvania for research and development of novel treatment for phenylketonuria.

Phenylketonuria Treatment Market Restraint

According to data published by The ZB Foundation in 2017, Phenylketonuria is a rare inherited disease that affects only about 1 in 10,000 to 15,000 newborn children. The lack of awareness about the rare disease is a major factor, which is expected to restrain growth of the market in the near future.