Pharmaceutical Industry Reports Phenylketonuria Treatment | Page 2

REPORT DESCRIPTION
Phenylketonuria Treatment Market – Overview
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.
Phenylketonuria (PKU) is a rare genetic disorder caused due to increased level of phenylalanine in the blood. The
excess amount of phenylalanine can cause intellectual disability and other serious health problems. The most severe
condition of this disorder is termed as classic PKU and symptoms related to it are seizures, tremors, or trembling and
shaking, stunted growth, hyperactivity, skin conditions such as eczema, and musty odor of their breath, skin, or
urine. PKU can be managed by maintaining phenylalanine free diet.
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