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European Commission Approves Palynziq® (pegvaliase injection) for Treatment of Phenylketonuria (PKU) in Patients Aged 16 Years or Older
SAN RAFAEL, Calif., May 6, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the European Commission (EC) has granted
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"SAN RAFAEL, Calif., May 6, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the European Commission (EC) has granted marketing authorization for Palynziq® (pegvaliase injection) at doses of up to 60 mg once daily, to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first enzyme substitution therapy approved in Europe to target the underlying cause of PKU by helping the body to break down Phe. In addition, the EC acknowledged that the Phase 3 trial and extension study is suggestive of an improvement in inattention and mood symptoms.\n\n \nOn March 1, 2019 BioMarin announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), adopted a positive opinion for the company's Marketing Authorization Application (MAA) for Palynziq. This is BioMarin's second approved treatment for this rare genetic disease.\nPKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain and is marked by an inability to break down Phe, an amino acid that is found in most forms of protein. PKU affects approximately 50,000 diagnosed patients in the developed world, and in Europe, approximately 1 in every 10,000 newborn babies are affected by this disease.I Approximately 18,000 patients aged 16 and older are affected by PKU in Europe and the Middle East. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychiatric-related issues, affecting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to EU treatment guidelines, PKU patients should maintain lifelong control of their Phe levels. \n\"The approval of Palynziq is the latest milestone after more than 15 years of an ongoing commitment to the PKU community. BioMarin has brought the only two approved therapies for PKU to patients...