An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40 mg/Day Dose) in Adults With Phenylketonuria
Status: Active, no longer recruiting
Phase of Trial: Phase III
Latest Information Update: 23 Oct 2019
Price : $35 *
At a glance
- Drugs Pegvaliase (Primary)
- Indications Phenylketonuria
- Focus Adverse reactions; Therapeutic Use
- Sponsors BioMarin Pharmaceutical
- 23 Oct 2019 According to a BioMarin Pharmaceutical media release, On May 6, 2019, the European Commission (EC) granted marketing authorization for Palynziq at doses of up to 60 milligrams once daily, to reduce blood Phe concentrations in patients with 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.
- 01 Mar 2019 According to a BioMarin Pharmaceutical media release, CHMP of EMA adopted a positive opinion on MAA, recommending the authorization of pegvaliase, to reduce blood phenylalanine concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control despite prior management with available treatment options.The CHMP based its opinion on the totality of data from the Palynziq clinical development program including this and PRISM-2 study.
- 10 Oct 2018 New trial record