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A Four-Part, Phase 3, Randomized, Double-Blind, Placebo- Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self-Administered by Adults With Phenylketonuria (PKU)

Trial Profile

A Four-Part, Phase 3, Randomized, Double-Blind, Placebo- Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self-Administered by Adults With Phenylketonuria (PKU)

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 23 Oct 2019

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At a glance

  • Drugs Pegvaliase (Primary)
  • Indications Phenylketonuria
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Acronyms PRISM-2; Prism302
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 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.
    • 18 Jun 2019 Status changed from active, no longer recruiting to completed.
    • 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 aged 16 and older, who have inadequate blood Phe control despite prior management with available treatment options.The CHMP based its opinion on data from the Palynziq clinical development programincluding this and ongoing open-label extension study (NCT036943).
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