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)
Active, no longer recruiting
Phase of Trial: Phase III
Latest Information Update: 14 Sep 2017
At a glance
- Drugs Pegvaliase (Primary)
- Indications Phenylketonuria
- Focus Adverse reactions; Registrational
- Acronyms PRISM-2; Prism302
- Sponsors BioMarin Pharmaceutical
- 14 Sep 2017 According to a BioMarin Pharmaceutical media release, the US FDA has provided the company with the "Day-74" filing communication for its Biologics License Application for pegvaliase to reduce blood phenylalanine levels in adult patients with phenylketonuria who have uncontrolled blood phenylalanine levels on existing management. The FDA advised that, it is not planning to hold an advisory committee meeting for the review of the application.
- 05 Sep 2017 According to a BioMarin Pharmaceutical media release, data will be presented at the 13th International Congress of Inborn Errors of Metabolism (ICIEM) 2017.
- 29 Aug 2017 According to a BioMarin Pharmaceutical media release, the US FDA accepted for Priority Review the BLA for pegvaliase to reduce blood phenylalanine (Phe) levels in adult patients with phenylketonuria (PKU) who have uncontrolled blood Phe levels on existing management. The PDUFA action date is February 28, 2018.
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Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History