The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.
This trial has completed. Results may be available via ClinicalTrials.gov or peer-reviewed publications.
PTC Therapeutics (PTCT) is the sponsoring company for this trial. BiotechSign currently grades this company D (46/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.