The largest clinical trial program ever conducted in adult patients with PKU1
More than 350 patients over 10 years participated in the extensive clinical trial program that established efficacy, safety, and dosing regimen of PALYNZIQ® (pegvaliase-pqpz) Injection.1,2
PRISM-1 (Study 301): Treatment induction, titration, and maintenance study (N=261)1,3
PRISM-2 (Study 302): Pivotal efficacy study of PALYNZIQ1,3,4
Patients were randomized 2:1 to either continue their randomized dosage of PALYNZIQ (20 mg or 40 mg once daily) or receive matching placebo.
Patient characteristics at baseline
Baseline patient demographics included patients with significantly elevated blood Phe levels >1200 µmol/L (20 mg/dL).3
Patient demographics and characteristics at treatment-naïve baseline (ITT population: N=261)1,3
The majority of patients were not on a Phe-restricted diet* prior to and during the study.3
- A Phe-restricted diet is not required in conjunction with PALYNZIQ3
- At baseline, 57% of patients were taking medical food (149 of 261) and 16% were on a Phe-restricted
diet (41 of 261)3
A Phe-restricted diet is defined as >75% of protein intake from medical food.2
The US recommendation for total protein intake from natural food is
References: 1. Thomas J, Levy H, Amato S, et al, for the PRISM investigators. Pegvaliase for the treatment of phenylketonuria: results of a long-term phase 3 clinical trial program (PRISM). Mol Genet Metab. 2018;124(1):27-38. 2. BioMarin Pharmaceutical. Dose-finding study to evaluate the safety, efficacy, & tolerability of multiple doses of rAvPAL-PEG in subjects with PKU. Available from: https://clinicaltrials.gov/ct2/show/NCT00925054?term=pegvaliase&cond=PKU&draw=4&rank=5. NLM identifier: NCT00925054. Accessed February 12, 2021. 3. PALYNZIQ [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2020. 4. Singh RH, Cunningham AC, Mofidi S, et al. Updated, web-based nutrition management guideline for PKU: an evidence and consensus based approach. Mol Genet Metab. 2016;118(2):72-83.