{"id":7121,"date":"2025-06-12T09:56:35","date_gmt":"2025-06-12T09:56:35","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/?page_id=7121"},"modified":"2026-04-15T10:43:48","modified_gmt":"2026-04-15T10:43:48","slug":"trial-results-design","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/trial-results-design\/","title":{"rendered":"Trial Design"},"content":{"rendered":"
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PALYNZIQ WAS STUDIED
\nACROSS PKU AND
\nPATIENT TYPES1,2<\/sup><\/span>\n<\/h1>\n <\/div>\n <\/div>\n <\/div>\n <\/div>\n <\/div>\n\n\n
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PRISM: Clinical trials in adults\n<\/p>\n <\/div>\n

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PEGASUS: Clinical trials in adolescents\n<\/p>\n <\/div>\n <\/div>\n <\/div>\n\n

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The Phase 3 PRISM trials for PALYNZIQ\u00ae<\/sup> (pegvaliase-pqpz) Injection enrolled 261 adults aged 18-55 years1,2<\/sup>*<\/span>\u2020<\/sup>\n<\/h2>\n \n \n \n
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The Phase 3 PEGASUS trials for PALYNZIQ\u00ae<\/sup> (pegvaliase-pqpz) Injection enrolled 55 adolescents aged 12-17 years1,6<\/sup>\n<\/h2>\n

Of these, 36 received PALYNZIQ in Part 1:<\/p>\n \n \n \n

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Patients taking PALYNZIQ achieved substantial Phe reduction. <\/h3>\n \n Explore the data<\/a>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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*The Phase 3 PRISM trial for PALYNZIQ enrolled 261 individuals regardless of baseline Phe or dietary Phe restriction; adherence to existing management did not exclude participation.1,2<\/sup>
\n<\/small>\u2020<\/sup>Patients aged 16-17 years were initially included in the trial prior to a protocol amendment.1,2<\/sup>
\n\u2021<\/sup>A Phe-restricted diet is defined as >75% of protein intake from medical food.1<\/sup>
\n\u00a7<\/sup>Patients were randomized 2:1 to either continue their randomized dosage of PALYNZIQ (20 mg or 40 mg once daily) or receive matching placebo.1,2<\/sup><\/small><\/p>\n

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References: 1.<\/strong> PALYNZIQ package insert. Novato, CA: BioMarin Pharmaceutical Inc; February 2026. 2.<\/strong> 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<\/em>. 2018;124(1):27-38. doi:10.1016\/j.ymgme.2018.03.006 3.<\/strong> Data on file. BioMarin Pharmaceutical Inc. 4.<\/strong> Qi Y, Patel G, Henshaw J, et al. Pharmacokinetic, pharmacodynamic, and immunogenic rationale for optimal dosing of pegvaliase, a PEGylated bacterial enzyme, in adult patients with phenylketonuria. Clin Transl Sci<\/em>. 2021;14(5):1894-1905. doi:10.1111\/cts.13043 5.<\/strong> Harding CO, Longo N, Northrup H, et al. Pegvaliase for the treatment of phenylketonuria: final results of a long-term phase 3 clinical trial program. Mol Genet Metab<\/em> Rep.<\/em> 2024;39:101084. doi:10.1016\/j.ymgmr.2024.101084 6.<\/strong> Sacharow S, Andrews A, Burrow TA, et al. Safety and efficacy of pegvaliase in adolescents with phenylketonuria: primary results from PEGASUS, a phase 3 open-label randomized controlled study. Presented at: International Congress of Inborn Errors of Metabolism (ICIEM) 2025; September 2-6, 2025; Kyoto, Japan.<\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n <\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":5,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-7121","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nPALYNZIQ\u00ae Clinical Trials | Study Design<\/title>\n<meta name=\"description\" content=\"Review study designs for both adult and adolescent trials and baseline characteristics ofeach population.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/trial-results-design\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" 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