{"id":7145,"date":"2025-06-12T10:40:45","date_gmt":"2025-06-12T10:40:45","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/?page_id=7145"},"modified":"2026-04-15T10:44:58","modified_gmt":"2026-04-15T10:44:58","slug":"trial-results-efficacy","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/trial-results-efficacy\/","title":{"rendered":"Efficacy Data"},"content":{"rendered":"
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SUBSTANTIAL
\nPHE REDUCTION,
\nREGARDLESS OF
\nBASELINE DIET1,2<\/sup>*<\/span><\/span>\n<\/h1>\n <\/div>\n <\/div>\n <\/div>\n <\/div>\n <\/div>\n\n\n
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In adults\n<\/p>\n <\/div>\n

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In adolescents\n<\/p>\n <\/div>\n <\/div>\n <\/div>\n\n

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PALYNZIQ\u00ae<\/sup> (pegvaliase-pqpz) Injection is proven to lower Phe levels to \u2264120 \u00b5mol\/L1<\/sup>\n<\/h2>\n

The Phase 3 PRISM trials enrolled 261 adults aged 18-55 years to evaluate the safety and efficacy of PALYNZIQ in PKU management.1\u2020\u2021<\/small><\/sup><\/p>\n \n \n \n

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Some PKU patients treated with PALYNZIQ have experienced hypophenylalaninemia. Monitor blood Phe levels periodically during treatment.1<\/sup><\/p>\n

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>8 out of 10 patients in the phase 3 PRISM studies were not on a Phe-restricted diet (>75% of protein intake from medical food) at baseline.<\/strong>1<\/sup><\/p>\n <\/div>\n <\/div>\n \n

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Adolescents taking PALYNZIQ\u00ae<\/sup> (pegvaliase-pqpz) Injection achieved Phe levels \u2264120 \u00b5mol\/L3<\/sup>\n<\/h2>\n

At 17 months, the end of the Part 1 primary treatment phase, the mean Phe reduction in adolescents aged 12-17 taking PALYNZIQ was 473 \u00b5mol\/L<\/strong> vs 19 \u00b5mol\/L in the diet-only arm.1<\/sup><\/p>\n \n \n \n

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Limitation: These are exploratory endpoints from an ongoing study and results should be interpreted with caution.<\/strong><\/p>\n

Some PKU patients treat with PALYNZIQ have experienced hypophenylalaninemia. Monitor blood Phe levels periodically during treatment.1<\/sup><\/p>\n \n \n \n

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PALYNZIQ has a well-established safety profile in adults that has been reinforced in adolescents.<\/h3>\n \n See the safety data<\/a>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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\u00a0<\/small><\/p>\n

*Monitor patients\u2019 dietary protein and phenylalanine intake throughout treatment with PALYNZIQ and counsel them on how to adjust their dietary intake, as needed, based on blood phenylalanine concentrations.1<\/sup>
\n\u2020<\/sup>Patients aged 16-17 years were initially included in the trial prior to a protocol amendment.1,4<\/sup>
\n\u2021<\/sup>Of the 261 patients who enrolled in Study 301, 54 (21%) patients discontinued treatment during Study 301, 4 patients completed Study 301 and did not continue to Study 165-302 (referred to as Study 302, NCT01889862), 152 patients continued to the eligibility period of Study 302, and 51 patients continued directly from Study 301 into the long-term treatment period of Study 302.1<\/sup>
\n\u00a7<\/sup>Of 118 patients from Study 301 with a pre-treatment baseline blood phenylalanine concentration greater than 600 micromol\/L who were randomized to and received at least one dose of 20 mg once daily. 21% (25\/118) of patients discontinued the study prior to achieving \u2264360 \u00b5mol\/L. Of those, 10 patients discontinued due to adverse events. 3% (4\/118) did not achieve \u2264360 \u00b5mol\/L before study completion.1,3<\/sup>
\n||<\/sup>The majority of those patients (86\/89) achieved at least one Phe level \u2264360 \u00b5mol\/L by 36 months.3<\/sup>
\n\u00b6<\/sup>Of 118 patients from Study 301 with a pre-treatment baseline blood phenylalanine concentration greater than 600 micromol\/L who were randomized to and received at least one dose of 20 mg once daily. 24% (28\/118) of patients discontinued the study prior to achieving \u2264120 \u00b5mol\/L. 11 patients did not achieve \u2264120 \u00b5mol\/L before study completion.1,3<\/sup>
\n#<\/sup>The 25th percentile of patients who achieved \u2264120 \u00b5mol\/L did so by 7 months; the 75th percentile, by 18 months.3<\/sup>
\n**Results are from an interim analysis using data collected through October 2025.<\/small><\/p>\n

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References: 1.<\/strong> PALYNZIQ package insert. Novato, CA: BioMarin Pharmaceutical Inc; February 2026. 2.<\/strong> Burton B, Sacharow S, Northrup H, et al. Efficacy and safety of the recommended pegvaliase dosing regimen in adults with phenylketonuria in the phase 3 PRISM studies. Poster presented at: SSIEM Annual Symposium; August 30-September 2, 2022; Freiburg, Germany. 3.<\/strong> Data on file. BioMarin Pharmaceutical Inc. 4.<\/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 5.<\/strong> Beghini M, Pichler M, Tinnefeld FC, et al. Poor adherence during adolescence is a risk factor for becoming lost to follow-up in patients with phenylketonuria. Mol Genet Metab Rep<\/em>. 2024;39:101087. doi:10.1016\/j.ymgmr.2024.101087 6.<\/strong> Ashe K, Kelso W, Farrand S, et al. Psychiatric and cognitive aspects of phenylketonuria: the limitations of diet and promise of new treatments. Front Psychiatry<\/em>. 2019;10:561. doi:10.3389\/fpsyt.2019.00561 7.<\/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-7145","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nPALYNZIQ\u00ae Efficacy | Phe Reduction Data<\/title>\n<meta name=\"description\" content=\"See how PALYNZIQ\u00ae reduces phenylalanine levels in adults and adolescents with PKU,regardless of diet.\" \/>\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-efficacy\/\" \/>\n<meta property=\"og:locale\" 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