{"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":"2025-11-17T19:13:27","modified_gmt":"2025-11-17T19:13:27","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 DIET1-3*<\/sup><\/span>\n<\/h1>\n <\/div>\n <\/div>\n <\/div>\n <\/div>\n <\/div>\n\n
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PALYNZIQ®<\/sup> (pegvaliase-pqpz) Injection is proven to lower Phe levels to at or below 120 \u00b5mol\/L3<\/sup><\/h3>\n

The Phase 3 PRISM trials enrolled 261 patients to evaluate the safety and efficacy of PALYNZIQ in PKU management.1\u2020<\/sup><\/p>\n <\/div>\n\n

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Phe reduction to \u2264360 \u00b5mol\/L<\/span><\/h3>\n <\/div>\n\n
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Of the 77 patients treated for at least 36 months, 66% (51\/77) had a Phe level \u2264360 \u00b5mol\/L.1<\/sup><\/p>\n

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Time to achieve at least one Phe level \u2264360 \u00b5mol\/L for the majority of people (89\/118)\u2021<\/small><\/sup> in a subpopulation of 118 adults1,3<\/sup>:<\/span><\/h3>\n <\/div>\n\n
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Phe reduction to \u2264120 \u00b5mol\/L<\/span><\/h3>\n <\/div>\n\n
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Of the 77 patients treated for at least 36 months, 37 (48%) had a Phe level \u2264120 \u00b5mol\/L.1<\/sup>
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Time to achieve at least one Phe level \u2264120 \u00b5mol\/L for the majority of people (79\/118) in a subpopulation of 118 adults1,3\u00b6<\/sup>:<\/span><\/h3>\n <\/div>\n\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.1<\/sup>\n<\/h2>\n \t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n <\/div>\n <\/div>\n <\/div>\n\n
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Review PALYNZIQ safety data<\/a><\/p>\n <\/div>\n<\/div>\n\n

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*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><\/small>
\n\u2020<\/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><\/small>
\n\u2021<\/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><\/small>
\n\u00a7<\/sup>The majority of those patients (86\/89) achieved at least one Phe level \u2264360 \u00b5mol\/L by 36 months.3<\/sup><\/small>
\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><\/small>
\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><\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n <\/div>\n\n

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References: 1.<\/strong> PALYNZIQ [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2025. 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.<\/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":"\nBioMarin | PALYNZIQ\u00ae Trial Results<\/title>\n<meta name=\"description\" content=\"See outcomes from Phase 3 studies evaluating PALYNZIQ efficacy.\" \/>\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\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"BioMarin | PALYNZIQ\u00ae Trial 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