{"id":2675,"date":"2022-09-02T11:59:32","date_gmt":"2022-09-02T11:59:32","guid":{"rendered":"https:\/\/palynziqhcp-dev-001.azurewebsites.net\/en-us\/palynziq\/?page_id=2675"},"modified":"2024-06-12T13:38:39","modified_gmt":"2024-06-12T13:38:39","slug":"palynziq-trial-results","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/palynziq-trial-results\/","title":{"rendered":"Efficacy Data"},"content":{"rendered":"<div id=\"acf-block-63ff5c5227501\" class=\"wrapper hero-wrapper\">\n    <div class=\"hero hero-no-overlay\">\n                    <div class=\"hero-background-image hero-background-image-desktop\" style=\"background-image: url(https:\/\/hcp.biomarin.com\/en-us\/palynziq\/wp-content\/uploads\/sites\/3\/2022\/09\/stars_bg-2000x394.jpg);\"><\/div>\n            <div class=\"hero-background-image hero-background-image-mobile\" style=\"background-image: url(https:\/\/hcp.biomarin.com\/en-us\/palynziq\/wp-content\/uploads\/sites\/3\/2022\/09\/stars_bg-2000x394.jpg);\"><\/div>\n                <div class=\"overlay\"><\/div>\n        <div class=\"wrapper\">\n            <div class=\"inner-wrapper\">\n                <div class=\"hero-content\">\n                                                                                    <h1>Efficacy Data\n<\/h1>\n                                                                                                <\/div>\n            <\/div>\n        <\/div>\n    <\/div>\n    <\/div>\n\n<div id=\"acf-block-63ff5c5227550\" class=\"block wrapped-content block-tight-top block-tight-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-63ff5c5227569\" class=\"block-wysiwyg\">\n            <h3>In clinical trials, 75% of patients in the study achieved blood Phe levels \u2264360 \u00b5mol\/L<sup>1,2<\/sup><\/h3>\n    <\/div>\n\n<figure id=\"acf-block-63ff5c5227583\">\n    <div class=\"image\">\n                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/wp-content\/uploads\/sites\/3\/2023\/03\/2022-10-20-at-18.27.57-opt.png?v=0.77\" alt=\"\" \/>            <\/div>\n    <\/figure>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-63ff5c5227596\" class=\"block wrapped-content block-tight-top block-tight-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-63ff5c52275a9\" class=\"block-wysiwyg\">\n            <h3>The majority of patients achieved blood Phe levels \u2264360 \u00b5mol\/L and most achieved this Phe level with the recommended dosing schedule<sup>1,2<\/sup><\/h3>\n    <\/div>\n\n<figure id=\"acf-block-63ff5c52275bd\">\n    <div class=\"image\">\n                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/wp-content\/uploads\/sites\/3\/2023\/03\/2022-10-20-at-18.27.45-opt.png?v=0.77\" alt=\"\" \/>            <\/div>\n    <\/figure>\n\n<div id=\"acf-block-63ff5c52275ce\" class=\"block-wysiwyg\">\n            <p>You can view the dosing schedule <a href=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/dosing-administration\/\">here<\/a>.<\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-63ff5c52275e4\" class=\"block toggled-content block-tight-top block-tight-bottom\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"toggled-content-item\">\n\t\t\t\t<div class=\"toggled-content-title\">\n\t\t\t\t\t<div class=\"toggled-content-trigger\">\n\t\t\t\t\t\t<span><\/span>\n\t\t\t\t\t\t<span><\/span>\n\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t    <h2>Learn about patients achieving \u2264360 \u00b5mol\/L by dose <sup>1,2<\/sup>\n<\/h2>\n\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<div class=\"toggled-content-expanded\">\n                    \n<div id=\"acf-block-63ff5c52275f9\" class=\"block-wysiwyg\">\n            <h3>Patients who achieved \u2264360 \u00b5mol\/L by dose<sup>1,2<\/sup><\/h3>\n<p>The chart below shows the percentage of patients who achieved \u2264360 \u00b5mol\/L at each maintenance dose*<\/p>\n    <\/div>\n\n<figure id=\"acf-block-63ff5c5227614\">\n    <div class=\"image image-responsive\">\n                    <div class=\"image-desktop\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/wp-content\/uploads\/sites\/3\/2022\/08\/Graphic-2.1.7-Desktop.svg\" alt=\"Chart showing the percentage of patients who achieved \u2264360 \u00b5mol\/L at each maintenance dose\" \/>\t\t\t<\/div>\n\t\t\t<div class=\"image-mobile\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/wp-content\/uploads\/sites\/3\/2022\/09\/Graphic-2.1.7-Mobile.svg\" alt=\"Chart showing the percentage of patients who achieved \u2264360 \u00b5mol\/L at each maintenance dose\" \/>\t\t\t<\/div>\n            <\/div>\n            <figcaption><p class=\"footnote reference re-mt-20 re-mb-0\">*118 patients with pre-treatment baseline blood Phe levels greater than 600 \u03bcmol\/L were randomized to and received at least one dose of 20 mg once-daily PALYNZIQ<sup>\u00ae<\/sup> (pegvaliase-pqpz) Injection.<\/p>\n<p class=\"dagger reference mb-0\">\u2020Did not achieve \u2264360 \u00b5mol\/L before study completion.<\/p>\n<\/figcaption>\n    <\/figure>\n\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-63ff5c522762b\" class=\"block wrapped-content block-tight-top block-tight-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-63ff5c522763b\" class=\"block wrapped-content block-tight-top block-tight-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-63ff5c522764b\" class=\"block-wysiwyg\">\n            <h3>A cross-sectional analysis showed that more patients had blood Phe levels within or below ACMG recommendations at each time point up to 24 months<sup>1,3<\/sup><\/h3>\n    <\/div>\n\n<figure id=\"acf-block-63ff5c522765e\">\n    <div class=\"image\">\n                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/wp-content\/uploads\/sites\/3\/2023\/03\/2022-10-20-at-18.27.34-opt.png?v=0.77\" alt=\"\" \/>            <\/div>\n    <\/figure>\n\n<figure id=\"acf-block-63ff5c522766f\">\n    <div class=\"image image-responsive\">\n                    <div class=\"image-desktop\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/wp-content\/uploads\/sites\/3\/2022\/08\/Graphic-2.1.4-Desktop-1.svg\" alt=\"Blood phe levels graphic\" \/>\t\t\t<\/div>\n\t\t\t<div class=\"image-mobile\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/wp-content\/uploads\/sites\/3\/2022\/09\/Graphic-2.1.4-Mobile.svg\" alt=\"\" \/>\t\t\t<\/div>\n            <\/div>\n    <\/figure>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-63ff5c522767f\" class=\"block wrapped-content block-tight-top block-tight-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-63ff5c522769a\" class=\"block-wysiwyg\">\n            <ul>\n<li>If patients experience blood Phe levels \u226430 \u00b5mol\/L (0.5 mg\/dL), reduce dosage of PALYNZIQ and\/or modify dietary Phe intake to keep levels within a clinically acceptable range and above 30 \u00b5mol\/L (0.5 mg\/dL)<sup>1<\/sup>\n<ul>\n<li>In clinical trials, it was recommended that patients not change their dietary Phe intake unless Phe levels decreased to \u226430 \u00b5mol\/L<sup>4<\/sup><\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<p><small>ACMG, American College of Medical Genetics and Genomics.<br \/>\n<\/small><small>*Changes in sample size are due to patients\u2019 Phe levels not being collected at all time points and subjects terminating from study early.<sup>2<\/sup><\/small><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-63ff5c52276c1\" class=\"block toggled-content block-tight-top block-tight-bottom\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"toggled-content-item\">\n\t\t\t\t<div class=\"toggled-content-title\">\n\t\t\t\t\t<div class=\"toggled-content-trigger\">\n\t\t\t\t\t\t<span><\/span>\n\t\t\t\t\t\t<span><\/span>\n\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t    <h2>View visual percentage of patients reaching meaningful blood Phe targets up to 36 months\n<\/h2>\n\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<div class=\"toggled-content-expanded\">\n                    \n<div id=\"acf-block-63ff5c52276d5\" class=\"block-wysiwyg\">\n            <h3>With PALYNZIQ, patients are able to reach meaningful blood Phe targets<sup>1<\/sup><\/h3>\n    <\/div>\n\n<figure id=\"acf-block-63ff5c52276e8\">\n    <div class=\"image\">\n                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/wp-content\/uploads\/sites\/3\/2022\/10\/Screenshot-2022-10-20-at-18.35.29.png?v=0.77\" alt=\"A cross-sectional analysis showing percentage of patients at or below blood Phe thresholds at specific time points (N=118) graph\" \/>            <\/div>\n    <\/figure>\n\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-63ff5c52276f7\" class=\"block wrapped-content block-tight-top block-tight-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-63ff5c5227719\" class=\"block-wysiwyg\">\n            <ul>\n<li>66% (57 of 86) of patients achieved levels at or below ACMG guidelines (\u2264360 \u00b5mol\/L) at 2 years of treatment<sup>1<\/sup><\/li>\n<li>The percentage of patients reaching a measurement of \u2264360 \u00b5mol\/L increased at each assessment point up to the 24-month mark and sustained at the 36-month mark<sup>1<\/sup><\/li>\n<li>In clinical trials, 15% of patients (44 of 285) discontinued treatment due to an adverse event. The most common adverse reactions leading to treatment discontinuation were hypersensitivity reactions (6%), including anaphylaxis (3%), angioedema (1%), arthralgia (4%), generalized skin reactions lasting at least 14 days (2%), and ISRs (1%)<sup>1<\/sup><\/li>\n<\/ul>\n<p><small>*Patients who remained in the trial.<sup>1<\/sup><\/small><\/p>\n<p><small><sup>\u00a0<\/sup><\/small><\/p>\n<h3><a href=\"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/side-effects-and-safety-profile\/\">NEXT: Safety Profile &gt;<\/a><\/h3>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-63ff5c522774e\" class=\"block references\">\n    <div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t    \t\t\t    <h4>References:\n<\/h4>\n\t\t\t\t\t\t                <ol>\n                                                                                                                        <li><span>PALYNZIQ [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2020. \n<\/span><\/li>\n                                                                                                                                                <li><span>Data on file. BioMarin Pharmaceutical Inc. \n<\/span><\/li>\n                                                                                                                                                <li><span>Vockley J, Andersson HC, Antshel KM, <em>et al<\/em>; for the American College of Medical Genetics and Genomics Therapeutic Committee. Phenylalanine hydroxylase deficiency: diagnosis and management guideline. <em>Genet Med<\/em>. 2014;16(2):188-200. \n<\/span><\/li>\n                                                                                                                                                <li><span>Thomas J, Levy H, Amato S, <em>et al<\/em>, for the PRISM investigators. Pegvaliase for the treatment of phenylketonuria: results of a long-term phase 3 clinical trial program (PRISM). <em>Mol Genet Metab<\/em>. 2018;124(1):27-38.\n<\/span><\/li>\n                                                            <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>\n\n","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v23.1 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>Efficacy Data | Palynziq HCP<\/title>\n<meta name=\"description\" content=\"In clinical trials, 75% of patients in the study achieved blood Phe levels \u2264360 \u00b5mol\/L1,2\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link 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