{"id":7101,"date":"2025-06-12T09:18:09","date_gmt":"2025-06-12T09:18:09","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/?page_id=7101"},"modified":"2026-04-15T10:52:11","modified_gmt":"2026-04-15T10:52:11","slug":"trial-results-safety","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/palynziq\/trial-results-safety\/","title":{"rendered":"Safety Data"},"content":{"rendered":"
In adults\n<\/p>\n <\/div>\n
In adolescents\n<\/p>\n <\/div>\n <\/div>\n <\/div>\n\n
Hypersensitivity reactions other than anaphylaxis were reported in 72% (204\/285) of patients. Management should be based on the severity of the reaction, recurrence, and clinical judgment, and may include dosage adjustment, temporary drug interruption, or treatment with antihistamines, antipyretics, and\/or corticosteroids.1<\/sup><\/p>\n PALYNZIQ can be resumed in patients who experience anaphylaxis\u2014as reported cases were found to be non\u00ad\u2013IgE-mediated.<\/strong>1<\/sup>*<\/p>\n Consider the risks and benefits of readministering PALYNZIQ following an episode of anaphylaxis. If the decision is made to readminister PALYNZIQ, administer the first dose under the supervision of a healthcare provider equipped to manage anaphylaxis and closely observe the patient for at least 60 minutes following the dose. Subsequent PALYNZIQ dose titration should be based on patient tolerability and therapeutic response.1<\/sup><\/p>\n *27 of 29 patients who had anaphylaxis were tested for anti\u2013pegvaliase-pqpz IgE antibodies, which recognize the PEGylated protein product. Of those 27 patients, 26 tested negative. The one patient who screened positive for anti\u2013pegvaliase-pqpz IgE had insufficient sample to confirm IgE positivity. This patient tested negative for anti\u2013pegvaliase-pqpz IgE at routine visits before and after the anaphylaxis episode.1<\/sup><\/small><\/p>\n <\/p>\n<\/div>\n Remind patients and caregivers about the importance of using aseptic injection techniques and rotating injection sites with each dose.<\/p>\n <\/p>\n Monitor blood Phe levels periodically during treatment. For Phe levels below 30 \u00b5mol\/L, dosage may be reduced and\/or dietary protein and Phe intake modified.<\/p>\n <\/p>\n <\/p>\n <\/p>\n The most common adverse reactions leading to discontinuation were hypersensitivity reactions (6%)<\/strong>, including anaphylaxis (3%)<\/strong>, angioedema (1%)<\/strong>, arthralgia (4%)<\/strong>, generalized skin reactions<\/strong> lasting at least 14 days (2%)<\/strong>, and injection site reactions (1%)<\/strong>.1<\/sup><\/p>\n <\/p>\n In Part 1 of the adolescent clinical trial, 4 patients taking PALYNZIQ (N=36) experienced one episode of anaphylaxis each, without recurrence or further complications.<\/strong>1,4 <\/sup>Treatment was discontinued due to anaphylaxis in 2 patients, while the remaining 2 were rechallenged with PALYNZIQ.1<\/sup><\/p>\n Hypersensitivity reactions other than anaphylaxis were reported in 33% (12\/36) of adolescent patients. Management should be based on the severity of the reaction, recurrence, and clinical judgment, and may include dosage adjustment, temporary drug interruption, or treatment with antihistamines, antipyretics, and\/or corticosteroids.1<\/sup><\/p>\n Remind patients and caregivers about the importance of using aseptic injection techniques and rotating injection sites with each dose.<\/p>\n Monitor blood Phe levels periodically during treatment and frequently in the pediatric population. For Phe levels below 30 \u00b5mol\/L, dosage may be reduced and\/or dietary protein and Phe intake modified.<\/p>\n <\/p>\n 6% (2\/36) of patients in the adolescent clinical trial discontinued PALYNZIQ due to an adverse reaction\u2014fewer than in the adult clinical trial.<\/strong>1<\/sup><\/p>\n <\/div>\n \n \n \n IgE, immunoglobulin E.<\/small><\/p>\n \u00a0<\/strong><\/small><\/p>\n 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> Sacharow S, Northrup H, Whitehall KB, et al. Efficacy and safety of the recommended pegvaliase dosing regimen in adults with phenylketonuria in the phase 3 PRISM studies. Presented at the International Congress of Inborn Errors of Metabolism (ICIEM); November 21-24, 2021; Sydney, Australia. 4.<\/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-7101","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nOf all patients, only 1 in 10 experienced anaphylaxis, with all episodes resolving without sequelae1<\/sup>\n<\/h2>\n \n \n \n
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<\/div>\n <\/div>\n <\/figure>\n\n Serious injection site infections, including abscess, cellulitis, necrosis, and ulcers, have been reported1<\/sup>\n<\/h2>\n
Some patients have experienced hypophenylalaninemia1<\/sup>\n<\/h2>\n
The majority of adverse reactions during the induction\/titration phase were mild to moderate in severity1,2<\/sup>\n<\/h2>\n \n \n \n
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<\/div>\n <\/div>\n <\/figure>\n\n 15% of adults (44 of 285) discontinued treatment due to an adverse event1<\/sup>\n\n<\/h2>\n
Effective management of the most common adverse reactions is possible with1<\/sup>:\n \n<\/h2>\n
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<\/div>\n <\/figure>\n\n In adults, rates of the 3 most common adverse reactions decreased from induction\/titration to maintenance\u2021<\/sup> despite increasing dose1,3<\/sup>\n<\/h2>\n \n \n \n
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<\/div>\n <\/div>\n <\/figure>\n\n <\/div>\n \n The safety profile of PALYNZIQ\u00ae<\/sup> (pegvaliase-pqpz) Injection was reinforced in adolescents aged 12-171<\/sup>\n<\/h2>\n
Serious injection site infections, including abscess, cellulitis, necrosis, and ulcers, have been reported<\/strong>1<\/sup>\n<\/h2>\n
Some patients have experienced hypophenylalaninemia<\/strong>1<\/sup>\n<\/h2>\n
Adverse reactions reported in \u226515% of adolescents taking PALYNZIQ1<\/sup>\n<\/h2>\n \n \n \n
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