{"id":233,"date":"2022-09-26T22:35:33","date_gmt":"2022-09-26T22:35:33","guid":{"rendered":"https:\/\/naglazymehcp-dev-001.azurewebsites.net\/en-us\/naglazyme\/?page_id=233"},"modified":"2023-05-25T15:42:43","modified_gmt":"2023-05-25T15:42:43","slug":"naglazyme-efficacy","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/naglazyme\/naglazyme-efficacy\/","title":{"rendered":"NAGLAZYME Efficacy"},"content":{"rendered":"
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Clinically significant improvements with NAGLAZYME\u00ae<\/sup> (galsulfase) therapy<\/strong><\/h1>\n

Phase 3 Pivotal Trial<\/h2>\n

In the randomized, double\u2010blind, multicenter, placebo\u2010controlled clinical trial, 39 patients with MPS VI received 1 mg\/kg NAGLAZYME\u00ae<\/sup> (galsulfase) or placebo once\u2010weekly for 24 weeks. Patients were then enrolled in a 72-week open-label extension study, for a total of up to 96 weeks. Enrollment was restricted to patients with a 12\u2010minute walk distance of 5 to 400 meters. All patients were treated with antihistamines prior to each infusion. Results from the 10-year Resurvey Study, which evaluated patients who were involved in the initial Survey Study, are consistent with the phase 3 trial results.1-3<\/sup><\/p>\n

NAGLAZYME improved endurance as shown by the 12\u2010minute walk test (12MWT) 1,2,4<\/sup><\/h3>\n

12-minute walk test: mean meters walked1,2,4 a,b<\/sup><\/strong><\/h4>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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\n \"12MWD <\/div>\n

a <\/sup>Fitted values.1<\/span><\/sup><\/small>
\nb <\/sup>The difference between the NAGLAZYME and placebo groups at baseline was statistically significant (P<\/em>=0.014).1<\/sup><\/small>
\nc<\/sup> By week 96, patients in the drug group had been taking NAGLAZYME for 96 weeks; patients in the placebo group had only been taking NAGLAZYME for the 72-week open-label extension period.2,4<\/sup><\/small>
\nd<\/sup> One patient in the placebo group left the study for reasons unrelated to treatment. Patients receiving placebo were switched to NAGLAZYME in the trial extension period.1,2<\/sup><\/small>
\ne<\/sup> One patient failed to complete the assessment.2<\/sup><\/small><\/p>\n<\/figcaption>\n <\/figure>\n\n

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Week 24<\/strong>: Patients treated with NAGLAZYME demonstrated statistically and clinically significant improvement in endurance compared to placebo as measured by a 12MWT at week 24.1<\/sup><\/p>\n

Week 96<\/strong>: In the group originally treated with NAGLAZYME, improvement in the 12MWT was sustained through week 96.2<\/sup><\/p>\n