Seven-year safety and efficacy with velaglucerase alfa for treatment-naive adult patients with type 1 Gaucher disease.

ABSTRACT: Velaglucerase alfa is a human β-glucocerebrosidase approved for Gaucher disease type 1 (GD1) treatment. This report summarizes the 7-year experience of the now-completed phase I/II and extension studies of adult GD1 patients who received velaglucerase alfa. Ten patients who completed the 9-month, phase I/II study entered the extension trial TKT025EXT, of which eight completed this study. Doses were reduced after a cumulative treatment period of 15 to 18 months. Although all patients experienced ≥1 adverse event, no patient withdrew due to a drug-related adverse event or required premedication. No patient developed anti-drug antibodies, compliance remained high (median 98%), and seven of eight eligible patients transitioned to home infusions under supervision by healthcare professionals. St

SUBMITTER: Zimran A 

PROVIDER: S-EPMC5033020 | biostudies-literature | 2015 Jul

REPOSITORIES: biostudies-literature