NYMC Faculty Publications

Efficacy, Safety Profile, and Immunogenicity of Alglucosidase Alfa Produced at the 4,000-Liter Scale in US Children and Adolescents with Pompe Disease: ADVANCE, A Phase IV, Open-Label, Prospective Study

DOI

10.1038/gim.2018.2

Journal Title

Genetics in Medicine

First Page

1284

Last Page

1294

Document Type

Article

Publication Date

January 2018

Department

Pediatrics

Abstract

Purpose: Pompe disease results from lysosomal acid alpha-glucosidase (GAA) deficiency and its associated glycogen accumulation and muscle damage. Alglucosidase alfa (recombinant human GAA (rhGAA)) received approval in 2006 as a treatment for Pompe disease at the 160 L production scale. In 2010, larger-scale rhGAA was approved for patients up to 8 years old without cardiomyopathy. NCT01526785 evaluated 4,000 L rhGAA efficacy/safety in US infantile- or late-onset Pompe disease (IOPD, LOPD) patients up to 1 year old transitioned from 160 L rhGAA.MethodsA total of 113 patients (87 with IOPD; 26 with LOPD) received 4,000 L rhGAA for 52 weeks dosed the same as previous 160 L rhGAA. Efficacy was calculated as the percentage of patients stable/improved at week 52 (without death, new requirement for invasive ventilation, left ventricular mass z-score increase >1 if baseline was >2, upright forced vital capacity decrease >/=15% predicted, or Gross Motor Function Measure-88 decrease >/=8 percentage points). Safety evaluation included an extension

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