Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease

Derralynn Hughes; Derlis Gonzalez; Gustavo Maegawa; John A. Bernat; Myrl Holida; Pilar Giraldo; Mohamed G. Atta; Raul Chertkoff; Sari Alon; Einat Brill Almon; Rossana Rocco; Ozlem Goker-Alpan

doi:10.1016/j.gim.2023.100968

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Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease

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Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease

Derralynn Hughes, Derlis Gonzalez, Gustavo Maegawa, John A. Bernat, Myrl Holida, Pilar Giraldo, Mohamed G. Atta, Raul Chertkoff, Sari Alon, Einat Brill Almon, …

Genetics in medicine, Vol.25(12), 100968

12/2023

DOI: 10.1016/j.gim.2023.100968

PMID: 37634127

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Abstract

Purpose Fabry disease (FD) is a rare lysosomal storage disorder caused by pathogenic variants in the GLA gene encoding α-galactosidase (α-Gal-A). We evaluated long-term safety/efficacy of pegunigalsidase alfa, a novel PEGylated α-Gal-A enzyme replacement therapy (ERT) now approved for FD. Methods In a phase-1/2 dose-ranging study, 15 ERT-naïve adults with FD completed 12 months of pegunigalsidase alfa and enrolled in this 60-month, open-label, extension of 1 mg/kg pegunigalsidase alfa infusions every 2 weeks. Results 15 patients enrolled (8 males; 7 females); 10 completed ≥48 months (60 months total treatment), and 2 completed 60 months (72 months total treatment). During treatment, most treatment-emergent adverse events were mild/moderate and all infusion-related reactions were mild/moderate. Four patients were transiently positive for anti-pegunigalsidase alfa IgG. Patients showed continuous reduction in plasma lyso-Gb3 concentrations with mean (standard error [SE]) reduction of 76.1 [25.1] ng/mL from baseline to month 24. At 60 months, eGFR slope was comparable to that observed in patients treated with other ERTs. Cardiac function assessments revealed stability; no cardiac fibrosis was observed. Conclusions In this first long-term assessment of pegunigalsidase alfa administration in FD, we found favorable safety/efficacy. Our data suggest long-term continuous benefits of pegunigalsidase alfa treatment in adults with FD.

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Title: Subtitle: Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease
Creators: Derralynn Hughes
Derlis Gonzalez
Gustavo Maegawa
John A. Bernat
Myrl Holida
Pilar Giraldo
Mohamed G. Atta
Raul Chertkoff
Sari Alon
Einat Brill Almon
Rossana Rocco
Ozlem Goker-Alpan
Resource Type: Journal article
Publication Details: Genetics in medicine, Vol.25(12), 100968
DOI: 10.1016/j.gim.2023.100968
PMID: 37634127
NLM abbreviation: Genet Med
ISSN: 1098-3600
eISSN: 1530-0366
Language: English
Electronic publication date: 08/24/2023
Date published: 12/2023
Academic Unit: Stead Family Department of Pediatrics; Medical Genetics and Genomics
Record Identifier: 9984460279502771

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