pegunigalsidase alfa for treating Fabry disease

Last edited 10/2023 and last reviewed 11/2023

NICE state (1):

• pegunigalsidase alfa is recommended, within its marketing authorisation, as an option for treating Fabry disease (also known as alpha-galactosidase deficiency) in adults. It is recommended only if the company provides it according to the commercial arrangement
• NICE committee stated "..Clinical trial evidence shows that pegunigalsidase alfa works as well as agalsidase beta. There is no direct clinical trial evidence comparing pegunigalsidase alfa with agalsidase alfa or migalastat. But, clinical experts advised that pegunigalsidase alfa is also likely to work as well as these 2 treatments.."

Notes:

• pegunigalsidase alfa is a novel PEGylated, covalently crosslinked form of alpha-galactosidase A developed as enzyme replacement therapy (ERT) for Fabry disease (FD)
  • designed to increase plasma half-life and reduce immunogenicity
  • study evidence showed unique pharmacokinetics and the apparent attenuated immune response demonstrated by pegunigalsidase alfa may improve the safety and clinical response to this ERT in patients with FD (2)
    • is supported by the favorable AE profile, stability of renal function and cardiac parameters, and improved disease symptoms observed in the study participants during the 12-month treatment period

Reference:

1. NICE (October 2023). Pegunigalsidase alfa for treating Fabry disease
2. Schiffmann R et al. Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial. J Inherit Metab Dis. 2019 May;42(3):534-544.