name: | PabinafuspAlfa |
ATC code: | A16AB27 | route: | intravenous |
n-compartments | 2 |
Pabinafusp alfa is a recombinant fusion protein consisting of human iduronate-2-sulfatase (IDS) coupled with an antibody targeting the human transferrin receptor. It is designed to treat mucopolysaccharidosis type II (MPS II, Hunter syndrome), a lysosomal storage disorder, by facilitating IDS delivery across the blood-brain barrier. Pabinafusp alfa is approved in Japan for the treatment of MPS II.
Pharmacokinetic parameters estimated for typical pediatric patients (children with MPS II) following intravenous infusion. No published peer-reviewed PK studies found as of June 2024.