Abstract
Most drugs cannot be delivered to the brain from the systemic circulation due to the presence of the blood-brain barrier, and therefore it is quite challenging to develop therapeutics addressing central nervous system disorders. IZCARGOⓇ（general name: pabinafusp alfa） is the world’s first biological drug applied with the brain drug delivery technology J-Brain CargoⓇ, which utilizes the transferrin receptor-mediated Fe/transferrin transport to the brain, and was launched in Japan in May 2021 to treat patients with all forms of MPS II. The drug is a fusion protein consisting of anti-human transferrin receptor antibody and iduronate-2-sulfatase and penetrates the blood-brain barrier into the brain parenchyma where it exerts pharmacological effects. J-Brain CargoⓇ is a fundamental technology for drug delivery to the central nervous system, and its application is expected to be expanded to various diseases.
