JCR Pharmaceuticals Co., Ltd. announced that the U.S. Food and Drug Administration ("FDA") has granted Rare Pediatric Disease Designation (RPDD) for JR-141 (INN: pabinafusp alfa) for the treatment of Mucopolysaccharidosis type II (MPS II also known as Hunter syndrome) on December 16, 2022. JR-141 is a blood brain-barrier (BBB)-penetrating recombinant Iduronate-2-sulfatase, linked to J- Brain Cargo® BBB penetration technology. The sponsor of a marketing application of a rare pediatric disease designated product may become eligible to receive a voucher for a priority review of a subsequent marketing application in the U.S. In May 2021, JCR started marketing of JR-141 under the brand name "IZCARGO® I.V. infusion 10mg" in Japan.

JCR is currently conducting a global phase 3 clinical trial of JR-141 in the U.S., Brazil and Europe. In September 2021, JCR and Takeda announced a geographically-focused exclusive collaboration and license agreement in which Takeda will exclusively commercialize JR- 141 outside of the United States, including Canada, Europe, and other regions (excluding Japan and certain other Asia-Pacific countries. Following JR-141, JCR plans to harness its J-Brain Cargo® technology platform and progress its robust pipeline of innovative enzyme replacement therapies (ERTs) for other lysosomal storage disorders.

JCR, as a specialty pharma in the rare disease arena, will continue to proactively engage in research and development of transformative treatment options for patients with rare diseases. This designation is expected to have a minor impact on consolidated financial results for this fiscal year ending on March 31, 2023.