JCR Pharmaceuticals Co., Ltd. announced that the US Food and Drug Administration has granted Fast Track designation for the investigational drug JR-171 for the treatment of Mucopolysaccaridosis (MPS) I (Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier (BBB)-penetrating form recombinant -L-iduronidase that was developed using JCR's proprietary J-Brain Cargo BBB technology. MPS I is a lysosomal storage disorder characterized by multiple somatic and central nervous system signs and symptoms. JR-171 is a recombinant fusion protein of an antibody against the human transferrin receptor and -L-iduronidase, the enzyme that is missing or malfunctioning in subjects with MPS I. By crossing the BBB through transferrin receptor mediated transcytosis it is expected to be effective against central nervous system (CNS) symptoms of the disease, thereby addressing a significant unmet need in the treatment of MPS I. JR-171 currently started a global Phase 1/2 clinical trial part 2 that is conducted in Japan, US and Brazil. The summary of this study is also registered on clinicaltrials.gov (Identifier NCT04453085). Granting Fast Track designation for JR-171 allows JCR to interact more frequently with the FDA. The Agency may also consider reviewing portions of the marketing application before the sponsor submits the complete application if FDA determines, after preliminary evaluation of clinical data that the fast-track product may be effective. In addition, JR-171 was designated as an orphan drug by the FDA in February and by European Commission in March of this year. Following JR-171, JCR plans to harness its J-Brain Cargo technology platform and progress its robust pipeline of innovative ERT products for other lysosomal storage disorders (LSDs). 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.