News Release 28/2/2019- Patient enrollment in Phase 2 trial in Brazil completed
JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today the orphan designation of JR-141 for Hunter Syndrome being granted by European Medicines Agency (EMA). JR-141 is a blood-brain-barrier (BBB)-penetrating recombinant iduronate-2-sulfatase product candidate for the treatment of patients with Hunter syndrome, to which J-Brain Cargo®, JCR’s proprietary BBB technology, is applied.
JR-141 was designated under the SAKIGAKE Designation System in Japan by the Ministry of Health, Labour and Welfare in March 2018, and received the orphan drug designation from the US Food Drug Administration in October 2018. Currently, the drug is undergoing a Phase 3 clinical trial so that an application for its marketing approval can be filed in 2020. As an initial step in the global development of JR-141, JCR initiated a Phase 2 clinical trial in Brazil in June 2018, whilst development strategies for other areas are being planned as well.
Following JR-141, JCR harnesses its J-Brain Cargo® technology platform to develop a robust pipeline of innovative enzyme replacement therapy products for other lysosomal storage disorders. JCR, as a pharma dedicated to specialty care, will continue to proactively engage in research and development of treatment options for the patients with rare diseases.
This designation is expected to have a minor impact on JCR’s consolidated financial results for the year ending on March 31, 2019.
Orphan designation (EMA)
The EMA implements orphan designation for promoting new drug development for rare diseases in which the prevalence of the condition in the EU must not be more than 5 in 10,000. Designated drugs are granted market exclusivity for ten years in the EU, as well as scientific guidelines.
[About JCR Pharmaceuticals]
JCR is a specialty pharma engaged in the research, development, manufacture and marketing of biopharmaceuticals and regenerative medicine with a focus on rare diseases. Its philosophy, “Contributing towards people’s healthcare through pharmaceutical products” drives JCR to create innovative pharmaceutical products as value-added treatment options for the under-served patient community.
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JCR Receives EMA Orphan Designation for JR-141 in Hunter Syndrome
February 28, 2019 JCR Pharmaceuticals Co., Ltd.
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