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JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that European Medicines Agency (EMA) has granted orphan drug designation to JR-171, an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier (BBB)-penetrating form recombinant α-L-iduronidase that was developed using JCR’s proprietary J-Brain Cargo® BBB technology.
JR-171 received the orphan drug designation from the US Food Drug Administration (FDA) in February 2021. Currently JR-171 is undergoing a global Phase 1/2 clinical trial and the first patient in Japan was dosed in October, 2020 and in Brazil in March, 2021. The trial is also scheduled for enrolling patients in the US. The summary of this study is registered on clinicaltrials.gov.
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. Therefore, following JR-171, JCR plans to harness its J-Brain Cargo® technology platform and progress its robust pipeline of innovative enzyme replacement therapy (ERT) products for additional lysosomal storage disorders (LSDs).
This designation is expected to have a minor impact on JCR’s consolidated financial results for the year ending on March 31, 2021.
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 guidance.
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