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EMA Grants Orphan Drug Designation to JR-171 for the Treatment of Mucopolysaccaridosis Type I (MPS I)
JCR Pharmaceuticals Co., Ltd. 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.
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