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Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by European Commission (EC)
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TOKYO & HYOGO, Japan--(BUSINESS WIRE)--Jun 24, 2025-- MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the European Commission (EC) has granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B), following the recent the U.S. Food and Drug Administration (FDA) designation. 1 This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250624122250/en/ MPS IIIB affects an estimated 500 to 1,000 individuals worldwide, 2 causing severe central nervous system (CNS) symptoms.