JCR Pharmaceuticals' JR-441 Receives Orphan Drug Designation in Japan

MT Newswires Live

2024/12/30

JCR Pharmaceuticals Co (TYO:4552) has received orphan drug designation (ODD) from Japan's Ministry of Health, Labour and Welfare (MHLW) for JR-441, an investigational drug for treating mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo syndrome type A), according to a Wednesday filing on the Tokyo Stock Exchange.

JR-441, developed using JCR's J-Brain Cargo technology, is a blood-brain barrier-penetrating form of recombinant heparan N-sulfatase.

The drug has also received ODDs from the European Commission and the US FDA. It is currently in clinical trials in Germany and Japan. MPS IIIA is a rare, debilitating condition with no approved treatment in Japan.

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