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.
免责声明：投资有风险，本文并非投资建议，以上内容不应被视为任何金融产品的购买或出售要约、建议或邀请，作者或其他用户的任何相关讨论、评论或帖子也不应被视为此类内容。本文仅供一般参考，不考虑您的个人投资目标、财务状况或需求。TTM对信息的准确性和完整性不承担任何责任或保证，投资者应自行研究并在投资前寻求专业建议。