Orphazyme, a biotech company based in Denmark, announced today (9/16) that the FDA has accepted its New Drug Application (NDA) for treatment of Niemann-Pick disease Type C. The genetic disease is both rare as well as fatal, with no currently approved treatments. Arimoclomol, the drug developed to treat the rare disease, has already received Fast Track Designation by the FDA for ALS. It is not set to be reviewed for March 2021.
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More on: News Regulatory
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