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- Orphazyme (NASDAQ:ORPH) shares surge more than 14% premarket after the company announces successful Type A meeting with the U.S. FDA for arimoclomol for the treatment of Niemann-Pick disease type C (NPC).
- NPC is a rare, genetic, progressively debilitating, and often fatal neurodegenerative disease.
- The company's Type A meeting was earlier held in order to discuss the key topics in the Complete Response Letter (CRL) issued by the FDA in June 2021.
- FDA has recommended the company provides supplemental information and analyses, with the health regulator offering to have further interactions to identify a path to resubmission for arimoclomol in NPC.
- The EU Marketing Authorisation Application (MAA) for arimoclomol in NPC was filed with the European Medicines Agency in November 2020.