Orphazyme provides regulatory update following Type A meeting with FDA on arimoclomol in Niemann-Pick disease type C
Retrieved on:
Sunday, October 31, 2021
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We are pleased to have gained a greater understanding from the FDA on the information that could address topics in the CRL.
Key Points:
- We are pleased to have gained a greater understanding from the FDA on the information that could address topics in the CRL.
- Niemann-Pick disease type C (NPC) is a rare, genetic, progressively debilitating, and often fatal neurodegenerative disease.
- On June 17, 2021, Orphazyme received a Complete Response Letter from the FDA regarding its New Drug Application for arimoclomol for the treatment of NPC.
- Orphazyme is a late-stage biopharmaceutical company developing arimoclomol for Niemann-Pick disease type C (NPC).