KemPharm Announces Strategic Acquisition of Arimoclomol from Orphazyme, Expanding its Rare CNS Diseases Pipeline

KemPharm Announces Strategic Acquisition of Arimoclomol from Orphazyme, Expanding its Rare CNS Diseases Pipeline

By, Admin 16 May 2022

Arimoclomol is an NDA-stage, revenue-generating investigational drug candidate being developed for the treatment of Niemann-Pick disease type C (NPC), a rare progressive neurodegenerative disease KemPharm plans to refile the New Drug Application (NDA) for arimoclomol in NPC with the U.S. Food and Drug Administration (FDA) as early as the First Quarter of 2023 Conference call and live audio webcast with slide presentation is scheduled for tomorrow, May 16, 2022, at 8:30 a.m., EDT CELEBRATION, Fla., May 15, 2022 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system (CNS) diseases, today announced a definitive agreement with Orphazyme A/S (in reconstruction) (ORPHA.CO; ORPH) (Orphazyme) to acquire arimoclomol, an orally-delivered, first-in-class heat shock protein (HSP) amplifier being developed as a treatment for Niemann-Pick disease type C (NPC). NPC is a rare progressive neurodegenerative disease that impacts children, adolescents, and adults, and is characterized by an inability of the body to transport cholesterol and lipids inside of cells, which leads to the abnormal accumulation of these substances within various tissues of the body, including the brain. Arimoclomol is currently being made available to NPC patients in the U.S., France and Germany under Orphazyme

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