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FDA Denies Orphazyme’s Application for Drug Treating Rare Genetic Disorder

June 22, 2021

Orphazyme has drawn a Complete Response Letter from the FDA, rejecting its New Drug Application for arimoclomol for treatment of the rare genetic disorder Niemann-Pick disease type C (NPC).

NPC is a progressive disease marked by the body’s inability to transport cholesterol and other lipids inside cells, causing a buildup of the fatty substances in various tissues, including in the brain.

The Danish drugmaker said the FDA called for more evidence of arimoclomol’s efficacy in reducing disease severity and for confirmation of data from a phase 2/3 study.

Orphazyme said it plans to focus on pursuing approval from the European Medicines Agency and is anticipating a decision from that agency late this year. 

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