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More disappointment for Orphazyme as FDA rejects Niemann Pick C application

The FDA have rejected an application for marketing authorization for Orphazyme's main drug candidate, arimoclomol, as a treatment for metabolic disease Niemann Pick C. The authority wants more data.

Photo: Orphazyme/PR

The bad news just keeps on coming for Orphazyme.

The biotech company's key candidate, arimoclomol, got the thumbs down from the US Food and Drug Administration (FDA) as a treatment for Niemann Pick type C, also known as NPC.

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