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BioWorld - Monday, January 5, 2026
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Home » Orphazyme’s rare disease drug set for rejection after European regulators’ meeting
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Orphazyme’s rare disease drug set for rejection after European regulators’ meeting

Feb. 25, 2022
By Richard Staines
After a rejection by the FDA in June, it looks like Orphazyme A/S is headed for disappointment in Europe too with arimoclomol for Niemann-Pick disease type C, a rare and potentially fatal inherited condition in which fat builds in tissues and organs. The Copenhagen-based company said it was summoned before experts to give an “oral explanation” about the drug, something that only occurs if the European Medicines Agency’s CHMP has developed major doubts during its review.
BioWorld Regulatory Europe CHMP EMA

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