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Update on regulatory review of arimoclomol in the European Union for treatment of Niemann-Pick disease type C.

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Published:1st Mar 2022

Orphazyme A/S , announced an update on the ongoing review of the Marketing Authorisation Application (MAA) for its investigational product candidate, arimoclomol, for the treatment of Niemann-Pick disease type C (NPC) by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

While Orphazyme was encouraged by the positive feedback of the ad-hoc expert group meeting held on February 17, 2022, Orphazyme has been notified by the CHMP of a negative trend vote on the MAA for arimoclomol in NPC following an Oral Explanation. The trend vote indicates that the CHMP's current orientation is to not approve arimoclomol when it convenes by the end of March 2022. Orphazyme considers it unlikely that this position will change before the formal vote is undertaken next month.

In the light of the recent development and the Company’s financial situation, the Company will now assess the Company’s strategic options and provide an update to the market at the applicable time.

Condition: Niemann Pick Disease/ASMD
Type: drug

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