Research & Development
Orphazyme to file MAA for Niemann-Pick disease Type C (NPC) for arimoclomol in Europe
10 June 2019 -

Orphazyme A/S (CPH:ORPHA), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, announced on Friday the receipt of constructive advice from the European Medicines Agency (EMA) Scientific Advice Working Group in response to the request for advice submitted to the agency.

After receiving this feedback, Orphazyme confirms its intention to file the Marketing Authorisation Application (MAA) for Niemann-Pick disease Type C (NPC) in the first half of 2020.

According to the company, it is pleased with the advice received from EMA, which provides good guidance on topics that it will take into careful consideration as it continues the preparation of a Marketing Authorisation Application for arimoclomol in Europe.

Also, additional data, including 12-month open-label extension data, will become available during H2 2019 and must be included in the MAA.

Orphazyme plans to meet with the US Food and Drug Administration (FDA) to discuss the pathway forward during the summer of 2019 and will provide an update after it has received written comments from the FDA.