· Expected submission of Marketing Authorization Application (MAA) in H1 2020
Copenhagen, June 7, 2019 – Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announces that it has received constructive advice from the European Medicines Agency (EMA)’s 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 MAA for NPC in the first half of 2020.
Anders Hinsby, Chief Executive Officer, said: “We are pleased with the advice received from EMA, which provides good guidance on topics that we will take into careful consideration as we continue the preparation of a Marketing Authorization Application for arimoclomol in Europe. NPC is a severely debilitating and fatal disease that predominantly affects children and we are committed to bringing arimoclomol to patients suffering from this devastating disease”.
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 and will provide an update after it has received written comments from the FDA.