· US Food and Drug Administration (FDA) agrees to Orphazyme’s plan
to file New Drug Application (NDA) in US
· On track to file NDA and Marketing Authorisation Application (MAA) in NPC
in both US and EU in H1 2020
Copenhagen, July 21, 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 had a positive meeting with the FDA and remains on track to submit an NDA for arimoclomol for NPC in H1 2020.
Anders Hinsby, Chief Executive Officer of Orphazyme, said: “We are very pleased with the collaborative interactions we have had with the FDA who provided us with thorough guidance on data presentation in the NDA for arimoclomol in the US for NPC. The valuable advice we have received from health authorities in both the US and Europe increases our optimism that we may soon be able to provide an important treatment option for NPC, a severely debilitating and fatal disease that predominantly affects children.”
Following the encouraging regulatory agency feedback, Orphazyme plans to introduce an Early Access Program for NPC in the fall of 2019, to further accelerate access to treatment with arimoclomol for people living with NPC.
As previously disclosed, Orphazyme also intends to file an MAA in Europe in H1 2020.