Orphazyme A/S reports positive FDA meeting and plan to submit NDA for arimoclomol for Niemann-Pick Disease.

Orphazyme A/S announced that it has had a positive meeting with the FDA and remains on track to submit an NDA for arimoclomol for Niemann-Pick type C disease (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. Orphazyme also intends to file an MAA in Europe in H1 2020.