Copenhagen, Denmark, September 17, 2019 – Orphazyme A/S (ticker: ORPHA.CO), a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, has been invited to participate in a meeting hosted by the National Organization for Rare Disorders (NORD) and the Critical Path Institute (C-PATH) to launch their joint initiative: the Rare Disease Cures Accelerator Data and Analytics Platform (RDCA-DAP). Funded by the US Food and Drug Administration (FDA), the goal of RDCA-DAP is to accelerate the process of moving therapies from bench to bedside for rare diseases. 

Today, Orphazyme’s Chief Medical Officer, Thomas Blaettler, MD, will participate in a moderated panel discussion on “Current Challenges in Rare Disease Drug Development,” during which he will share Orphazyme’s experiences in working to bring new treatments for orphan diseases to market. The event, taking place in Rockville, MD, USA, is open to the public and expected to draw attendance from regulators, patient organizations, clinicians, researchers, and pharmaceutical companies focused on rare disease drug development. 

“Orphazyme is proud of the work we have done, in collaboration with leading disease experts and patient organizations, to develop arimoclomol for Niemann-Pick disease Type C (NPC) and other underserved patient communities” said Blaettler. “We are hopeful our experiences can help to smooth the path for others in pursuit of new treatments for those living with rare diseases.”

Orphazyme has confirmed its filing strategy for arimoclomol in NPC with both European and US regulators and remains on track to file for its first marketing authorization, in NPC, during H1 2020. Arimoclomol is in clinical development for three additional indications of high unmet need: Amyotrophic Lateral Sclerosis (ALS), sporadic Inclusion Body Myositis (sIBM), and Gaucher disease.