Copenhagen, Denmark, January 19, 2018 – Orphazyme A/S, a Danish biotech company listed on Nasdaq Copenhagen (TICKER: ORPHA.CO), with a late-stage, orphan-drug pipeline, today announced that arimoclomol has been granted rare pediatric disease designation by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease Type C (NPC).

NPC is a genetic disease affecting around 1 in 120,000 newborns. Although the time of symptoms onset is variable, NPC is most often diagnosed in childhood and adolescence. Progressive neurological pathology is the hallmark of NPC and is responsible for disability and premature death in most patients. Arimoclomol, an orally available small molecule, is currently being investigated in a clinical Phase II/III trial as a potential treatment for NPC.

Anders Hinsby, CEO of Orphazyme, says: “We are very pleased with the FDA’s continued support for the development of therapies for children with rare diseases. Arimoclomol has now been granted orphan drug, fast track, and rare pediatric disease designations by the FDA, which underpin the high unmet need in NPC, providing acknowledgement of the arimoclomol program. We look forward to continue working closely with the FDA in our effort to provide a treatment option for patients suffering from NPC.”  

With the rare pediatric disease designation, the FDA provides incentive for the development of treatments for rare pediatric diseases. A drug qualifies for this category if the entire prevalence of the disease in the US is below 200,000 and if more than 50% of the patients are between 0-18 years.

If a drug candidate with a rare pediatric disease designation receives marketing approval in the US, the FDA will, if certain criteria are met, issue a Priority Review Voucher to the sponsor company. This voucher can be redeemed to provide Priority Review of a subsequent marketing application for a different product.