Clinigen and Orphazyme to introduce and prescribe an early access system for Arimoclomol in Niemann-Pick type C (NPC)

Clinigen Group plc (AIM: CLIN,’ Clinigen’), a multinational pharmaceutical and service group, has partnered with Orphazyme A / S (ORPHA.CO,’ Orphazyme’), a global biopharmaceutical company dedicated to developing therapies for rare disease patients, to introduce and prescribe an early access system for arimoclomol in Niemann-Pick type C (NPC) patients in the United States.

Arimoclomol received orphan drug, fast track and breakthrough designations from the U.S. Food and Drug Administration (FDA) and announced positive results from a clinical Phase II / III trial in NPC. Orphazyme expects to submit arimoclomol in NPC in H1 2020 to the FDA for marketing approval.

NPC is a rare, genetic and progressive disease caused by the formation of lipids in the body’s organs, including the brain tissue, which causes damage to the affected areas. NPC has a devastating effect on the quality of life of an individual, with most patients in need of full-time care.

Arimoclomol is a candidate for an investigational drug that increases the development of heat-shock proteins (HSPs). HSPs can rescue defective proteins, clear protein aggregates, and improve lysosomal function. Arimoclomol is administered orally, crosses the blood-brain barrier, and was tested in 7 Phase I and 3 Phase 2 tests. Arimoclomol is in the NPC, Gaucher disease, IBM, and ALS clinical development.

Type C (NPC) Niemann-Pick disease is a hereditary, slowly worsening, and often fatal neurovisceral disease. This belongs to a family known as lysosomal storage diseases and is caused by faulty NPC protein mutations. As a consequence, lipids that are usually cleared in tissues and organs, including the brain, by the lysosome build-up and drive the disease pathology. The combined prevalence of NPC in the United States and Europe is estimated at 1,000. In the USA there are no approved NPC treatments and in Europe, there is only one approved product. Arimoclomol has been granted the designation of Orphan Drug (EU and USA), the designation of Rare Pediatric Condition (USA), and the designation of Fast Track (USA) for NPC care.

Clinigen Group plc (AIM: CLIN) is a multinational product and service company with a unique mix of businesses focused on providing ethical access to medicines. The mission is to provide the right medicine at the right time to the right patient across three fields of the global supply of drugs; clinical trials, unlicensed and licensed medicines. The Corporation is based in North America, Europe, Africa and the Asia Pacific.