Clinigen Group plc (AIM: CLIN, ‘Clinigen’), the global pharmaceutical and services company, has partnered with Orphazyme A/S (ORPHA.CO, ‘Orphazyme’), a global biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, to introduce and administer an Early Access Program for arimoclomol in patients with Niemann-Pick disease type C (NPC) in the US.
Arimoclomol has received orphan drug, fast track and breakthrough designations from the US Food and Drug Administration (FDA) and has reported positive results from a Phase II/III clinical trial in NPC. Orphazyme expects to submit to the FDA for marketing approval of arimoclomol in NPC in H1 2020.
NPC is a rare, genetic and progressive disease caused by the build-up of lipids within the organs of the body, including the brain tissue, causing damage to the affected areas. NPC has a devastating effect on an individual’s quality of life, with most sufferers requiring full-time care.
Healthcare professionals in the US can obtain details about the arimoclomol Early Access Program by calling the customer service team at +1 877-768-4303 or emailing firstname.lastname@example.org.
Patients seeking medical information should contact their physician.
Notes to Editors
Arimoclomol is an investigational drug candidate that amplifies the production of heat-shock proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, crosses the blood brain barrier, and has been studied in seven Phase I and three Phase II trials. Arimoclomol is in clinical development for NPC, Gaucher disease, sIBM, and ALS.
Niemann-Pick disease Type C (NPC) is a genetic, progressively debilitating, and often fatal neurovisceral disease. It belongs to a family know as lysosomal storage diseases and is caused by mutations leading to defective NPC protein. As a consequence, lipids that are normally cleared by the lysosome build-up in tissues and organs, including the brain, and drive the disease pathology. The estimated prevalence of NPC in the USA and Europe combined is 1,000. There are no approved treatments for NPC in the USA and only one approved product in Europe. Arimoclomol has been granted Orphan Drug Designation (EU and USA), Rare Pediatric Disease Designation (USA), and Fast Track designation (USA) for the treatment of NPC.
About Clinigen Group
Clinigen Group plc (AIM: CLIN) is a global pharmaceutical and services company with a unique combination of businesses focused on providing ethical access to medicines. Its mission is to deliver the right medicine to the right patient at the right time through three areas of global medicine supply; clinical trial, unlicensed and licensed medicines. The Group has sites in North America, Europe, Africa and Asia Pacific.
Clinigen now has over 1,100 employees across five continents in 14 countries, with supply and distribution hubs and operational centres of excellence in key long-term growth regions. The Group works with 22 of the top 25 pharmaceutical companies; interacting with over 15,000 registered users across over 100 countries, shipping approximately 6.4 million units in the year.
For more information on Clinigen, please visit www.clinigengroup.com
About Orphazyme A/S
Orphazyme is a biopharmaceutical company focused on bringing novel treatments to patients living with life-threatening or debilitating rare diseases. Our research focuses on developing therapies for diseases caused by misfolding of proteins, including lysosomal storage diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. The Denmark-based company is listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit www.orphazyme.com.
Issued for and on behalf of Clinigen by Instinctif Partners.
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Melanie Toyne-SewellManaging Partner