Copenhagen, October 15, 2018 – Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced the company’s endorsement of published guidelines to support interactions with rare disease patient advocacy organizations.
Published in the Orphanet Journal of Rare Diseases earlier this year, Principles for interactions with biopharmaceutical companies: The development of guidelines for patient advocacy organizations in the field of rare diseases, is a landmark article for the rare disease community. Toni Mathieson, Chief Executive of Niemann-Pick UK (NPUK), an organization that works in partnership with Orphazyme, is a co-author on the paper.
Orphazyme colleagues who oversee engagement with patient communities will attend the National Organization of Rare Diseases (NORD) Rare Summit this week in Washington, D.C. We welcome the opportunity to meet with patient organizations working to support NPC, ALS, IBM, and Gaucher disease communities.
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.
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 1,000-2,000. 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.
Sporadic Inclusion Body Myositis (sIBM) is a progressively debilitating muscle-wasting disease. sIBM is characterized by a build-up of protein aggregates and atrophy of muscle cells, which leads to weakness and over time severe disability. The estimated prevalence of sIBM is 24.8-45.6 per million or 17,000-31,000 patients in the USA and Europe. There are no approved treatments for sIBM. Arimoclomol has been granted Orphan Drug Designation (EU and USA) for the treatment of sIBM.
Amyotrophic Lateral Sclerosis (ALS) is a rare, rapidly progressive, and always fatal neurodegenerative disease. Protein misfolding and aggregation in motor neurons are important contributors to the disease process, which ultimately leads to paralysis of skeletal muscles as well as the muscles that enable breathing. The patient population in Europe and the United States is estimated to be approximately 50,000 patients. Currently, there are only limited treatment options available. Arimoclomol has been granted Orphan Drug Designation (EU and USA) for the treatment of ALS.
Gaucher disease is a genetic, progressively debilitating lysosomal storage disease caused by mutations leading to defective glucocerebosidase (GCase) protein. As a consequence, lipids that are normally cleared by the lysosome build-up in tissues and organs, including the bone marrow, liver, spleen, and sometimes brain, and drive the disease pathology. The estimated number of people affected with Gaucher disease in the USA and Europe combined is 10,000-15,000. Effective treatments are available for the systemic manifestations of the disease – however, no therapies are available for the neurological symptoms.
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