Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium - Seite 2

 

Rescue of NPC1 protein by the Heat-Shock response amplifier arimoclomol across multiple genotypes

• Presented by Nikolaj Petersen, Ph.D.
• Thursday, February 11, Poster 197
   

Impacts and burden of NPC: A Patient and Caregiver Perspective

• Presented by Eugen Mengel, M.D.
• Thursday, February 11, Poster 158
   

Validation of a short-form 5-domain Niemann-Pick disease type C clinical severity scale (5-domain NPCCSS)

• Presented by Marc Patterson, M.D.
• Thursday, February 11, Poster 190

Arimoclomol is under Priority Review by the U.S. Food and Drug administration (FDA) for the treatment of NPC with a target action date of June 17, 2021. NPC is a rare, progressive, life-threatening, neurodegenerative disease for which there is no cure and no approved treatment in the United States. Arimoclomol is also under review in Europe for the NPC indication.

“We continue to engage in productive dialogue with the FDA related to the NDA for arimoclomol,” said Thomas Blaettler, Chief Medical Officer, Orphazyme. “In parallel, we continue to enroll patients in our Early Access Program (EAP) in the US and we are initiating EAPs in European countries, including France and Germany. These activities advance Orphazyme’s mission to bring an innovative treatment to rare disease communities with significant unmet need.”

For additional information, please contact

Orphazyme US

Chicago, USA: Molly Carey Poarch                                      [phone]

About Orphazyme A/S 
Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Arimoclomol, the company’s lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease. Orphazyme is headquartered in Denmark and has operations in the U.S. and Switzerland. Orphazyme’s shares are listed on Nasdaq U.S. (ORPH) and Nasdaq Copenhagen (ORPHA).