COMP issues positive opinion on orphan drug application for Arimoclomol

COMP issues a positive opinion on the orphan drug application for Arimoclomol as a new treatment for Niemann-Pick disease type C

The Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion on the orphan drug application for Arimoclomol citrate as a treatment of Niemann-Pick disease type C (NP-C), recommending it for designation as an orphan medicinal product to the European Commission (EC). The opinion is the regulatory confirmation of Arimoclomol as a valuable therapeutic option for NP-C patients.

“The positive COMP opinion brings further confidence in the preparation of the imminent clinical trial of Arimoclomol in NP-C. We are committed to providing new high quality therapeutic options to patients suffering from devastating degenerative diseases and their families and look forward to commencing the clinical trial in NP-C”, says Dr. Carlos R. Camozzi, Chief Medical Officer of Orphazyme.

Orphazyme plans to start a clinical trial in NP-C in 2015.

Read COMP's release of 14 October 2014 here

Further information: 
Carlos Camozzi, CMO of Orphazyme

About Orphazyme

Orphazyme ApS is a Danish biopharmaceutical company, which develops paradigm-changing medicines for the treatment of genetic diseases. The lead program is in preclinical development as treatment for lysosomal storage diseases. This family of genetic disorders consists of more than 45 diseases, most of which are currently untreatable and often fatal. Orphazyme has raised more than 17 million euro in seed and series A financing, from a strong investor base which includes Novo Ventures, Sunstone Capital and Aescap Venture. For more information, please visit