FDA grants orphan drug designation for Arimoclomol

Orphazyme today announced that FDA’s OOPD has granted an orphan drug designation for Orphazyme’s Arimoclomol as a new treatment for Niemann-Pick disease type C

The US orphan drug designation complements the corresponding EMA orphan drug designation that was granted for the program in 2014.

"The recent successful completion of a 20 million EUR Series B financing round, together with the FDA and EMA orphan drug designations, establish a solid foundation for initiating our clinical studies in NP‑C with the purpose of providing arimoclomol as a safe and effective therapeutic option to patients suffering from this devastating disease as well as relief to their families,” says Dr. Carlos R. Camozzi, Chief Medical Officer of Orphazyme.

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


See the FDA log of its decision here and the EMA log here.


Further information: 
Carlos Camozzi, CMO of Orphazyme
E-mail: crc@orphazyme.com

About Orphazyme

Orphazyme ApS is a Danish biopharmaceutical company, which develops paradigm-changing medicines for the treatment of genetic diseases. The lead program is entering clinical development as treatment for lysosomal storage disease. This family of genetic disorders includes NP-C and consists of more than 45 diseases, often affecting children, most of which are currently untreatable and often fatal. Orphazyme has raised more than €37m in seed, Series A and Series B financing from a strong investor base, which includes Novo A/S, Sunstone Capital, Aescap Venture, Kurma Partners and Idinvest Partners. For more information, please visit www.orphazyme.com.