FDA grants orphan drug designation for Arimoclomol 

Copenhagen, Denmark, January 26, 2015: 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 €20m 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 EMA log of its decision here.

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