Orphazyme, a pioneer in the development of heat shock protein based therapies for genetic diseases, receives from the European Commission an orphan drug designation for its lead compound, recombinant human Heat Shock Protein 70 (OR0005) for the indication "Treatment of Niemann-Pick’s disease, type C".
Thomas Kirkegaard, Orphazyme CSO, says: ”The orphan drug designation represents an important step forward for Orphazyme and our aspiration to improve the lives of patients suffering from severe genetic diseases such as Niemann-Pick disease. It is an acknowledgement of the potential for Orphazyme’s HSP70 therapy to generate significant benefit to patients with lysosomal storage diseases as evidenced by the body of non-clinical data that supports the progression of this protein therapy for Niemann-Pick disease type C.”
The European orphan drug designation is intended to facilitate drug development for rare diseases, and provides substantial benefits to the sponsor, including 10 years of market exclusivity for the product upon regulatory approval.
Anders Hinsby, CEO of 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 www.orphazyme.com.