Orphan designation for Huntington's candidate

Drug development company Prana Biotechnology (ASX:PBT) today announced that the European Commission has approved orphan designation for its lead drug candidate, PBT2, for the treatment of Huntington’s disease.

Orphan designation is granted by the EC to encourage the development of medicine to treat rare diseases. It provides for 10 years of market exclusivity in the European Union from the granting of a marketing authorisation (approval for sale), as well as assistance in developing clinical protocols, reduced fees and access to EU-funded research grants.

The approval of PBT2 was based on the recommendation of a positive opinion from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP). According to the minutes of a COMP committee meeting, the intention to use the product to treat Huntington’s disease “was justified based on pre-clinical in vivo data and preliminary clinical data showing improvement in parameters associated with the condition”.

In February 2014, Prana announced that PBT2 had met its primary endpoint for safety and tolerability, as well as improved measures of cognitive performance in a clinical trial. The drug received orphan drug status for the treatment of Huntington’s disease from the US FDA in September that year.

Prana Biotechnology (ASX:PBT) shares were trading 22.22% higher at $0.220 as of around 3.30 pm on Monday.