EMA publishes minutes recommending Orphan Drug Designation for PBT2
Prana Biotechnology Limited today announced that the European Medicines Agency (EMA) has published the Minutes from the April 2015 Meeting of its Committee for Orphan Medicinal Products (COMP), detailing the recommendation from the COMP that PBT2 be granted Orphan Designation in Europe. Prana announced the positive COMP recommendation on 28 April, 2015.
The relevant extract of the Minutes states:
“The Committee agreed that the condition, Huntington’s disease, is a distinct medical entity and meets the criteria for orphan designation.
The intention to treat the condition with the medicinal product containing 5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride* was considered justified based on pre-clinical in vivo data and preliminary clinical data showing improvement in parameters associated with the condition.
The condition is life-threatening and chronically debilitating due to severe behavioural and cognitive disturbances, progressive motor dysfunction and potentially fatal complications.
The condition was estimated to be affecting approximately 1 in 10,000 persons in the European Union, at the time the application was made.
In addition, although satisfactory methods of treatment of the condition have been authorised in the European Union, the sponsor has provided sufficient justification for the assumption that the medicinal product containing 5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride may be of significant benefit to those affected by the condition. The sponsor has provided preliminary clinical data that demonstrate an improvement in a relevant parameter when the product is used in combination with tetrabenazine. The Committee considered that this could constitute a clinically relevant advantage.
A positive opinion for containing 5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride, for treatment of Huntington’s disease, was adopted by consensus.”
Prana’s Chairman and CEO, Geoffrey Kempler, said “The Minutes support our strategy to gain Orphan disease status for PBT2 to treat Huntington Disease and I am optimistic that the European Commission will review the recommendation and grant approval for Orphan drug designation for PBT2 in the near future”.
*the extract refers to PBT2’s chemical name.