European Commission Approves PBT2 Orphan Designation for Huntington’s Disease

Prana Biotechnology Limited has today announced that the European Commission has approved orphan designation for PBT2 for the treatment of Huntington’s disease, stating that Prana has shown that PBT2 might be of significant benefit for patients with Huntington’s disease.

The approval was based on the recommendation of a positive opinion from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP). The COMP assessed the scientific documentation for PBT2 against the key criteria for orphan designation.  The criteria require that PBT2 be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating; have a prevalence in the EU not more than five in 10,000, and no satisfactory method of diagnosis, prevention or treatment of the condition concerned, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

Orphan designation provides for ten years of market exclusivity in the European Union (EU) from the granting of a marketing authorisation (approval for sale). Other benefits relate to assistance in developing clinical protocols, reduced fees, and access to EU-funded research grants.

Last year Prana announced positive findings in its Phase 2 clinical trial for Huntington’s disease (Reach2HD) and is planning for further clinical trials.  The company has Orphan Drug designation with the US Food and Drug Administration. Further opportunities for the development of PBT2 in the US can commence on the removal of the current Partial Clinical Hold.