News Room

Investor Announcement, Huntington's Disease

PBT2 recommended for orphan designation in Europe

MELBOURNE, 28th April, 2015: Prana Biotechnology (ASX: PBT/NASDAQ:PRAN) is pleased to announce the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending designation of PBT2 for the treatment of Huntington disease as an orphan medicinal product to the European Commission (EC).

Following finalisation of relevant documents the European Medicines Agency forwards the opinion to the EC for its decision on the designation.

“We welcome the positive opinion from COMP for PBT2 to receive orphan designation to treat Huntington disease in Europe,” said Prana Chairman and CEO Geoffrey Kempler.

“Prana is dedicated to progressing development of PBT2 as a treatment for this terrible disease that devastates lives and families.”

Orphan designation is granted by the EC to encourage the development of medicines to treat rare diseases. Rare diseases are defined as life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in Europe.

Huntington disease is a neurodegenerative genetic disorder that affects muscle coordination and leads to cognitive decline and behavioral symptoms. The Huntington’s Outreach Project for Education estimates there are between 40-100 cases of Huntington disease per million people in Europe.

The EC offers orphan medicinal product developers incentives including scientific advice, market exclusivity for 10 years for approved drugs and reduced fees.

In September last year the US Food and Drug Administration granted PBT2 orphan drug status for the treatment of Huntington disease.

In February last year Prana announced that PBT2 had met its primary end point of safety and tolerability, and improved measures of cognitive performance – a secondary endpoint in its REACH2HD Phase 2 clinical trial involving 109 people with Huntington disease.