OPMD is a rare inherited myopathy characterised by dysphagia (difficulty in swallowing), the loss of muscle strength, and weakness in multiple parts of the body.
The orphan drug designation provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition.
To meet the criteria as an orphan drug, the condition should not affect more than five in 10,000 persons in the European Union (EU) and no satisfactory treatment should be available.
In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives from the European Medicines Agency during the product development phase.
Benitec Biopharma’s BB-301 is a ddRNAi-based therapeutic that enables ‘knockdown and replace strategy’ of the principle cellular component that leads to OPMD disease condition in humans.
BB-301 is currently in preclinical development and Benitec plans to initiate IND (investigational new drug)-enabling studies later this year.
Entry into the clinic with a Phase I/II study in OPMD patients is anticipated in 2018, subject to toxicity results and future regulatory review.
Benitec Biopharma’s share price has increased by about 27% since the beginning of this year, last trading at $0.145.