Soligenix announced the European Commission, acting on the positive recommendation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products, has granted orphan drug designation to the company's recombinant modified ricin toxin A-chain subunit (the active pharmaceutical ingredient in RiVax) for the prevention of ricin poisoning. RiVax has previously been granted orphan drug designation from the US Food and Drug Administration (FDA).
The European Commission grants orphan designations for medicines that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU) and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure which allows access to all 28 EU member states' markets.
"We are extremely pleased to have received European orphan drug designation for the RiVax program," said Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "This EU orphan designation, combined with our US orphan designation, positions this biodefense program for a potentially accelerated global regulatory product development pathway to address this unmet need. RiVax has shown up to 100% protection against aerosolized ricin exposure in non-human primates and safety in humans and we look forward to further developing our thermostable formulation for human use."
Ricin toxin is a lethal plant-derived toxin and potential biological weapon because of its stability and high potency, and the fact it is readily extracted from by-products of castor oil production. Ricin comes in many forms including powder, mist or pellet. Ricin can also be dissolved in water and other liquids. The US Centers for Disease Control and Prevention estimates that the lethal dose in humans is about the size of a grain of salt. Ricin toxin illness causes tissue necrosis and general organ failure leading to death within several days of exposure. Ricin is especially toxic when inhaled. Ricin works by entering cells of the body and preventing the cells from making the proteins it needs. Without the proteins, cells die, which is eventually harmful to the entire body.
There are currently no effective treatments for ricin poisoning. The successful development of an effective vaccine against ricin toxin may act as a deterrent against the actual use of ricin as a biological weapon and could be used to vaccinate military personnel and civilian emergency responders at high risk of potential exposure in the event of a biological attack.