ReNeuron Receives European and US Orphan Drug Designation
News Aug 30, 2013
ReNeuron Group plc has announced that its ReN003 retinal stem cell therapy candidate for retinitis pigmentosa has achieved a significant regulatory milestone.
The therapy has been granted Orphan Drug Designation in both Europe and the US by the European Commission and the Food and Drug Administration, respectively.
Orphan Drug Designation is typically granted to treatments that provide significant benefit to patients with rare diseases that are life-threatening or chronically debilitating.
Treatments with this designation benefit from significant commercial and regulatory advantages such as market exclusivity for 10 years from approval in the disease concerned, against other treatments offering no greater therapeutic advantage.
ReNeuron is using its proprietary human retinal progenitor cells as the basis of its ReN003 therapeutic candidate targeting retinitis pigmentosa, a group of hereditary diseases of the eye that lead to progressive loss of sight due to cells in the retina becoming damaged and eventually dying.
Retinitis pigmentosa affects approximately 4 in 10,000 people in the EU and US.
The late pre-clinical development of the ReN003 programme also benefits from a recently awarded £0.8 million Biomedical Catalyst grant from the UK Technology Strategy Board.
The Company is targeting an initial Phase I/II clinical trial with ReN003 in the UK and the US, in retinitis pigmentosa patients, with clinical trial applications expected to be filed in the middle part of 2014.
Michael Hunt, Chief Executive Officer of ReNeuron, said: “We are delighted to have received Orphan Drug Designation for our ReN003 stem cell therapy candidate for retinitis pigmentosa from both the European Commission in Europe and the FDA in the US. Orphan status diseases are an increasing area of therapeutic and commercial focus by the mainstream pharmaceutical industry due to the distinct regulatory and market exclusivity advantages that Orphan Drug Designation confers. This fact bodes well for the commercial potential of our ReN003 therapy.”
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