ReNeuron’s investigational stem cell therapy has received orphan status in the EU and US for use in a rare eye disorder.
Both the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) determined that ReN003 had the potential to provide significant benefits for patients with retinitis pigmentosa, a diverse group of inherited eye disorders that affect the retina and can cause severe impairment to vision.
The condition is thought to affect just 4 in 10,000 people in the EU and US, meaning any treatment would be eligible for orphan status in both regions, with the FDA stating that rare diseases must affect fewer than 200,000 people in the US while the EMA states the prevalence of the condition in the EU must not be more than 5 in 10,000.
By winning these designations, ReNeuron is entitle to several advantages during the regulatory process for ReN003, including scientific advice and, once approved, ten-years market exclusivity for the disease in the EU and tax credits in the US.
ReNeuron’s CEO Michael Hunt said the company was “delighted” to receive orphan designation for ReN003, adding that it fitted with a wider industry movement towards rare diseases.
“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,” he said. “This fact bodes well for the commercial potential of our ReN003 therapy.”
ReN003 is one of several ‘off-the-shelf’ stem cell therapies in development by ReNeuron, including its lead candidate ReN001, which is being investigated as a treatment for patients left disabled by the effects of a stroke.
ReNeuron is banking on the ability of these therapies to address the underlying causes of the target disease rather than just its symptoms, and pre-clinical testing has demonstrated their effectiveness in stimulating natural repair mechanisms in the organs.
The company’s potential has been noted by the government of Wales, which offered grant money to ReNeuron to move its operations from England to establish a manufacturing and development facility in South Wales.
At the time, ReNeuron’s CEO Hunt said: “The Welsh grant package enables us to take control over the manufacture of our stem cell therapy candidates as they get closer to market.”
