The designation, for severe tracheal stenosis (narrowing of the windpipe), is an important recognition by the European Medicines Agency of the potential of the replacement trachea in the treatment of this life-limiting rare disease. Orphan drug status will help expedite development of the replacement trachea, which is beginning a Phase I clinical trial, into a commercially available product. Designed to bring patient benefit by encouraging research into rare diseases, EU orphan designation covers products for the prevention or treatment of life-threatening or severe conditions which affect no more than 5/10,000 people in the EU (no more than 250,000 in total).
The designation brings a variety of benefits for the development of products like the replacement trachea, such as 10 years’ market exclusivity in the EU, additional technical and scientific guidance, reduced costs and potential access to funding. Videregen will, in due course, also apply for a US orphan drug designation, which brings similar benefits. Tracheal stenosis has been estimated to have a prevalence of 1.8 per 10,000 which translates to 91,440 in the EU population. Of this population of tracheal stenosis sufferers, a proportion would be appropriate for a tracheal replacement. Other estimates, such as ICD9 data, put the overall incidence of severe structural airway disease at c 12,000 in the EU pa. The Phase I INSPIRE clinical trial, which is supported by a grant from Innovate UK, will treat four patients with severe structural airways disease. Videregen is leading the consortium1 of experts in tissue engineering and cell therapy which is undertaking the trial. Dr Steve Bloor, CEO of Videregen, said, ‘Orphan medical product designation for our tissueengineered personalised replacement trachea is a significant milestone on its development pathway, and recognition of its potential in the treatment of severe structural airways disease. It will also help to expedite availability of the product on a commercial basis with resultant patient benefit.’ Liverpool, UK – October 3 2016