Videregen's Lead Product Granted EU Orphan Drug Status
10/3/2016 9:17:30 AM
Recognition of potential of personalised trachea replacement in airways disease
Liverpool, UK - October 3 2016: Videregen Limited, a clinical stage regenerative medicine company developing personalised organ replacements, has been awarded European orphan medicinal product status for its lead tissue-engineered replacement trachea product. 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 tissue-engineered 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.'
1 For more details of the INSPIRE trial and consortium please go to http://www.videregen.com/news-events/
For more information, please contact:
Dr Steve Bloor, CEO
+44 (0) 151 482 9350
Emma Palmer Foster, Strategic Communications Consultant
+44 (0) 7880 787185
Videregen is a clinical-stage regenerative medicine company using its proprietary stem cell-based technology platform to develop a range of personalised, non-immunogenic organ replacement products for orphan indications. Its lead programme is a tissue-engineered trachea replacement, and its patented technology and know-how is also being applied to the development of other organ replacement products, including mucosal lining, small bowel and liver replacements. The platform technology, which uses decellularised organ scaffolds seeded with the patient’s own cells to create new organs, has the potential to generate cost-effective and curative therapies for a range of devastating diseases. The decellularisation technology was originally developed at Northwick Park Institute for Medical Research. Founded in 2011, Videregen is an SME based in Liverpool. For more information please go to www.videregen.com
About severe structural airway disease
Severe structural airway disease is associated with significant airway obstruction leading to high levels of morbidity and mortality if not treated successfully. Currently patients are subjected to repeated limited efficacy surgical interventions, such as stent insertions, which ultimately have a high incidence of failure. In contrast, Videregen's tracheal replacement approach, which involves repopulating an acellular trachea 'scaffold' with the patient's own stem cells, is designed to be a one-off curative treatment without the need for a lifetime of anti-rejection drugs thereafter. The overall incidence of severe structural airway disease is estimated at c.12,000 in EU per year. On average this patient group will spend 22 days in hospital at a cost of €218,619 per patient translating to an EU-wide population cost of €2.6bn per year.
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