In order to offer the InBreath System widely, we will need to successfully complete clinical trials. The first indication for which we intend to seek approval is to repair or replace the airway in a patient who has had a cancerous trachea removed or other severe damage to the trachea. Because trachea and bronchus cancers are relatively rare diseases, affecting only approximately 2,400 patients per year in the developed world but are quickly fatal, (e.g., median survival in trachea cancer is only 10 months) we anticipate that our clinical trials will involve relatively few patients and will have a relatively short follow up period. If, as we expect, in the U.S. the regulatory pathway is the biologics pathway, then we will seek approval under the Orphan Drug pathway. The Orphan Drug pathway can be used if the disease or condition for which the regenerated trachea is intended affects fewer than 200,000 people in the U.S. This pathway is advantageous because, although the safety and efficacy standards for orphan drug products are the same as for all biologics, approval under this pathway could grant the company a seven year exclusivity on marketing the product for that indication in the U.S.
As regenerated trachea products are implantable products, if they were classified as medical devices in the EU, we would need to conduct related clinical studies, unless we can justify relying on already-existing clinical data. If, in the EU, the regenerated trachea products are classified as medicinal products, it would be possible to seek orphan medicinal product classification if we can demonstrate that the products are intended to treat a condition affecting no more than five per 10,000 persons in the EU, or that they are intended for treating a serious or debilitating disease and it is unlikely that without incentives marketing the products would generate sufficient return to justify the necessary related investment. If the regenerated trachea products were classified as orphan medicinal products, they could be entitled to market exclusivity for ten years. We believe we meet the standard to be classified as an orphan medicinal product in the EU.
Russian and EU Patients
There have been four patients in Russia treated with the HART-Trachea, in 2013 and 2014, This was a single arm, open-label study. This means that there is no control group of patients that are being used to compare the treatment to standard of care. The outcome of the trial is being measured principally on the basis of survival or extension of life. Of the three patients who have died so far, the average survival is 22 months from the first surgery (some of these patients had also had a prior implant of a scaffold not made by us), compared to an expectation of survival at the time of surgery of typically only a few months. One of these patients treated in Russia is still alive as is the one patient treated in Stockholm, Sweden.
HART has made progress in our preclinical program in preparation for filing an application for Clinical Trial Authorization (CTA) with the Medicines and Healthcare Products Regulatory Agency of the U.K. (MHRA). Also, we began large animal studies as part of the preclinical program being pursued in preparation for filing an Investigational New Drug application (IND) with the FDA. As previously disclosed, we expect the preclinical program to continue well into 2015 and for the submissions of a CTA and IND to take place during in the first half of 2016.