We are proud to announce that our product autologous adult bone marrow-derived non-expanded CD133+ haematopoietic stem cells (IGX1) was granted the Orphan Drug Designation (ODD) on March 15th 2017 by the European Medicines Agency (EMA). The product is aimed at the treatment of patients with the Asherman’s syndrome (AS), a rare disease defined by the presence of intrauterine adhesions. This is the first time that the EMA issues a positive opinion for this rare disease. Therefore IGX1 represents the only drug with ODD for this pathological condition.
We have been working together with Asphalion in this challenging process for ODD application. Asphalion regulatory affairs experts, Núria Coderch, Christopher Mann, Joel Montané, Jesús Delgado and Mireia Lorenzo, have been giving support to the Igenomix team formed by Prof. Carlos Simón (CSO), Dr. Xavi Santamaría (Scientific Project Director), Dr. Diana Valbuena and Carlos Gómez on the strategy and preparation of the regulatory procedure. Asphalion contributed in justification of AS as a rare disease, including the product´s medical plausibility, its chronically debilitating condition, the significant benefit to patients and the justification of the low European prevalence. Once the Committee for Orphan Medicinal Products (COMP) raised significant issues related to the application, Asphalion and IGENOMIX attended a face-to-face meeting at the EMA in London and successfully defended the application.
The EMA Orphan Drug Designation program provides a special status to drugs and biologics intended to treat, diagnose or prevent diseases and disorders that affect not more than 5 in 10,000 people in EU, are life-threatening or chronically debilitating and are of significant benefit to those affected by the condition.
This designation provides IGENOMIX significant benefits such as protocol assistance that will help to launch the Phase I/II Clinical Trial planned on 2018, market exclusivity against competition once the medicine is on the market, and fee reductions. These are incentives that allow medicines with low/small commercialization profit to be in the market under normal conditions, and thus yielding an overall benefit for society.
