On the Rare Disease Day, Asphalion would like to show our commitment to the development of new treatments for rare diseases. We are actively contributing to bringing the treatments to the market and thus improving patients’s lives.
Health Agencies, like EMA and FDA, run Orphan Drug Designation (ODD) programmes to provide a special status to drugs and biologics intended to treat, diagnose or prevent diseases and disorders that are life-threatening or chronically debilitating and are of significant benefit to those affected by the condition.
Our Scientific and Regulatory Affairs team is passionate and dedicated, supporting organisations throughout the regulatory pathway to obtain the ODD. This status provides benefits for a fee reduction or exemption, protocol assistance, access to research grants and 10-year market exclusivity (after authorisation).
These incentives allow medicines with small commercial potential to be on the market under normal conditions, and thus yielding an overall benefit for the society.
So far, our experts have participated in 20 successful Orphan Drug Designations. A notable example is Igenomix ODD granted in 2017 for its product indicated for Asherman’s Syndrome. It was the first time that the EMA issued a positive opinion for this rare disease.
Asphalion is working on many projects related to the research and development of therapies for rare diseases. We are very proud to have been awarded a grant for the MYOCURE project as part of the H2020 topic PHC-14-2015 – New therapies for rare diseases by the European Commission. This European Commission funded initiative is aimed at developing an innovative gene therapy platform to cure rare hereditary muscle disorders. Eight organisations, including universities, public institutions and private companies from five different countries work together on this project, which is estimated to have a direct impact on 20,000 people in EU, who are suffering from MTM or GSD II. Moreover, MYOCURE will contribute to the IRDiRC objective of achieving 200 new therapies for rare diseases by year 2020.
The Rare Disease Day takes place today and it is celebrated worldwide. The main purpose is to raise the awareness about rare diseases and their impact on patients’ lives in order to motivate responsiveness of the public in general, but more importantly, the policy makers, public authorities, industry representatives, researchers and healthcare professionals.
At present, there have been more than 7,000 different rare diseases diagnosed, affecting 1 out of every 2,000 people in the world. Only in Europe, 30 million people suffer from a rare disease. 80% of these diseases have a genetic origin, and 65% are serious and disabling.
#ShowYourRare #ShowYourCare #RareDiseaseDay