Rare Disease Day 2021 - Biotech Spain

On the occasion of Rare Disease Day, at Asphalion we would like to highlight our commitment to the development of new treatments for rare diseases. We actively contribute to bringing treatments to the market and thus improving patient´s lives.

Regulatory Agencies, like the EMA and the FDA, have Orphan Drug Designation (ODD) programs that provide a special status to medicinal products intended to treat, diagnose or prevent diseases and disorders that are life-threatening or chronically debilitating, with very low prevalence (not more than 5 in 10,000 in EU) and are of significant benefit to those existing methods affected by the condition.

Our Scientific and Regulatory Affairs team contributes with its knowledge and expertise in supporting organizations throughout the regulatory pathway to obtain the ODD in EU and US. This status provides benefits for a fee reduction or exemption, protocol assistance, access to research grants and 10-year EU market exclusivity (after authorization).

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 more than 47 projects related to successful Orphan Drug Designations.

Currently, there are 300 million people living with one of over 6000 rare diseases worldwide. Today is a day to raise awareness and improve access to treatment for those individuals with rare diseases.