Today is the Rare Disease Day! As a Rare Disease Day’s Friend, from Asphalion we would like to show our commitment to the development of new treatments for rare diseases.
Our Scientific and Regulatory Affairs team is passionate and dedicated, supporting organizations throughout the regulatory pathway to obtain the Orphan Drug Designation (ODD) status. Health Agencies run ODD programs to provide a special status to drugs and biologics that could be used for treating, preventing or diagnosing a rare and serious condition. This status provides benefits for a fee reduction or exemption, protocol assistance, access to research grants and 10-year market exclusivity, 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.
Asphalion has extensive experience in ODD applications and has successfully participated in several ODD projects. The most recent example is the acquisition in 2018 of the ODD status of Ciclopirox, for the treatment of patients with Congenital Erythropoietic Porphyria. This is the first time that the agencies issue a positive opinion for this rare disease. Therefore, Ciclopirox represents the only drug with ODD status for this genetic condition.