Release Date: 07-Oct-2021
The European Commission (EC) has given orphan designation to AOC 1001 for the treatment of myotonic dystrophy type 1 (DM1). The decision by the EC follows the U.S. Food and Drug Administration (FDA) also granting orphan drug designation to AOC 1001 earlier this year. The drug AOC 1001 represents to new category RNA therapies called antibody oligonucleotide conjugates (AOCs).
Orphan designation is given to investigational therapies that have the power to treat rare and life-threatening diseases that affect less than five in 10,000 patients in the European Union. It provides some benefits to companies, like clinical protocol advice, fee reductions, and ten years of protection from competition following regulatory approval.