European Medicines Agency (EMA) grants Orphan Designation for AOC 1001

Avidity Biosciences has announced that the European Commission (EC) has granted orphan designation for Antibody Oligonucleotide Conjugates (AOC) 1001. The US Food and Drug Administration (FDA) also granted orphan drug designation to AOC 1001 earlier this summer, the company said in a statement.

The FDA cleared Avidity to proceed with the phase-I/II Marinatm trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1) in the United States, the statement added.

“We are pleased that both the EC and the FDA have granted AOC 1001 Orphan Designation, further validating AOCs as a powerful new class of drugs that can bring benefit to people suffering from untreated and undertreated diseases,” said Sarah Boyce, president and Chief Executive Officer, Avidity.

She added, “AOC 1001 is designed to address the root cause of DM1 and has the potential to be a first-in-class treatment for people living with this debilitating rare disease.”

The EC grants orphan designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically-debilitating diseases or conditions that impact fewer than five in 10,000 patients in the European Union (EU). Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval, said the statement.