Proteostasis Therapeutics Inc. said the European Commission granted the orphan drug designation to its therapy PTI-428 for treating cystic fibrosis.
Cystic fibrosis is a rare and inherited disease characterized by the buildup of sticky mucus in the lungs and digestive system, which leads to persistent lung infections and other complications.
The designation is based on a positive opinion by the European Medicines Agency's Committee for Orphan Medicinal Products. The regulator grants the orphan drug tag for therapies that treat serious disorders affecting no more than five in 10,000 people in the EU.
Once granted, companies benefit from incentives such as a 10-year marketing exclusivity in the EU, eligibility for protocol assistance, reduced fees and access to the EU's centralized marketing authorization procedure.
PTI-428 also previously received orphan drug, breakthrough therapy and fast track designations from the U.S. Food and Drug Administration.
Cambridge, Mass.-based Proteostasis Therapeutics develops small molecule therapeutics that can address malfunctions in protein handling processes within the cells, which can cause a wide range of diseases, including cancer.