Pluvia Receives European Orphan Drug Designation

Published: Fri May 31 2024

Pluvia newly announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted Orphan Drug Designation (ODD) for the company’s lead compound PBAS499, as confirmed by the European Commission Decision of 30 May 2024. The designation covers the treatment of hyperphenylalaninaemia, a slightly broader definition of the disease which includes PKU as a more severe subset of patients.

The European Commission grants ODD to products that treat a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 people in the EU and where no satisfactory treatment is available. Products receiving Orphan Drug Designation are eligible to receive market exclusivity for a period of up to 10 years in the EU upon approval, as well as eligibility for protocol assistance, reduced fees and access to the EU's centralized marketing authorization procedure. PKU affects between 1 to 2 in 10,000 in the EU, which amounts to 45,000 to 90,000 EU citizens.