The European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on BioCryst Pharmaceuticals, Inc.‘s application for orphan designation of BCX7353 for the treatment of HAE. In addition, the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted a Promising Innovative Medicine (PIM) designation to BCX7353.
The positive opinion issued by COMP is expected to be adopted by the European Commission within 30 days. Orphan Drug Designation in Europe is available to companies developing products intended to treat a life-threatening or chronically debilitating condition that affects fewer than five in 10,000 persons in the European Union. This designation allows for financial and regulatory incentives that include a 10-year period of marketing exclusivity in the EU after product approval. BCX7353 has previously received Orphan Drug Designation in the United States from the Food and Drug Administration.
A PIM designation is an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme (EAMS) in the United Kingdom, which supports products intended for the treatment, diagnosis or prevention of a life-threatening or seriously debilitating condition, with the potential to address an unmet medical need.
“We are pleased to receive these important regulatory designations granted to BCX7353. These provide further support for the potential for BCX7353 to address a clear unmet medical need with an oral treatment for patients with HAE,” said Jon P. Stonehouse, President & CEO.