The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending DX-2930 for designation as an orphan medicinal product for the treatment of HAE. Dyax is developing DX-2930, an investigational fully human monoclonal antibody inhibitor of plasma kallikrein (pKal), as a subcutaneous injection for prevention of HAE attacks.
“There is a significant unmet medical need for a prophylactic treatment option for HAE,” said Burt Adelman, M.D., Executive Vice President of Research and Development and Chief Medical Officer at Dyax. “Our regulatory strategy supports our global development plan for DX-2930 and our goal for providing an improved therapy for patients with HAE around the world. We look forward to initiating a Phase 3 clinical trial for DX-2930 for HAE prophylaxis by year-end 2015.”
Under the EMA guidelines, the COMP adopts an opinion on the granting of orphan drug designation, after which the opinion is submitted to the European Commission (EC) for the endorsement of the opinion. Orphan drug designation by the EC provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the EU, and where no satisfactory treatment exists, or where a treatment exists, a new treatment may provide a significant benefit to patients affected by the condition. Additionally, this designation provides up to 10 years of market exclusivity if the product candidate is approved for marketing in the European Union and the orphan designation is maintained. Orphan status also permits EMA assistance in optimizing the candidate’s clinical development through participation in clinical trial design and preparation of the product marketing application. Orphan Medicinal Product designation does not change the standards for approval.
DX-2930 has previously been granted Orphan Drug, Fast Track and Breakthrough Therapy designations from the U.S. Food and Drug Administration (FDA) for the prevention of attacks of HAE.