European Commission grants orphan medicinal product designation for navenibart, Astria Therapeutics’ investigational therapy for the treatment of hereditary angioedema

Astra Therapeutics announced that the European Commission (EC) has granted Orphan Medicinal Product Designation (OMPD) to navenibart for the treatment of hereditary angioedema (HAE). Navenibart is a monoclonal antibody inhibitor of plasma kallikrein designed to provide long-acting attack prevention for HAE.

Initial results from the Phase 1b/2 ALPHA-STAR clinical trial of navenibart have demonstrated a favorable safety and tolerability profile and a reduction of monthly attack rates by 90-96% when dosed once or twice over six months.

Jill C Milne PhD, Chief Executive Officer of Astria, said: “We are very pleased to receive Orphan Medicinal Product Designation for navenibart in Europe,” said. We believe that OMPD status, in combination with the FDA’s designation of navenibart as an orphan drug in the United States, reinforces both the global unmet need for people with HAE and the potential for navenibart to change the way that people live with HAE. We look forward to the expected progression of navenibart into a Phase 3 trial in the first quarter of 2025 and continuing our efforts to bring a new treatment option to people living with HAE around the world.”

In the European Union (EU), OMPD is granted by the EC following a recommendation from the European Medicines Agency (EMA) via its Committee for Orphan Medicinal Products (COMP). OMPD status provides incentives for drug development sponsors to develop products for diagnosing, preventing or treating life-threatening or very serious conditions that are rare and affect not more than five in 10,000 persons in the EU. Sponsors may receive regulatory and financial incentives as well as eligibility for EMA assistance in optimizing the candidate’s clinical development.

(Source: Astria)