Astria Therapeutics, Inc. announced that navenibart (STAR-0215) has been granted Orphan Drug Designation for the treatment of hereditary angioedema (HAE) by the FDA. 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.
“Receiving orphan drug designation for navenibart is an important affirmation of our belief that there is a significant unmet need for people living with HAE,” said Jill C. Milne, Ph.D., Chief Executive Officer. “We believe navenibart has the potential to be the market-leading HAE treatment because of its trusted mechanism and modality, efficacy observed to date, and low treatment burden with infrequent dosing, and think that navenibart could change the way that people live with their HAE. We expect to share additional results from the ALPHA-STAR trial in Q4 and to progress navenibart into a Phase 3 trial initiating in the first quarter of 2025.”
The FDA’s Orphan Drug Designation program grants orphan status to medicines intended for the safe and effective prevention, diagnosis, or treatment of rare diseases or disorders that affect fewer than 200,000 people in the United States. Orphan status provides sponsors with development and commercial incentives for designated compounds and medicines.
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