Astria Gets FDA Orphan Designation for Navenibart in Hereditary Angioedema

By Colin Kellaher

Astria Therapeutics won Food and Drug Administration orphan-drug designation for navenibart, its lead program candidate, for the treatment of the rare genetic disease hereditary angioedema.

The Boston biopharmaceutical company on Monday said there is a significant unmet need for people living with hereditary angioedema, which can cause painful and debilitating attacks of tissue swelling in various parts of the body.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. It also provides for an extended marketing exclusivity period against competition.

Astria said initial results from a Phase 1b/2 study of navenibart have shown a favorable safety and tolerability profile, along with a reduction of monthly attack rates by 90% to 96% when dosed once or twice over six months.

The company said it expects to share additional study results later this year and to advance navenibart into a Phase 3 study launching in the first quarter of 2025.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

September 30, 2024 08:36 ET (12:36 GMT)

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