According to a story from Business Wire, the biopharmaceutical company Astria Therapeutics, Inc., recently announced that its experimental therapy navenibart has earned the US Food and Drug Administration’s Orphan Drug Designation for the treatment for hereditary angioedema (HAE). The decision follows promising findings in an ongoing Phase 1b/2 clinical trial. Initial results demonstrated a reduction in monthly attacks by up to 96% with one or two doses in a six-month period. Meanwhile, the treatment appears to have a favorable safety and tolerability profile.

About Navenibart

This therapy belongs to the monoclonal antibody class and inhibits plasma kallikrein. It is currently under development for hereditary angioedema as a preventative therapy. Navenibart is intended to achieve this affect with treatment administration every three to six months. The therapy has earned FDA Fast Track Designation in addition to Orphan Drug Designation.

About Orphan Drug Designation

Orphan Drug Designation is a special status organized by the FDA for therapies that are in development to treat rare diseases, which is defined as any condition affected under 200,000 people in the US. To qualify, the therapy must either address a need that isn’t being met by currently available medications or offer significant advantages in safety or effectiveness in comparison to current medicines. Orphan Drug designation grants benefits to the recipient company, such as more frequent communications with the agency, the waiving of certain fees, tax benefits, and a seven-year period of market exclusivity if the drug gains approval.

About Hereditary Angioedema

Hereditary angioedema is a genetic disorder which is characterized by chronic episodes of swelling that can affect multiple areas of the body. The condition is caused by mutations affecting the SERPING1 gene. Swelling attacks generally occur every two weeks or so; they can usually last for several days. Swelling may affect the limbs, digestive tract, face, and airway, with blockage of the airway being the most dangerous complication. Vomiting and abdominal pain may accompany attacks as well if the digestive tract is involved. Treatment involves reducing the likelihood for attacks to appear and preventing them from worsening when they do. Hereditary angioedema is typically only life-threatening if left untreated. Prevalence of the condition is estimated to be around one in 10,000 to one in 50,000, at least in the US and Canada. To learn more about hereditary angioedema, click here.