FDA Approves Lanadelumab for Rare Hereditary Angioedema

The US Food and Drug Administration (FDA) has approved lanadelumab (Takhzyro) for the treatment of types I and II hereditary angioedema in patients 12 years of age and older.

Hereditary angioedema (HAE) is a rare and serious genetic condition that affects individuals with low levels of C1-INH proteins in the body. The result is recurrent and unpredictable episodes of severe swelling in areas of the body including the stomach, limbs, face, and throat.

Lanadelumab is the first monoclonal antibody approved in the United States for the treatment of hereditary angioedema. In 2017, the FDA approved Haegarda, a subcutaneous C1 esterase inhibitor for the prevention hereditary angioedema attacks in adolescent and adult patients.

The FDA’s approval decision was based in part on data from a multicenter, randomized, double-blind, placebo-controlled, parallel-group study in 125 patients with hereditary angioedema. Patients who were randomized to receive lanadelumab experienced clinically meaningful and statistically significant reductions in HAE attacks (confirmed by investigators) compared to patients receiving placebo. The study included 6 months of treatment.

The most common adverse drug reactions reported in clinical trials include injection site reactions, upper respiratory infections, headache, rash, muscle pain, dizziness and diarrhea.

Lanadelumab has previously been granted Priority Review and Breakthrough Therapy designations by the FDA. The drug was given Orphan Drug designation because of the rare occurrence of hereditary angioedema.