FDA Approves Drug Treatment for Rare Hereditary Disease


Officials with the FDA announced Thursday approval of lanadelumab (Takhzyro) for treatment of hereditary angioedema (HAE).

Officials with the FDA announced Thursday approval of lanadelumab (Takhzyro) for treatment of hereditary angioedema (HAE). The drug is the first monoclonal antibody approved in the United States to treat patients aged 12 years and older with types I and II HAE.

HAE is a rare and serious genetic disease that affects people with low levels of and poorly functioning C1-INH proteins in the body. Individuals with the condition experience recurrent, unpredictable episodes of severe swelling in different areas of the body, including the stomach, limbs, face and throat.

According to the FDA, the approval of lanadelumab gives patients with HAE and healthcare professionals an additional treatment option. Lanadelumab is a plasma kallikrein inhibitor that prevents swelling attacks from occurring. Takhzyro is a fully human IgG1 monoclonal antibody made in recombinant Chinese Hamster Ovary cells.

HAE affects an estimated 1 in 50,000 men and women. Type I is the most common, and accounts for 85% of cases. Symptoms of HAE typically begin in childhood and worsen following puberty. Some patients may have many attacks each month, while others will go months without an attack, according to the FDA.

The agency based its approval on data from a multicenter, randomized, double-blind, placebo-controlled, parallel-group study in 125 patients with HAE. Patients with HAE who received Takhzyro had clinically meaningful and statistically significant reductions in the rate of investigator-confirmed HAE attacks compared to placebo over a 6-month treatment period.

The most common adverse drug reactions in patients with HAE taking Takhzyro in clinical trials are injection site reactions, upper respiratory infections, headache, rash, muscle pain, dizziness, and diarrhea.

The FDA granted Takhzyro’s application with Priority Review and Breakthrough Therapy designations. The medication also received an Orphan Drug designation.


FDA approves new treatment for rare hereditary disease [news release]. Silver Spring, MD; August 23, 2018: FDA website. http://www.pharmacytimes.com/link/211. Accessed August 23, 2018.

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