FDA Approves New Treatment for Rare Hereditary Disease

Officials with the FDA have approved the first monoclonal antibody, lanadelumab (Takhzyro, Shire) for the treatment of patients 12 years and older with types 1 and 2 hereditary angioedema (HAE), according to the agency.

Officials with the FDA have approved the first monoclonal antibody, lanadelumab (Takhzyro, Shire) for the treatment of patients 12 years and older with types 1 and 2 hereditary angioedema (HAE), according to the agency.

HAE, a rare hereditary disease, affects individuals with low levels of and poorly functioning C1-INH proteins in the body. According to the FDA, HAE occurs in an estimated 1 in 50,000 individuals, and type 1 accounts for 85% of cases. Patients with the disease can experience recurrent, unpredictable episodes of severe swelling in different areas of the body, including the stomach, limbs, face, and throat.

The approval was based on data from a clinical trial including 125 patients with HAE. According to the data, patients who were treated with lanadelumab demonstrated clinically meaningful and statistically significant reductions in the rate of investigator-confirmed HAE attacks, compared with a placebo over a 6-month period.

Of the 125 patients in the trial, 38 had a baseline attack rate of 1 to < 2 attacks per month. Compared with a placebo, patients taking 150 mg every 4 weeks had a 51% reduction in attacks, patients taking 300 mg every 4 weeks had an 80.4% reduction, and patients taking 300 mg every 2 weeks had a 92.8% reduction. Twenty-two patients had a baseline attack rate of 2 to < 3 per month. These patients had a 90.6% reduction in attack rates using 150 mg every four weeks, 77% with 300 mg every four weeks and 88.2% with 300 mg every two weeks, compared with a placebo. For patients with a baseline attack rate of ≥ 3 attacks per 4 weeks, patients taking 150 mg every four weeks had a 78.8% reduction in attacks. Patients receiving 300 mg every four weeks experienced a 70.8% reduction. Patients receiving 300 mg every two weeks had an 85.9% reduction compared to placebo. Findings also demonstrated an improvement in quality of life in patients with HAE receiving lanadelumab across all dosing regimens versus placebo.

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