WASHINGTON -- A monoclonal antibody drug for hereditary angioedema (HAE) , making it the sixth agent to reach the U.S. market for the rare genetic disorder.
Lanadelumab (Takhzyro), made by Shire, was approved for preventing attacks in patients age 12 and older with types I and II HAE.
The drug inhibits plasma kallikrein, a target shared by the previously approved drug ecallantide (Kalbitor). Four other agents are also approved for HAE, either as preventive treatments or to shut down acute attacks: the complement C1 inhibitor agents Cinryze, Berinert, and Ruconest, and the bradykinin receptor antagonist icatibant (Firazyr).
Approval of lanadelumab was based on a 125-patient, placebo-controlled trial lasting 6 months in which the active drug provided "clinically meaningful and statistically significant reductions in the rate of investigator-confirmed HAE attacks," the FDA said.
Shire received orphan drug designation for lanadelumab -- which has also been that were previously approved.
The condition is believed to affect about 1 in 50,000 people. Attacks take the form of sudden, often painful swelling in various parts of the body, including the abdomen, extremities, throat, and face. It is often mistaken for allergic reactions, but it actually results from a deficiency in endogenous C1 inhibitor protein, which allows over-activation of the complement pathway.