FDA approves Takhzyro to prevent attacks of hereditary angioedema. - Shire plc.

Shire plc announced that following priority review, the FDA has approved Takhzyro (lanadelumab-flyo) injection, for prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 years of age and older. HAE is a rare, genetic and potentially life-threatening disorder that can result in recurrent attacks of edema (swelling) in various parts of the body. Takhzyro is the only monoclonal antibody (mAb) that provides targeted inhibition of plasma kallikrein, an enzyme which is chronically uncontrolled in people with HAE, to help prevent attacks. The recommended starting dose of Takhzyro is 300 mg every two weeks. A dosing interval of 300 mg every four weeks is also effective and may be considered if the patient is well-controlled (e.g., attack free) for more than six months.

In the Phase III HELP (Hereditary Angioedema Long-term Prophylaxis) Study supporting FDA approval, Takhzyro reduced the number of monthly HAE attacks an average of 87% (n=27) vs. placebo (n=41) when administered at 300 mg every two weeks and 73% (n=29) vs placebo (n=41) when administered at 300 mg every four weeks (Adjusted P<0.001). takhzyro has a half-life of approximately two weeks and is administered as one subcutaneous self-injection every two weeks at the recommended starting dose. in clinical trials the majority of patients took one minute or less to complete the injection. the most commonly observed adverse reactions greater than 10 and higher than placebo associated with takhzyro were injection site reactions consisting mainly of pain erythema and bruising at the injection site upper respiratory infection headache rash myalgia dizziness and diarrhea.>

The FDA approval of Takhzyro was based on data from four clinical trials, including the HELP Study, the largest prevention study conducted to date in HAE. Of the patients who completed the HELP Study who received Takhzyro, 97% opted in to an ongoing open-label extension study designed to evaluate the long-term safety and efficacy of Takhzyro.