Pharma Focus America

U.S. FDA Approves Takhzyro® to Prevent Hereditary Angioedema (HAE) Attacks

Friday, February 10, 2023

Takeda said that the supplemental Biologics License Application (sBLA) for Takhzyro (lanadelumab-flyo) as a preventative against hereditary angioedema (HAE) attacks has been authorized by the U.S. Food and Drug Administration.

The first prophylactic drug authorized for use in children with HAE between the ages of 2 and under 6 is takhzyro. Prior to its approval, the only approved routine prophylactic therapy options for children aged 6 to under 12 years old were doses every three to four days.

A dose of 150 mg/mL solution in a single-dose prefilled syringe is recommended every four weeks for patients aged two to less than six years, and every two weeks for patients aged six to less than twelve years.

Hereditary angioedema (HAE), a genetic disorder, can result in severe and disabling swelling in the hands, feet, genitalia, belly, face, and throat. The illness is thought to afflict 1 in 50,000 people worldwide.

To reduce swelling episodes, a monoclonal antibody named Takhzyro is produced. In doing so, it prevents plasma kallikrein from generating potent vasodilators like bradykinin.

The Phase III SPRING Study's safety and pharmacodynamic data, which included 21 HAE patients between the ages of 2 and less than 12 years, as well as extrapolation of efficacy data from the Phase III HELP Study, which included patients aged 12 to less than 18 years, as well as additional pharmacokinetic analyses showing comparable drug exposures between adult and paediatric patients, all supported the sBLA approval.

According to the SPRING study, the most common side effects of the therapy were injection site soreness (29%), injection site erythema (14%), and injection site edoema (5%). Takhzyro reduced the frequency of HAE attacks from 1.84 per month to 0.08 attacks by a mean of 94.8% over the course of the 52-week treatment period, compared to the baseline. 16 out of the 21 patients, or 76.2%, had an average of 99.5% attack-free days.

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