FDA Expands Approval For Takeda’s Takhzyro To Prevent Hereditary Angioedema Attacks

Takeda has announced the US FDA’s approval for the supplemental Biologics License Application (sBLA) for Takhzyro (lanadelumab-flyo) as a prophylactic against hereditary angioedema (HAE) attacks in patients 2 to under 12 years of age.

Takhzyro received its first green light in the US in 2018 to prevent HAE attacks in patients 12 years and older.  

Prior to the approval, there were no approved preventative treatments for children with HAE 2 to <6 years of age, while children between the ages of 6 and 12 required dosing every three to four days. 

Takhzyro now presents an inviting long-term alternative as HAE patients between 2 to <6 could take a recommended 150 mg/mL dose in a prefilled syringe every four weeks, while children between 6 and <12 could take the same dose once every two weeks. 

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Reducing the Rate of HAE Attacks by 94%

Hereditary angioedema is a rare genetic condition that causes recurrent and painful swelling in various parts of the body. The disease affects an estimated 1 in 50,000 people worldwide  Takhzyro, a monoclonal antibody, is designed to prevent swelling episodes. It binds to and inhibits plasma kallikrein, which inhibits the release of potent vasodilators such as bradykinin.

Takhzyro’s latest approval is based on extrapolated efficacy data from the Phase 3 Help study and additional data from the Phase 3 Spring study. In the Spring study, the most common side effects of the treatment were injection site pain (29%), injection site erythema (14%), and injection site swelling (5%). 

Takhzyro reduced the rate of HAE attacks by a mean of 94.8% compared to baseline, from 1.84 attacks per month to 0.08 attacks during the 52-week treatment period. 76.2%, or 16 of the 21 patients were attack-free with an average of 99.5% attack-free days. 

“Today’s approval of the expanded indication of TAKHZYRO represents a significant step forward for the HAE community as it helps some of its youngest patients who are living with the disease to have a long-term prophylaxis treatment available to them,” said Julie Kim, president, US Business Unit and US country head at Takeda.

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Joy Lin

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