FDA Decision Due Next Year on Takhzyro for Children as Young as 2

An approval would make the therapy only treatment for US children under age 6

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has agreed to review an application from Takeda to extend the approval of Takhzyro (lanadelumab) for children as young as 2 with hereditary angioedema (HAE).

The application has been given priority review status and a decision from is expected in the first half of 2023, according to Takeda.

If the agency approves the extension, Takhzyro would become the first and only treatment approved in the U.S. to prevent swelling attacks in children with HAE younger than 6. The medication is currently approved for HAE patients ages 12 and older.

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“Unpredictable, debilitating and potentially life-threatening HAE swelling attacks can cause a physical and emotional toll on those living with this rare disorder; this is burdensome for young children and their caregivers,” Cheryl Schwartz, senior vice president of the U.S. rare disease business unit at Takeda, said in a press release.

Takeda’s supplemental biologics license application is supported by data from an open-label clinical trial called SPRING (NCT04070326) wherein 21 children with HAE, ages 2–11 were treated with Takhzyro. The therapy was administered via under-the-skin (subcutaneous) injection every two or every four weeks, with dosing based on age.

According to Takeda, the SPRING study is “the first and only open-label Phase 3 trial for HAE patients under the age of 12.”

The company announced results from the trial earlier this year. Most of the children in the study were completely free of swelling attacks over a year of treatment with Takhzyro and the overall rate of attacks decreased by 94.8%.

The therapy was generally well tolerated in these young patients, with the most common side effect being reactions at the injection site, such as pain or redness.

Takhzyro is an antibody-based therapy that works by blocking the activity of kallikrein, a protein that regulates levels of a signaling molecule called bradykinin. In HAE, excess bradykinin production triggers swelling attacks. Takhzyro is currently approved and available in more than 30 countries around the world, according to Takeda.