Takhzyro approved in US to treat HAE patients starting at age 2

FDA favors expanded use at 150 mg every 2 or 4 weeks, depending on age

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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The U.S. Food and Drug Administration (FDA) has extended the approval of Takhzyro (lanadelumab) to include children with hereditary angioedema (HAE) starting at age 2.

Takhzyro, available to patients ages 12 and older in the U.S. since 2018, is now the only approved treatment to prevent HAE swelling attacks in those younger than age 6.

“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 … to have a long-term prophylaxis [preventive] treatment available to them,” Julie Kim, president, business unit and country head for the U.S. at Takeda, the therapy’s developer, said in a company press release.

It also “underscores our confidence in Takhzyro, as well as our dedication to addressing the needs of HAE patients through continued research, clinical programs and real-world data collection,” Kim said.

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Takhzyro, an antibody-based therapy, is designed to block the activity of kallikrein, a protein that regulates levels of a signaling molecule called bradykinin. In HAE, excessive bradykinin production triggers the potentially life-threatening swelling attacks that patients experience.

The medication was initially approved by the FDA in August 2018 for patients ages 12 and older with HAE types 1 and 2. The recommended dose for this patient group is 300 mg, delivered as an under-the-skin injection once every two weeks.

Takeda later submitted a biologics license application seeking its approval for younger children, which the FDA placed under priority review in October.

This request was backed by efficacy data from the Phase 3 HELP trial (NCT02586805). Although its participants were ages 12 and older, additional analyses indicated that Takhzyro was as safe and effective in pediatric (ages 12 to 17) as in adult patients, and led to similar bloodstream exposure.

Further support came from findings in the open-label Phase 3 SPRING trial (NCT04070326) involving 21 children with HAE, ages 2 to 11. All were treated with 150 mg of Takhzyro delivered as an under-the-skin injection every four weeks (ages 2-5) or every two weeks (ages 6-11) for up to one year.

Children in the 6 to 11 age group could switch once monthly dosing if HAE attacks were well controlled after six months, and seven of the 17 patients in this group did so.

Trial results demonstrated that most children (76.2%) were free of swelling attacks over a year of treatment. The rate of HAE attacks declined by 94.8% , dropping from a mean of 1.84 attacks per month before treatment to 0.08 per month throughout Takhzyro’s use.

The treatment was also well tolerated, with the most common side effects being injection site reactions, such as pain (29%), redness (14%), or swelling (5%).

Recommended dosing for young patients is consistent with the trial’s dosing regimen: a 150 mg injection every four weeks for those ages 2 to 5, and generally every two weeks for those ages 6 to 11. The treatment will come in single-dose, pre-filled syringes containing 1 milliliter of liquid, and for children younger than age 12 is to be given by a caregiver or healthcare professional.

Takhzyro’s expanded approval also gives children access to an HAE treatment with longer dosing intervals, Takeda reported, nothing that current therapies approved for patients ages 6 and older typically require dosing every three to four days.

“Today’s approval for Takhzyro in pediatric patients as young as 2 years of age brings a welcome and important addition to treatment options available for children living with HAE,” said Anthony Castaldo, president and CEO of the U.S. Hereditary Angioedema Association.

Takhzyro is available in more than 60 countries worldwide, according to Takeda.