Takeda Reports Positive Phase 3 Data for Takhzyro in Children Ages 2 to Less Than 12 with HAE

Rare Daily Staff

Japanese pharmaceutical Takeda reported that the phase 3 SHP643-301 study evaluating the safety profile and pharmacokinetics Takhzyro in patients 2 to less than 12 years of age is complete and has met its objectives.

The safety profile was consistent with that seen in the clinical program for patients 12 years of age and older; there were no serious adverse events and no dropouts due to adverse events.

The study also successfully reached the secondary objective evaluating the clinical activity/outcome of Takhzyro in preventing hereditary angioedema (HAE) attacks as well as characterizing the pharmacodynamics of Takhzyro in pediatric subjects 2 to less than 12 years of age.

“We are encouraged by these results, as each objective met instills further confidence in the potential to bring a treatment option to this vulnerable population,” said Ashley Yegin, head of the Global Medical Unit for HAE, Global Medical Affairs, Takeda.

Hereditary angioedema (HAE) is a rare genetic disorder that results in recurring attacks of oedema – swelling – in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. The swelling can be debilitating and painful. Attacks that obstruct the airways can cause asphyxiation and are potentially life threatening. HAE affects an estimated 1 in 50,000 people worldwide. It is often under recognized, under diagnosed and under treated.

Takhzyro (lanadelumab) is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein and is indicated for routine prevention of recurrent attacks of HAE in patients aged 12 years and older. It was studied in one of the largest prevention studies in HAE with the longest active treatment duration, and Takhzyro consistently demonstrated HAE attack reduction. It is formulated for subcutaneous administration and has a half-life of approximately two weeks.

SHP643-301, also known as the SPRING study, is a multicenter, open-label phase 3 study to evaluate the safety, PK and PD of Takhzyro for prevention against acute attacks of HAE in pediatric patients 2 to less than 12 years of age. Participants aged 2 to less than 6 years received lanadelumab at a dose of 150 milligrams (mg) every 4 weeks over a 52-week treatment period. Participants aged 6 to less than 12 years received lanadelumab at a dose of 150 mg every 2 weeks over a 52-week treatment period.

Takhzyro is approved in the U.S. and EU for the treatment of HAE in patients 12 years of age and older.

Takeda plans a global regulatory filing in 2022 for children ages 2 to less than 12 year of age.

Photo: Ashley Yegin, head of the Global Medical Unit for HAE, Global Medical Affairs, Takeda