Takeda’s Takhzyro Demonstrates Positive Results in the Prevention of HAE Attacks in Children

Takeda reported late-breaking data from the phase 3 SPRING study demonstrating positive results of Takhzyro for preventing hereditary angioedema attacks in patients 2 to less than 12 years of age, who experienced 948 percent fewer attacks during the 52-week treatment period compared to baseline and were consistent with earlier studies in adult and adolescent patients.

Photo: Ming Yu, Global Clinical lead, Takeda

The data were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2022.

Hereditary angioedema (HAE) is a rare genetic disorder that results in recurring attacks of edema, or 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 attacks may occur early in childhood. Potentially fatal upper airway angioedema has been reported in patients as young as 3 years old. HAE diagnosis can take an average of 8.4 years after symptom onset. HAE affects an estimated 1 in 50,000 people worldwide and is often under recognized, under diagnosed, and under treated. There are currently no long-term prophylactic treatments approved for HAE patients younger than 6 years.

“HAE is a rare condition where unpredictable symptoms like severely debilitating swelling can take a toll on children both physically and emotionally,” said Marcus Maurer, professor of Dermatology and Allergy Charité – Universitätsmedizin Berlin, Germany, and principal investigator of the SPRING study. “In the SPRING study, we saw a majority of children who had been suffering nearly two HAE attacks per month on average at baseline, who were then attack-free throughout the 52-week trial of treatment with Takhzyro.”

The SPRING study is a multicenter, open-label phase 3 study to evaluate the safety and pharmacokinetics (PK) of Takhzyro (lanadelumab) in patients aged 2 to less than 12 years with HAE. Participants aged 2 to less than 6 years received lanadelumab at a dose of 150 mg every four weeks over a 52-week treatment period. Participants aged 6 to less than 12 years received lanadelumab at a dose of 150 mg every two weeks over a 52-week treatment period and had the option to reduce the dose frequency to every four weeks if they were well controlled (e.g., attack-free) for 26 weeks. HAE attack rates were analyzed using descriptive statistics. Clinical outcomes (prevention of HAE attacks) were measured as a secondary objective.

Takhzyro reduced the rate of HAE attacks in children by a mean of 94.8 percent compared to baseline, from 1.84 attacks per month to 0.08 attacks during treatment. The majority of patients (76.2 percent) were attack-free during the 52-week treatment period with an average of 99.5 percent attack-free days. No deaths or serious treatment-emergent adverse events (TEAEs) were reported during the study, and no patients withdrew from the study due to TEAEs. The most commonly reported TEAE was injection site pain, and most TEAEs were mild or moderate in severity. These results are consistent with the favorable efficacy and safety profile of Takhzyro observed in earlier studies with adult and adolescent patients.

“The SPRING study data reinforce the safety and efficacy of Takhzyro across a broad range of patients living with HAE,” said Ming Yu, Global Clinical lead, Takeda. “We are encouraged by these findings as they demonstrate the potential of long-term prophylaxis treatments to reduce recurrent HAE attacks in children as young as two who currently have no approved long-term preventative treatment option.”

Takeda says it will submit the data to global regulatory authorities to evaluate a potential label expansion for Takhzyro to include the younger patient population.

Author: Rare Daily Staff