Takeda Pharmaceutical in the Drug Discovery & Development Pharma 50Takeda (NYSE:TAK) recently announced that in the open-label Phase 3 SPRING study, children aged 2 to 12 had 94.8% fewer hereditary angioedema (HAE) attacks when receiving Takhzyro (lanadelumab-flyo) over 52 weeks compared to baseline

The primary objectives of the SPRING study was to evaluate the safety and pharmacokinetics of Takhzyro in HAE patients aged 2 to <12 years. The prevention of HAE attacks was a secondary outcome measure.

FDA first approved Takyzyro for heditary angiodema in 2018 for patients 12 years and older with types I and II HAE. The drug won marketing authorization  throughout the European Union that same year.

To learn more about the study, we reached out to Dr. Marcus Maurer, professor of dermatology and allergy at Charité – Universitätsmedizin Berlin, Germany and principal investigator of the SPRING study.

Drug Discovery & Development (DDD): The 94.8% reduction in HAE attacks for children aged 2 to <12 seems remarkable. Could you help put this figure in context and how it can reduce the unpredictable symptoms associated with HAE?

Maurer: Yes, we were very pleased with the data as we saw attacks among children reduce from 1.84 attacks per month to 0.08 attacks during treatment. Additionally, a majority (76.2%) of children were attack-free for the entirety of the treatment period with an average of 99.5% attack free days—promising results as we work to reduce the unpredictable and potentially fatal symptoms of HAE.

DDD: If Takhzyro wins regulatory approval for use in HAE patients younger than 6 years, how do you think availability of the drug would affect treatment of this demographic in particular?

Maurer: There currently are no long-term prophylactic treatments approved for HAE patients younger than 6 years old. Because of this, many children are experiencing unpredictable symptoms like debilitating swelling, including life-threatening laryngeal swelling as young as 3 years old. The potential approval of a long-term prophylactic treatment would provide pediatric patients and their caregivers the option to prevent HAE attacks rather than simply react to attacks as they happen.

DDD: The SPRING study results were in line with the favorable efficacy and safety profile of TAKHYZRO observed in earlier studies involving adults and adolescents. Could you provide a brief overview of its safety profile in younger children?

Maurer: The primary objective of the open-label, multicenter, Phase 3 SPRING study was to evaluate the safety and pharmacokinetics (PK) of Takhzyro in patients aged 2 to <12 years with HAE. Clinical outcomes were defined as prevention of HAE attacks and measured as a secondary objective. 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 — all of which was in line with the safety results seen in the older populations.