Takeda’s TAKHZYRO approved by EC for pediatric hereditary angioedema

Takeda has announced a significant advancement in the treatment of Hereditary Angioedema (HAE), with the European Commission’s approval of TAKHZYRO (lanadelumab) for routine prevention in patients aged 2 years and older. This approval marks the first long-term prophylactic HAE treatment available in the European Economic Area for patients under six years of age, broadening the medication’s initially approved use.

Introduction of New Strength for Pediatric Patients

Accompanying the extended indication for pediatric patients is the introduction of a new 150 mg strength of Lanadelumab solution for injection in a pre-filled syringe. This specific dosage is intended for patients aged 2 years and older, weighing less than 40 kg, to prevent angioedema attacks associated with HAE.

Clinical Data Backing the Expanded Indication

The approval for expanded use is supported by data from the Phase 3 Study SHP643-301, coupled with extrapolated data from the pivotal Adult and Adolescent Study DX-2930-037, as well as quality data underpinning the new 150 mg pre-filled syringe formulation. The safety and efficacy of TAKHZYRO in preventing HAE attacks in pediatric patients have been demonstrated, affirming a positive benefit-risk balance.

Global Recognition for Pediatric Treatment

The European Medicines Agency (EMA) follows the U.S. Food and Drug Administration’s (FDA) earlier approval this year for TAKHZYRO’s expanded use in the pediatric population. This marks a crucial step in addressing the acute unmet need in treating potentially fatal upper airway angioedema in young patients.

Takeda’s Commitment to Pediatric HAE Patients

Didier Relin, Head of International Regulatory at Takeda, emphasized the significance of this expanded label, highlighting TAKHZYRO as a new preventative treatment option for pediatric HAE patients, which can be administered at home with caregiver support.

Lanadelumab’s Role in HAE Treatment

Lanadelumab, a fully human monoclonal antibody, plays a key role in decreasing plasma kallikrein and has been studied extensively in one of the largest HAE prevention studies with the longest active treatment duration. It is formulated for subcutaneous administration and has a half-life of approximately two weeks, making it suitable for self-administration or administration by a trained caregiver.

The approval of TAKHZYRO for pediatric use in Europe represents a major milestone in the treatment of HAE, offering new hope and enhanced quality of life for young patients and their families.