U.S. FDA Approves Takeda’s TAKHZYRO® (lanadelumab-flyo) to Prevent Hereditary Angioedema (HAE) Attacks in Children 2 Years of Age and Older
TAKHZYRO is the First and Only Prophylaxis Treatment Approved in the
U.S.for Children 2 to <6 Years of Age With HAE1-4
- Approval Supported by Extrapolation of Efficacy Data From the Phase 3 HELP Study With Additional Data From the Phase 3 SPRING Study in Pediatric Patients 2 to <12 Years of Age1
- HAE is a Rare, Debilitating and Potentially Life-Threatening Condition That Causes Unpredictable and Serious Angioedema Attacks That May Occur Early in Childhood5,6
HAE attacks can involve serious and debilitating swelling in the abdomen, face, feet, genitals, hands and throat.5,7 Potentially fatal upper airway angioedema has been reported in patients as young as 3 years of age.6 In a survey from 2017 (N=445), the average HAE diagnosis took an average of 8.4 years after symptom onset.8 In this study of patients with HAE, 50% experienced anxiety, 34% had difficulty with social activity and 58% reported symptoms negatively affected career advancement.8
“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
The sBLA approval was supported by extrapolation of efficacy data from the HELP Study, a Phase 3 trial that included patients 12 to <18 years of age, and additional pharmacokinetic analyses showing similar drug exposures between adults and pediatric patients, as well as safety and pharmacodynamic data from the SPRING Study, an open-label Phase 3 trial in 21 HAE patients 2 to <12 years of age.1 The primary objectives of the SPRING Study were the safety and pharmacokinetics of TAKHZYRO.9 The most common treatment-related treatment emergent adverse events in the study were injection site pain (29%), injection site erythema (14%), injection site swelling (5%), administration site pain (5%) and injection site reaction (5%).9 The prevention of HAE attacks was measured as a secondary objective.9 TAKHZYRO reduced the rate of HAE attacks in pediatric patients by a mean of 94.8% compared to baseline, from 1.84 attacks per month to 0.08 attacks during the 52-week treatment period (N=21).9 The majority of patients (76.2%, n=16) were attack-free with an average of 99.5% attack-free days.9 These efficacy results are from an open-label, non-controlled trial, and the study was not designed for statistical hypothesis testing. Further confirmatory studies are required to draw any conclusions from these data.
“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 who are living with the disease to have a long-term prophylaxis treatment available to them,” said
TAKHZYRO was originally approved in the
About TAKHZYRO® (lanadelumab-flyo) Injection
TAKHZYRO is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein activity and is indicated for prophylaxis to prevent attacks of
TAKHZYRO Safety Information for
TAKHZYRO may cause serious side effects, including allergic reactions. Call your healthcare provider or get emergency help right away if you have any of the following symptoms:
- difficulty breathing
- chest tightness
- fast heartbeat
The most common side effects seen with TAKHZYRO were injection site reactions (pain, redness, and bruising), upper respiratory infection, headache, rash, dizziness, diarrhea, and muscle aches.
These are not all the possible side effects of TAKHZYRO. For more information, ask your healthcare provider or pharmacist. You may report side effects to FDA at 1-800-FDA-1088.
TAKHZYRO has not been studied in pregnant or breastfeeding women. Talk to your healthcare provider about the risk of taking TAKHZYRO if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed.
Please see full Prescribing Information , including information for patients.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare genetic disorder that results in recurring attacks of edema – swelling – in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. The swelling can be debilitating and painful.5,7 Attacks that obstruct the airways can cause asphyxiation and are potentially life-threatening.7 HAE affects an estimated 1 in 50,000 people worldwide.12 It is often under-recognized, under-diagnosed and under-treated.12
Takeda is a global, values-based, R&D-driven biopharmaceutical leader headquartered in
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TAKHZYRO® (lanadelumab-flyo) injection.
HAEGARDA® (C1 Esterase Inhibitor Subcutaneous [Human]).
CINRYZE® (C1 esterase inhibitor [human]).
- Farkas H, Martinez-Saguer I, Bork K, et al. International consensus on the diagnosis and management of pediatric patients with hereditary angioedema with C1 inhibitor deficiency. Allergy. 2017 Feb;72(2):300-313. doi:10.1111/all.13001.
Busse PJ, Christiansen SC, Riedl MA, et al.
US HAEA Medical Advisory Board2020 guidelines for the management of hereditary angioedema. J Allergy Clin Immunol Pract. 2021 Jan;9(1):132-150.e3. doi: 10.1016/j.jaip.2020.08.046.
- Bork K, Hardt J, Schicketanz KH, Ressel N. Clinical studies of sudden upper airway obstruction in patients with hereditary angioedema due to C1 esterase inhibitor deficiency. Arch Intern Med. 2003 May;163(10):1229–35. doi:10.1001/archinte.163.10.1229.
- Banerji A. Hereditary angioedema: Classification, pathogenesis, and diagnosis. Allergy Asthma Proc. 2011 Nov-Dec;32(6):403–407. doi:10.2500/aap.2011.32.3492.
Banerji A, Davis KH, Brown TM, et al. Patient-reported burden of hereditary angioedema: findings from a patient survey in
the United States. Ann Allergy Asthma Immunol. 2020 Jun;124(6):600-607. doi:10.1016/j.anai.2020.02.018.
Maurer M, Lumry WR, Li HH, et al. Efficacy and safety of lanadelumab in pediatric patients aged 2 to <12 years with hereditary angioedema: results from the open-label, multicenter Phase 3 SPRING study. Presented
July 1-3, 2022 Prague, Czech Republicat European Academy of Allergyand Clinical Immunology Hybrid Congress2022.
Takeda Pharmaceuticals. Data on File.
- Banerji A, Bernstein JA, Johnston DT, et al; for HELP OLE Investigators. Long-term prevention of hereditary angioedema attacks with lanadelumab: the HELP OLE Study. Allergy. 2022 Mar;77(3):979-990. doi:10.1111/all.15011.
Bork K. Hereditaryangioedema: an update on causes, manifestations, and treatment. Br J Hosp Med. 2019 Jul;80(7):391-398. doi:10.12968/hmed.2019.80.7.391.
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