FDA approves expanded use of Takhzyro for children with hereditary angioedema
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The FDA has approved a supplemental biologics license application to expand the use of lanadelumab-flyo for prophylaxis of hereditary angioedema attacks in children aged 2 to younger than 12 years, according to a press release.
Lanadelumab-flyo (Takhzyro, Takeda) is the first prophylaxis treatment for hereditary angioedema (HAE) for this age group, with recommended doses of 150 mg/1 mL in a single-dose prefilled syringe every 4 weeks for patients aged 2 to younger than 6 years and every 2 weeks for patients aged 6 to younger than 12 years.
Prior to this approval, children aged 6 to younger than 12 years required dosing every 3 to 4 days, whereas those aged 2 to younger than 6 years had no approved prophylaxis treatment.
“[This] 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,” Anthony J. Castaldo, president and CEO of the US Hereditary Angioedema Association, said in the press release.
Results from the open-label phase 3 SPRING study, which involved 21 patients with HAE aged 2 to younger than 12 years, supported the approval.
The most common treatment-related emergent adverse events in the study included injection site pain (29%), injection site erythema (14%), injection site swelling (5%), administration site pain (5%) and injection site reaction (5%).
Treatment also reduced HAE attack rates by a mean of 94.8% compared with baseline, a change of 1.84 attacks per month to 0.08 attacks during the 52-week treatment period. Also, 76.2% (n = 16) of the patients experienced an average of 99.5% attack-free days.
Results from the phase 3 HELP study of patients aged 12 to younger than 18 years in addition to other pharmacokinetic analyses of similar drug exposures between adult and pediatric patients supported the approval as well.
The FDA first approved lanadelumab-flyo for prophylaxis to prevent HAE attacks in adults and children aged 12 years and older in 2018. It is now available in more than 60 countries.
Reference:
- Maurer M, 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 at: European Academy of Allergy and Clinical Immunology Hybrid Congress; July 1-3, 2022; Prague.
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Hilary Longhurst, MA, MB BS, FRCP, PhD, FRCPath
This is great news. Children with HAE are so often overlooked as the condition is, erroneously, considered to be mild in childhood. However, many children suffer disruption to their education and enjoyment of their childhood from disfiguring angioedematous swellings or disabling abdominal pain, and fatalities have occurred. Access to lanadelumab-flyo will provide that freedom from attacks that our children need to be able to participate fully in education and sport and to grow into confident, healthy adults.
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