KING OF PRUSSIA, PA — CSL plans to seek approval to expand ANDEMBRY to children ages 2 through 11 after a Phase 3b study found most participants remained free of hereditary angioedema attacks during 12 months of treatment, potentially extending the preventive therapy to a younger patient population.
The biotechnology company (ASX: CSL; USOTC: CSLLY) expects to begin submitting applications to health authorities during the first half of its fiscal year. ANDEMBRY, or garadacimab-gxii, is currently approved in more than 40 countries for patients age 12 and older.
The multicenter, open-label study enrolled 22 children with hereditary angioedema, or HAE. Sixteen participants ages 6 through 11 received 100 milligrams of ANDEMBRY once a month, while six children ages 2 through 5 received the same dose every two months.
CSL reported a safety and tolerability profile consistent with earlier studies and observed treatment responses throughout the study population. The company did not disclose detailed efficacy rates, adverse-event data or statistical analyses in its top-line report.
“The top-line results support our plans to seek an expanded pediatric indication for ANDEMBRY in children aged 2 to 11 years,” Dr. Bill Mezzanotte, CSL’s executive vice president and head of research and development, stated.
CSL plans to present the full results at a scientific congress and submit the findings to a peer-reviewed journal.
ANDEMBRY is a monoclonal antibody designed to inhibit activated factor XII, a plasma protein near the beginning of the biological pathway that produces swelling attacks in people with HAE. The drug is administered by subcutaneous injection as a long-term preventive treatment.
HAE is a rare genetic disorder associated with deficient or dysfunctional C1 esterase inhibitor. The condition can cause recurrent swelling of the face, abdomen, extremities and airway, with attacks involving the throat posing a risk of asphyxiation or death.
The disorder is estimated to affect between one in 10,000 and one in 50,000 people. Current prescribing information notes that ANDEMBRY’s safety and effectiveness have not been established in children younger than 12.
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