CSL Reports Positive Top-Line Phase 3b Results Supporting Planned Expanded Pediatric Filing for ANDEMBRY® (garadacimab-gxii) in Children with Hereditary Angioedema (HAE)
Rhea-AI Summary
CSL (USOTC:CSLLY) reported positive top-line results from a multicenter, open-label Phase 3b study of ANDEMBRY (garadacimab-gxii) in children aged 2–11 years with hereditary angioedema (HAE). According to CSL, ANDEMBRY showed a favorable safety and tolerability profile consistent with earlier studies, and most participants remained attack-free over 12 months.
Children 6–11 years (n=16) received 100 mg once monthly, while those 2–5 years (n=6) received 100 mg every two months. ANDEMBRY is already approved in over 40 countries to prevent HAE attacks in patients 12 years and older. CSL plans to begin regulatory filings in the first half of its fiscal year to seek an expanded pediatric indication for ages 2–11, with full data to be presented at an upcoming scientific congress and submitted for publication.
Positive
- Majority pediatric patients attack-free over 12 months in Phase 3b HAE study
- Favorable safety and tolerability in children 2–11 years, consistent with prior ANDEMBRY studies
- Planned regulatory filings for expanded pediatric indication ages 2–11 in first half of fiscal year
- Existing approval in 40+ countries for prevention of HAE attacks in patients 12 years and older
Negative
- ANDEMBRY currently not established as safe or effective under 12, pending regulatory review
- Reported common adverse events include injection-site reactions, abdominal pain, and nasopharyngitis
- Only top-line Phase 3b data disclosed; full pediatric results still pending presentation and publication
AI-generated analysis. How Rhea-AI works. Not financial advice.
- CSL plans to begin submitting regulatory filings for expanded pediatric indication for ANDEMBRY in the first half of the company's fiscal year
- Full data to be presented at upcoming scientific congress
In this multicenter, open-label Phase 3b study evaluating ANDEMBRY in people living with HAE aged 2 to 11 years, ANDEMBRY demonstrated a favorable safety and tolerability profile consistent with previous studies. Treatment response was observed across the study population, with the majority of participants remaining attack-free during the 12-month treatment period. Participants aged 6 to 11 years (n = 16) received 100 mg of ANDEMBRY once monthly, while participants aged 2 to 5 years (n = 6) received 100 mg of ANDEMBRY every two months.
ANDEMBRY is a monoclonal antibody that targets factor XIIa, a plasma protein that plays a key role in attacks of swelling in people with HAE. By acting at the top of the HAE cascade, ANDEMBRY prevents attacks and represents a novel long-term prophylactic strategy.
"The top-line results support our plans to seek an expanded pediatric indication for ANDEMBRY in children aged 2 to 11 years," said Dr. Bill Mezzanotte, Executive Vice President, Head of R&D, CSL. "We are excited to share the full study findings with the scientific and patient communities at an upcoming medical congress and remain committed to advancing treatment options for people living with hereditary angioedema."
CSL plans to begin submitting filings to health authorities in the first half of the company's fiscal year to support an expanded pediatric indication for ANDEMBRY in children aged 2 to 11 years. Full study results will be presented at an upcoming scientific congress and submitted for publication in a peer-reviewed journal.
About Hereditary Angioedema (HAE)
HAE is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE is caused by deficient or dysfunctional C1 esterase inhibitor (C1INH), a protein in the blood that helps to control inflammation. Inadequate amounts of properly functioning C1INH can lead to the accumulation of fluid in body tissues, causing considerable swelling referred to as angioedema. HAE attacks can affect many parts of the body, including the face, abdomen, larynx, and extremities. Patients who have abdominal attacks of HAE can experience extreme pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face or throat can result in airway closure, asphyxiation and, if left untreated, death.
About ANDEMBRY
ANDEMBRY is a novel monoclonal antibody inhibiting factor XIIa (anti-FXIIa mAb) that has completed the Phase 3 pivotal study as a once-monthly subcutaneous prophylactic treatment for attacks related to HAE, a form of bradykinin-mediated angioedema. It was discovered and optimized by scientists at CSL's Bio21-based research site, with formulation and manufacturing for the clinical programs completed at the CSL Broadmeadows Biotech Manufacturing Facility. ANDEMBRY uniquely inhibits the plasma protein, FXIIa. FXII is the first protein activated in the HAE pathway, initiating the inflammatory bradykinin-producing kallikrein-kinin system. By targeting activated FXII (FXIIa), ANDEMBRY inhibits this cascade at the top as compared to other HAE therapies that target downstream mediators.
IMPORTANT SAFETY INFORMATION
What are the possible side effects of ANDEMBRY?
The most common side effects of ANDEMBRY include:
- Redness, itchiness, and bruising (injection-site reactions)
- Stomach (abdominal) pain
- Runny or stuffy nose, sneezing, watery eyes (nasopharyngitis)
What is ANDEMBRY?
ANDEMBRY® (garadacimab-gxii) injection, for subcutaneous use, is a prescription medication used to prevent attacks of hereditary angioedema (HAE) in people 12 years and older.
It is not known if ANDEMBRY is safe and effective in children under 12 years of age.
What should I tell my healthcare provider before using ANDEMBRY?
Before using ANDEMBRY, tell your healthcare provider about any medical condition you may have, especially if you are pregnant, planning to become pregnant, breastfeeding, or planning to breastfeed. It is not known if ANDEMBRY can harm your unborn baby or if ANDEMBRY passes into breastmilk. Talk to your healthcare provider about the best way to feed your baby while using ANDEMBRY.
Tell your healthcare provider about all medications you take, including prescription medicines, over-the-counter treatments, vitamins, and herbal supplements.
How should I use ANDEMBRY?
Use ANDEMBRY exactly as instructed by your healthcare provider. Detailed instructions for use can be found in the patient information section of the full prescribing information.
ANDEMBRY is given as an injection under your skin (subcutaneous) by you or a caregiver. Your healthcare provider should show you or your caregiver how to prepare and inject your dose of ANDEMBRY before you inject yourself for the first time. Do not try to inject ANDEMBRY unless you have been trained by your healthcare provider.
Please see full prescribing information for ANDEMBRY,
including patient information and instructions for use.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
You can also report side effects to CSL Behring's Pharmacovigilance Department at 1-866-915-6958.
About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a leading global biopharma company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency, dialysis and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses, CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 29,000+ people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSL.com/Vita.
For more information about CSL, visit CSL.com.
Media Contacts
Etanjalie Ayala
Mobile: +1 610 297 1069
Email: etanjalie.ayala@cslbehring.com
Bernadette Murdoch
Mobile: +61 449 950 745
Email: bernadette.murdoch@csl.com.au
Investors Contact
Bernard Ronchi
Mobile: +61 3 9389 3470
Email: bernard.ronchi@csl.com.au
ANDEMBRY is manufactured by CSL Behring GmbH and distributed by CSL Behring LLC. ANDEMBRY® is a registered trademark of CSL Behring GmbH.
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SOURCE CSL