CSL Receives Positive CHMP Opinion for Garadacimab in Hereditary Angioedema (HAE)
Rhea-AI Summary
CSL (ASX:CSL; USOTC:CSLLY) received a positive CHMP opinion recommending marketing authorization for garadacimab, a first-of-its-kind once-monthly treatment for hereditary angioedema (HAE) prevention in patients aged 12 and older. The treatment, which targets factor XIIa to prevent HAE attacks, demonstrated impressive results in the Phase 3 VANGUARD trial, with 62% of patients achieving attack-free status and an 86.5% reduction in mean monthly HAE attacks compared to placebo.
The European Commission's final decision is expected in Q1 2025. The recommendation is supported by favorable long-term safety data from an ongoing open-label extension study, with median exposure of 13.8 months. If approved, garadacimab would be available across all EU member states.
Positive
- First-in-class once-monthly treatment for HAE prevention
- 62% of patients achieved attack-free status in Phase 3 trial
- 86.5% reduction in mean monthly HAE attacks vs placebo
- Favorable long-term safety profile demonstrated in extension study
Negative
- None.
If approved, garadacimab will be the first and only once-monthly treatment inhibiting factor XIIa to prevent attacks in HAE patients – a community CSL has been serving for more than 40 years
MARBURG,
"CSL has a longstanding and relentless patient-focused approach to developing transformational medicines in areas of unmet need. This CHMP decision brings us closer to offering an innovative treatment to patients living with HAE, which is a debilitating and potentially life-threatening condition," said Emmanuelle Lecomte-Brisset, Senior Vice President and Head of Global Regulatory Affairs, CSL. "We look forward to making this therapy available to patients in
Attacks of HAE are often painful and can spread to multiple sites of the body, including the abdomen, larynx, face, and extremities. Current HAE preventive therapies work at various downstream steps in the cascade, but none prevent the cascade at its start.
The CHMP positive opinion is based on data from the pivotal placebo-controlled Phase 3 VANGUARD trial and its open-label extension study, both evaluating the efficacy and safety of garadacimab. The pivotal study met its primary endpoint and showed that garadacimab led to 62 percent of patients achieving attack-free status throughout the treatment period, reduced the median number of HAE attacks to zero and reduced the mean number of HAE attacks per month by 86.5 percent compared to placebo. Interim analysis of the ongoing open-label extension study (median garadacimab exposure 13.8 months) showed that garadacimab has a favorable long-term safety profile and provides sustained HAE attack reduction. The full results from VANGUARD were published in The Lancet (April 2023) and the primary results of the ongoing open-label extension study were published in Allergy (October 2024).
If granted, the centralized marketing authorization of garadacimab would be valid in all EU member states. The final opinion was published in the CHMP meeting highlights on the EMA website.
About 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-INH, a protein in the blood that helps to control inflammation. Inadequate amounts of properly functioning C1-INH 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 and can spread to multiple sites, 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 CSL
CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology 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 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 32,000 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 CSLBehring.com/Vita and follow us on Twitter.com/CSL.
For more information about CSL, visit www.CSL.com.
Media Contact
Valerie Bomberger, CSL
Office: +1 610-291-5388
Mobile: +1 267-280-3829
Email: valerie.bomberger@cslbehring.com
Stephanie Fuchs, CSL Behring
Mobile: +49 151 58438860
Email: Stephanie.Fuchs@cslbehring.com
In Australia:
Jimmy Baker, CSL
Mobile: +61 450 909 211
Email: Jimmy.Baker@csl.com.au
Chris Cooper, CSL
Mobile: +61 455 022 740
Email: Chris.cooper@csl.com.au
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SOURCE CSL Behring
FAQ
When will the European Commission make a final decision on CSLLY's garadacimab for HAE?
What are the key efficacy results from CSLLY's Phase 3 VANGUARD trial for garadacimab?
How is CSLLY's garadacimab different from other HAE treatments?
What age groups will CSLLY's garadacimab treat if approved in Europe?
How long was the safety profile of CSLLY's garadacimab studied in the extension trial?
