EMA’s CHMP Issues Positive Opinion for CSL’s HAE Treatment
Global biotechnology leader CSL has reached a significant milestone with the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) issuing a positive opinion on garadacimab.
This recommendation brings garadacimab closer to marketing authorisation as a groundbreaking once-monthly treatment for hereditary angioedema (HAE) in patients aged 12 years and older.
If approved, garadacimab will be the first therapy to inhibit factor XIIa, a key target in preventing HAE attacks.
Hereditary angioedema is a debilitating and potentially life-threatening condition, characterised by painful and unpredictable swelling episodes that can affect areas such as the abdomen, larynx, face, and extremities.
Current therapies address various stages of the biological cascade that causes these attacks, but garadacimab takes a novel approach by intervening at the very start of the process, offering potential for more effective prevention.
The CHMP’s positive opinion is supported by robust clinical data, particularly from the pivotal Phase 3 VANGUARD trial and its ongoing open-label extension study.
The VANGUARD trial met its primary endpoint, demonstrating remarkable efficacy: 62% of patients achieved attack-free status, the median number of monthly attacks was reduced to zero, and the mean number of attacks decreased by 86.5% compared to placebo.
Furthermore, interim analysis of the extension study further underscored garadacimab’s sustained safety and efficacy, with a median exposure period of 13.8 months.
CSL’s Senior Vice President and Head of Global Regulatory Affairs emphasised the company’s patient-focused approach to addressing unmet medical needs. They expressed that this decision brings them closer to offering an innovative treatment for HAE, a condition that has profound impacts on patients’ lives, furthermore, they are excited about the potential of garadacimab to transform HAE care in Europe.
The final decision from the European Commission is anticipated in the first quarter of 2025. Should the marketing authorisation be granted, it will apply across all EU member states, providing HAE patients with a new and convenient treatment option.
In conclusion, CSL’s garadacimab marks a groundbreaking advancement in the treatment of hereditary angioedema, offering hope to patients with a potentially life-threatening condition.
As the biotech leader awaits the final decision, garadacimab stands as a testament to CSL’s commitment to delivering transformative solutions for areas of critical unmet need.
News Credits: CSL receives positive opinion for garadacimab in hereditary angioedema
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