CSL receives positive results from rare disease drug phase 3 trial
pharmafile | February 28, 2023 | News story | Research and Development |
Biotech company CSL has reported positive results from its phase 3 trial for garadacimab, a monthly treatment for hereditary angioedema (HAE). The company is now preparing for regulatory filings and hopes to take some of the market share from Takeda’s Takhzyro.
The VANGUARD study of Factor Xlla inhibitor garadacimab concluded last summer, with topline results being reported shortly after. However, full data from the study was reported at the American Academy of Allergy, Asthma and Immunology (AAAAI) congress. This data suggests that it has at least equal efficacy as Takeda’s drug, as well as being more convenient to administer.
HAE is a rare genetic disorder affecting between 1 in 10,000 to 1 in 50,000 people globally. The disease can cause life-threatening attacks of painful swelling in various parts of the body lasting several days and sometimes leading to asphyxiation if affecting the airways.
Throughout the trial, patients treated with garadacimab experienced a reduced average number of attacks by 86.5% compared to the placebo group. 61.5% of patients on the drug were attack-free throughout the six-month study period.
Dr Timothy Craig, tenured professor of medicine, paediatrics and biomedical sciences at Penn State University and principal investigator of the study, commented: “Targeting FXlla and the HAE cascade from the start, as opposed to intervening downstream, is an innovative treatment approach that could help stop the process in its tracks. The phase 3 data we are presenting support the potential use of garadacimab as a prophylactic therapy for HAE.”
Catherine Milch, vice president R&D Immunology at CSL, added: “The data being shared at AAAAI showcase the efficacy and safety profile of garadacimab administered as a convenient monthly subcutaneous injection. The clinical trial results support garadacimab as a novel, first-in-class potential treatment that could offer a significant benefit to patients with HAE. Garadacimab represents the next chapter in delivering on our promise to bring disruptive innovation and treatment options to patients living with rare diseases.”
Betsy Goodfellow
