Haemonetics Stock Impacted by CSL's FDA Approval of ANDEMBRY for Hereditary Angioedema
- CSL Limited received FDA approval for ANDEMBRY®, a treatment aimed at preventing hereditary angioedema attacks.
- ANDEMBRY demonstrated over 99% reduction in HAE attack frequency during Phase 3 trials, significantly improving patient outcomes.
- The launch of ANDEMBRY enhances CSL's commitment to innovative treatments for rare diseases and bolsters its HAE franchise.
CSL Launches Revolutionary Treatment for Hereditary Angioedema
CSL Limited has recently achieved a significant milestone in the treatment of hereditary angioedema (HAE) with the FDA's approval of ANDEMBRY® (garadacimab-gxii). This groundbreaking therapy is the first designed specifically to target factor XIIa, aiming to prevent HAE attacks in patients aged 12 and older. ANDEMBRY is administered as a once-monthly subcutaneous injection that takes 15 seconds or less, offering a convenient and user-friendly option for patients. This innovative approach is particularly noteworthy as it acts at the top of the HAE cascade, providing sustained protection against attacks, which are often painful and debilitating.
The approval is substantiated by the results from the Phase 3 VANGUARD trial, where ANDEMBRY demonstrated a remarkable efficacy in reducing the frequency of HAE attacks. Participants experienced a median reduction in attacks of over 99% and a least squares mean reduction of 89.2% compared to those receiving a placebo. Additionally, 62% of patients treated with ANDEMBRY remained completely attack-free during the treatment period. The drug also significantly diminished the need for on-demand therapy, with more than 99% median and 88% mean reductions in attacks requiring such interventions, alongside a 90% mean reduction in moderate to severe attacks. These outcomes highlight ANDEMBRY's potential to transform HAE management and improve patient quality of life.
Dr. Bill Mezzanotte, Executive Vice President and Head of R&D at CSL, underscores the company’s long-standing commitment to advancing treatments for HAE. With over 40 years of innovation in this specialty, the introduction of ANDEMBRY marks a pivotal point for CSL, enhancing its HAE franchise and offering a new lifeline to patients grappling with this rare genetic disorder, which affects approximately 1 in 50,000 individuals. The development of this therapy not only showcases CSL's scientific advancements but also reinforces its dedication to addressing unmet medical needs in the field.
In addition to the drug's groundbreaking efficacy, the trial reported that the most common side effects included nasopharyngitis, affecting 7% or more of participants. As ANDEMBRY enters the market, CSL aims to provide patients with a transformative option, enhancing their treatment experience and fostering hope within the HAE community. The approval of ANDEMBRY reflects a significant step forward in the management of hereditary angioedema, positioning CSL as a leader in the biotechnology sector focused on rare diseases.