CSL Behring has announced that the National Institute for Health and Care Excellence (NICE) has published its final draft guidance recommending Andembry (garadacimab) for the prevention of recurrent attacks of hereditary angioedema (HAE) in people 12 years and older, if they have two or more attacks a month. This decision means that eligible patients will have access to the first and only prophylactic treatment with once monthly dosing from initiation licensed for routine prevention of recurrent HAE attacks.4
HAE is a rare, chronic, debilitating and potentially life-threatening genetic condition characterised by recurrent and unpredictable attacks of angioedema, which result in painful swelling in different areas of the body, particularly the abdomen, larynx, face and extremities (hands and feet).2 The condition affects approximately one in 50,000 people in the UK3 5 and can have a profound impact on quality of life, with many patients living in constant fear of the next attack.
Garadacimab is the only subcutaneous treatment with once monthly dosing from initiation licenced for routine prevention of recurrent attacks of hereditary angioedema (HAE) in people aged 12 years and older.1,2
This decision is supported by results from the pivotal Vanguard study, where garadacimab significantly reduced the mean number of monthly HAE attacks compared to placebo by 87%, and 62% of patients were consistently attack free over six months.2
HAE is a rare, potentially life-threatening genetic condition characterised by recurrent and unpredictable attacks of swelling,2 severely impacting quality of life, with many patients living in constant fear of the next attack.3
This medicine is subject to additional monitoring. This will allow quick identification of new safety information.
Clinical trials evidence
The NICE recommendation is based on results from the pivotal placebo-controlled Phase 3 VANGUARD trial (N=65) and its open-label extension study (N=161), both evaluating the efficacy and safety of garadacimab. Results from these studies demonstrated that garadacimab significantly reduced the frequency and severity of HAE attacks compared to placebo:8,9
• _87% reduction in mean monthly attacks versus placebo (95% CI –96 to –58; p<0·0001)
• _62% of patients achieving attack-free status throughout the six-month treatment period (p<0·0001)
• _Median number of attacks reduced to zero during the treatment period (IQR 0·00–0·31)
• _90% mean reduction in moderate and severe attacks (95% CI 0.03 to 0.22; p<0·0001).
During the six-month treatment period, 75 adverse events occurred in 25 (64%) of 39 patients in the garadacimab group and 54 adverse events occurred in 15 (60%) of 25 patients in the placebo group. The most common treatment-emergent adverse events in the garadacimab group (n=39) were upper-respiratory tract infections (10%, n=4), nasopharyngitis (8%, n=3), and headaches (8%, n=3). One serious, severe adverse event (laryngeal attack) managed with overnight hospitalisation occurred in the garadacimab group and was assessed by the GBR-AND-0125 Date of Preparation: September 2025.
investigator as unrelated to the investigational product; the patient made a full recovery. Three injection-site reactions, including injection-site erythema, bruising, or pruritus, occurred in two (5%) of 39 patients in the garadacimab
group and three (12%) of 25 patients in the placebo group.2 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).
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