GaradacimabAndembry
Routine prevention of hereditary angioedema (HAE) attacks in adult and adolescent patients aged 12 years and older with HAE Types I and II.
Decision on record
- Meeting Nov 2024 Recommended Hereditary angioedema Types I and II
Access path
- TGA registered · Andembry
TGA label narrower than the PBS population
- Nov 2024Recommended · restricted
vs lanadelumab
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the General Schedule, Authority Required listing of garadacimab for the prophylaxis of recurrent attacks of hereditary angioedema (HAE) Types I and II. The PBAC’s recommendation for listing was based on, among other matters, its assessment that the cost-effectiveness of garadacimab would be acceptable if it were cost-minimised against lanadelumab.PSD · Nov 2024
7.2 The PBAC noted and welcomed the input from individuals, health professionals and organisations via the Consumer Comments facility. The PBAC noted that the comments highlighted the variable individual responses and tolerability to existing treatments, along with the advantages that garadacimab may have given the potential for self-administration and less frequent dosing.PSD · Nov 2024
6.41 The submission presented a CMA to support the listing of garadacimab for the prevention of HAE attacks. The key components and assumptions of the approach are presented in Table 10.PSD · Nov 2024
Table 10: Key components and assumptions of the cost-minimisation approach presented in the submission Component Claim or assumption Therapeutic claim:PSD · Nov 2024
6.37 The submission described garadacimab as non-inferior in terms of effectiveness compared to lanadelumab. The evaluation considered that this claim was uncertain and may not be supported by the evidence.PSD · Nov 2024
• The heterogeneity between the VANGUARD and HELP trials, and overall impact of the transitivity issues on the indirect results, which cannot be reliably estimated.PSD · Nov 2024
6.3 The comments (including comments from individuals living with HAE and their caregivers, and from HAE Australia) described the debilitating physical and quality of life impacts of HAE attacks, due to both the attacks themselves and also due to the fear of attacks which can be unpredictable and life-threatening.PSD · Nov 2024
6.4 The comments described the challenges of treating attacks with ODT (e.g. icatibant and C1-INH) and the varying levels of response to other prophylactic therapies (e.g. lanadelumab, C1-INH and danazol), with many patients reporting adequate control of attacks, while others reported continued frequent hospitalisations.PSD · Nov 2024
Cost-effectiveness
Cost-minimisation analysis performed; no ICER calculated.
Decision context
PopulationAdult and adolescent patients aged 12 years and older with hereditary angioedema Types I and II who experience at least 12 treated acute HAE attacks within a 6-month period.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2024 | Recommended · restricted | lanadelumab | — | RCT · Other |
Consumer voice
Consumers and healthcare professionals reported that HAE causes debilitating physical and quality-of-life impacts with unpredictable, potentially life-threatening attacks. They highlighted challenges with existing treatments and described garadacimab's benefits including self-administration, less frequent dosing, room-temperature storage, and fewer adverse effects, with some patients experiencing
HAE attacks can be painful, and the condition can cause significant physical and emotional distress, and can impact on a patient's ability to work, maintain relationships and engage in everyday activities. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to HAE Types I and II only, and requires ≥12 treated attacks per 6 months; TGA label includes all HAE types without attack frequency requirement.