LanadelumabTakhzyro
Preventative treatment of hereditary angioedema (HAE) Types 1 or 2 in patients aged 2 to 11 years who have experienced at least one treated acute attack within a three-month period.
Decisions on record
- Meeting Nov 2024 Recommended Hereditary angioedema Types 1 or 2 in patients aged 2-11 years
- Meeting Jul 2021 Recommended Hereditary angioedema
- Meeting Jul 2020 Deferred Hereditary angioedema (HAE)
- Meeting Jul 2019 Not recommended Hereditary angioedema (HAE)
Access path
- TGA registered · Takhzyro
TGA label narrower than the PBS population
- Jul 2019Recommended · restricted
vs intravenous C1-esterase inhibitor (Berinert)
- Jul 2020Deferred
Comparator changed: intravenous C1-esterase inhibitor (Berinert) → Standard of care…
- Jul 2021Recommended
Comparator changed: Standard of care (on-demand treatment with oral routine prophylaxis…
- Nov 2024Recommended
Comparator changed: standard of care (SOC) comprising on-demand treatment with icatibant…
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the listing of lanadelumab for the preventative treatment of hereditary angioedema (HAE) Types 1 or 2 in patients aged less than 12 years. The PBAC was satisfied that lanadelumab provides, for some patients, a substantial clinical benefit in terms of a reduction in HAE attack frequency versus the comparator, ODT.PSD · Nov 2024
The PBAC noted that an economic model for the <12 yrs population was not provided, but considered that the model used to support listing in the ≥12 yrs population could be used to support listing in the expanded population.PSD · Nov 2024
6.29 The submission did not present an economic evaluation, stating that “there is insufficient data to populate an informative economic evaluation for lanadelumab in the proposed paediatric population.” The submission acknowledged the limited data on the baseline frequency of HAE attacks in the requested population.PSD · Nov 2024
6.30 The submission requested listing of the 150 mg injection at the same price per vial as the currently-listed 300 mg strength for the ≥12 year population. However, the evaluation considered that it was unclear whether:PSD · Nov 2024
Economic Cost-effectiveness of lanadelumab in this population cannot be quantified Source: Table 1, p1 of the submission.PSD · Nov 2024
6.26 The submission described lanadelumab as superior in terms of effectiveness compared with ODT with icatibant or C1-INH. Taking the evidence as a whole, the evaluation considered that it was reasonable to conclude that lanadelumab reduces the frequency of attacks in children below the age of 12 years, although the effect size is highly uncertain.PSD · Nov 2024
6.2 The PBAC noted and welcomed the input from individuals (2), and health care professionals (2). The comments described a range of benefits of treatment with lanadelumab including improved quality of life, avoidance of acute hospital presentations, and lower frequency of needle insertions (compared to other long term prophylactic options).PSD · Nov 2024
6.3 A health professional noted that treatment of most paediatric patients is usually based on ODT, with relatively low use of long-term prophylactic (LTP) therapies prior to puberty. They noted that some paediatric patients with a more severe phenotype do require LTP based on attack frequency and severity (e.g.PSD · Nov 2024
Cost-effectiveness
No economic evaluation presented in submission; cost-effectiveness cannot be quantified.
The submission did not present an economic evaluation. PSD · 2024
Decision context
PopulationPatients diagnosed with HAE Types 1 or 2 aged 2 to 11 years who experienced at least one treated acute attack within a three-month period prior to commencing treatment.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2024 | Recommended | on-demand treatment (icatibant or C1 esterase inhibitor) | — | RCT · HAE attack rate |
| Jul 2021 | Recommended | standard of care (SOC) comprising on-demand treatment with icatibant and/or intravenous C1-esterase inhibitor | — | RCT · HAE attack rate |
| Jul 2020 | Deferred | Standard of care (on-demand treatment with oral routine prophylaxis, or intravenous C1 esterase inhibitor) | — | RCT · HAE attack rate |
| Jul 2019 | Recommended · restricted | intravenous C1-esterase inhibitor (Berinert) | — | RCT · HAE attack rate |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| SPRING | Ph 3 | 21 | Number of Participants With Adverse Events Including Serious Adverse Events (SAEs) and Adv… | completed |
Consumer voice
Four individuals and health professionals provided input describing benefits of lanadelumab including improved quality of life, reduced hospital visits, and fewer injections compared to alternatives. Concerns were raised about treatment burden in paediatric patients and practical barriers such as refrigeration requirements for travel.
improved quality of life, avoidance of acute hospital presentations, and lower frequency of needle insertions (compared to other long term prophylactic options) Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to ages 2–11 years with ≥1 treated attack in 3 months; TGA label covers all ages ≥2 years without attack frequency requirement.