GivosiranGIVLAARI
Treatment of Acute Hepatic Porphyria (AHP) in adults and adolescents aged 12 years and older with recurrent disease, defined as ≥2 porphyria attacks requiring hospitalisation, presentation at an emergency department or medical intervention via a porphyria clinic within the 6 months prior to treatment initiation.
Decisions on record
- Meeting Nov 2025 Recommended Acute hepatic porphyria in patients aged 12 years and older no PSD
- Meeting Jul 2025 Deferred Acute hepatic porphyria in adults and adolescents aged 12+
Access path
- TGA registered · GIVLAARI
TGA label narrower than the PBS population
- Nov 2025Recommended · restricted
vs best supportive care (BSC)
- PBS listing · Authority Required
From the public summary
7.1 The PBAC deferred making a recommendation for the listing of givosiran for treatment of acute hepatic porphyria (AHP). The PBAC acknowledged the high unmet clinical need for treatments for AHP, which results in chronic symptoms and acute attacks that can include intense and persistent pain, muscle weakness, gastrointestinal, cardiovascular and neurological symptoms.PSD · Jul 2025
OFFICIAL Public Summary Document - July 2025 PBAC Meeting with November 2025 Addendum with Corrigendum for patients with AHP, but that in the context of this rare disease with very substantial impacts on patient quality of life, givosiran would be considered acceptably cost-effective with a price reduction that would result in an acceptable cost per patient per year, in line with other treatments for rare diseases funded on the PBS, accounting for …PSD · Jul 2025
4.11 Diagnosis of AHP in Australia follows the Australian Porphyria Association guidelines, which recommend biochemical testing (urine PBG ± ALA) as the first-line approach, ideally conducted during an acute attack. Elevated levels of PBG and aminolevulinic acid (ALA) are diagnostic markers, though some patients (especially those with AIP) may show elevated levels even outside of attacks.PSD · Jul 2025
2 CADTH 2021. CADTH Reimbursement Review Givosiran (Givlaari) Canadian Journal of Health Technologies Vol 1(11). https://www.cda-amc.ca/sites/default/files/DRR/2021/SR0679-Givlaari%20combined-meta.pdf 8 OFFICIALPSD · Jul 2025
6.29 The submission described givosiran as superior in terms of effectiveness compared with BSC and non-inferior in terms of safety compared to BSC.PSD · Jul 2025
6.30 The Sub-Committees agreed with the Commentary that the therapeutic conclusion regarding effectiveness is adequately supported by the evidence presented in the submission. However, there is some uncertainty regarding the generalisability and/or magnitude of the benefit as observed in the trials compared with the Australian population because: 22 OFFICIALPSD · Jul 2025
6.3 Patients described current treatments for AHP as ineffective, invasive, difficult to access and associated with serious side effects. Individuals who have received givosiran highlighted the substantial reductions in attack severity and hospitalisations and noted the major difference in quality of life, allowing patients to regain a normal life, restoring independence and the ability to participate in work and study.PSD · Jul 2025
Individuals who would like to access treatment also noted the burden of disease in terms of financial implications of disease management, loss of productivity and income, and noted that the cost of givosiran is financially prohibitive. Input from other interested individuals also noted the significant burden on carers and families of people with AHP.PSD · Jul 2025
Cost-effectiveness
ICER value not stated in the publicly available text; appears to be redacted or commercially sensitive.
Decision context
PopulationAdults and adolescents aged 12 years and older with confirmed Acute Hepatic Porphyria and recurrent disease (≥2 porphyria attacks requiring hospitalisation, emergency department presentation, or medical intervention via a porphyria clinic within 6 months prior to treatment initiation).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2025 | Recommended · restricted | best supportive care (BSC) | — | RCT · Annualised number of acute attacks |
Consumer voice
Consumer input from 27 individuals, 5 healthcare professionals, and 1 organisation (Porphyria Association Inc) described AHP as causing multiple continuous symptoms with limited treatment options. Patients receiving givosiran reported substantial reductions in attack severity and hospitalisations with major improvements in quality of life, though highlighted financial barriers to access and diseas
Input described givosiran as well-tolerated and life-changing due to its effectiveness resulting in significantly reduced or eliminated porphyria attacks, reduced hospital admissions and long-term complications such as renal failure, hypertension and hepatoma. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to recurrent disease (≥2 attacks in 6 months); TGA label includes all AHP regardless of attack frequency.