SotaterceptWinrevair
Add-on therapy for adult patients with Group 1 pulmonary arterial hypertension (PAH) WHO Functional Class II (with elevated NT-proBNP) or III who have been on stable PAH therapy for at least 90 days.
Decision on record
- Meeting Mar 2025 Not recommended Group 1 pulmonary arterial hypertension (PAH), add-on therapy
Access path
- TGA registered · Winrevair
TGA label narrower than the PBS population
- Mar 2025Not recommended
vs placebo (primary); selexipag (secondary)
From the public summary
The PBAC did not recommend the Section 100 (Highly Specialised Drugs Program) listing of sotatercept as add-on therapy for the treatment of patients with Group 1 pulmonary arterial hypertension (PAH). Listing was requested on the basis of a (i) cost- utility analysis versus placebo and (ii) price parity versus selexipag for a proportion of the patients.PSD · Mar 2025
The PBAC considered the primary reason for this outcome was due to the economic analysis provided in the submission. The PBAC also considered that the proposed PBS population was unclear in terms of the choice of comparator and the applicability of the clinical trial data.PSD · Mar 2025
The submission proposed a weighted effective price for sotatercept. The submission assumed that % of sotatercept’s PBS utilisation would be as add-on therapy to mono, dual or triple PAH therapy, with the comparator being placebo. Given the clinical claim, the submission presented a cost-utility analysis (CUA) of sotatercept plus background PAH therapy (BGT) versus BGT alone for this proportion of the population.PSD · Mar 2025
The remaining 30% of use was assumed to replace selexipag (as the third PAH therapy in triple therapy with ERA and PDE5i), for which the submission requested a price for 41PSD · Mar 2025
placebo Secondary comparator: sotatercept is superior in terms of efficacy and has different but manageable safety to selexipag Source: Table 1.1-1, p16 of the submission.PSD · Mar 2025
BNP=B-type natriuretic peptide; FC=functional class; IV PCA=intravenous prostacyclin; kg=kilogram; MCI=multicomponent improvement; mg=milligram; NT-proBNP=N-terminal pro-type natriuretic peptide; PAH=pulmonary arterial hypertension; PVR=pulmonary vascular resistance; SC=subcutaneous; TTCW=time to clinical worsening; WHO=World Health Organisation; 6MWD=6-minute walk distance. a WHO FC II patients with NT-proBNP ≥650 ng/L (BNP ≥200 ng/L).PSD · Mar 2025
Lung Foundation Australia wrote in support of sotatercept being listed on the PBS for PAH, noting the negative physical and mental impacts of PAH, as well as the inability of patients to work or study. The organisation also noted the impact of patients financing medications for their lung condition. The letter referred to personal stories emphasising the challenges faced by PAH patients.PSD · Mar 2025
The Australian Scleroderma Interest Group (ASIG) described PAH as a devastating complication of scleroderma that leads to shortness of breath, fatigue, and reduced exercise capacity. ASIG stated that patients with scleroderma-PAH have a worse prognosis than those with idiopathic PAH, with a high mortality rate and significant impacts on quality of life. ASIG noted the unmet need for effective treatments in scleroderma-PAH.PSD · Mar 2025
Cost-effectiveness
ICER not stated in the public summary document. The document indicates an economic evaluation was performed but the specific ICER value is not disclosed.
Decision context
PopulationAdult patients with PAH WHO Functional Class II (with NT-proBNP ≥650 ng/L or BNP ≥200 ng/L) or WHO Functional Class III who have been on stable PAH therapy for at least 90 days, as add-on to dual, triple, or quadruple combination therapy.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2025 | Not recommended | placebo (primary); selexipag (secondary) | — | RCT · Surrogate |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| ZENITH | Ph 3 | 173 | Time to First Confirmed Morbidity or Mortality Event | completed |
Consumer voice
Consumer input from 154 individuals, 4 healthcare professionals, and 2 organisations supported sotatercept listing for PAH, emphasizing the disease's severe physical, mental and financial impacts, unmet treatment needs, and potential benefits of the new therapy.
The Australian Scleroderma Interest Group (ASIG) described PAH as a devastating complication of scleroderma that leads to shortness of breath, fatigue, and reduced exercise capacity. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to add-on therapy after ≥90 days stable prior therapy with NT-proBNP/BNP thresholds; TGA label permits monotherapy and lacks biomarker/duration criteria.