Farabursen for Autosomal Dominant Polycystic Kidney Disease

As of September 2026, MARA’s assessment finds Farabursen’s reimbursement risk concentrated in clinical effectiveness, with care pathway integration a strength; the current MARA Rating and full rationale are available in the report.

The assessment also weighs evidence quality and robustness: how solid the underlying evidence is on its own terms — trial design, size and endpoints — before any question of price is reached. The strength recorded in care pathway integration carries weight because that domain asks how the drug fits into the way care is organised today; a treatment that demands new infrastructure or displaces an established pathway faces extra scrutiny.

Nephrology

This rating sits within MARA’s Nephrology coverage, alongside 15 other independently assessed treatments in the same area.

What payers will ask

Is the clinical benefit the kind payers reward? — Clinical effectiveness

The evidence shows a pharmacodynamic signal and short-term structural kidney signal, but lacks evidence of slowing eGFR decline, prevention of kidney failure, or improvement in symptoms or quality of life. The study was small, with only 10 placebo patients, and lasted only 12 weeks.

Does the economic case hold at the expected price? — Cost effectiveness

No economic model, ICER, or cost-effectiveness data were presented in the evidence.

Is there quality-of-life evidence payers weigh? — Quality of life

No HRQoL data or patient-reported outcomes were reported in the evidence.

Does the safety profile hold up for payers? — Safety and Adverse Effects

Short-term safety data indicate that farabursen was well tolerated with mostly mild injection-site reactions. However, the evidence is limited to short-term exposure, and long-term safety data are not available.

Was the drug compared against what payers expect? — Comparator Selection

The study used placebo as a comparator, which is appropriate for determining drug-specific efficacy but does not address comparative effectiveness against the active standard of care, tolvaptan.

Is the population defined the way payers need it? — Patient Population and Subgroups

The Phase Ib study enrolled a clinically relevant population but had limited demographic diversity, with predominantly White participants. Subgroup analyses were exploratory and based on small samples.

Does the drug fit how care is delivered and paid for? — Care Pathway Integration

The diagnostic process aligns well with specialist ADPKD practice, but the treatment sequencing and monitoring requirements for commercial use are not yet established.

Are the wider system costs understood? — Resource Use and Cost Implications

No budget-impact or resource-use information was presented in the evidence.

Would the evidence survive payer scrutiny? — Evidence Quality and Robustness

The Phase Ib study had strengths such as randomization and a clinically relevant population, but limitations include small sample size, short duration, and reliance on sponsor-supported congress presentations.

How exposed is the case to uncertainty? — Uncertainty, Sensitivity, and Broader Impacts

High uncertainty exists due to the short duration of the study and lack of long-term efficacy data. No sensitivity analysis was presented.

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