ProKidney Announces FDA Alignment on Accelerated Approval Pathway for Rilparencel

Analysis reveals significant industry trends and economic implications

Release Date

2025-07-16

Category

Drug Approval Event

Reference

Source

Breakthrough Clinical Results

ProKidney announced FDA confirmation that the accelerated approval pathway for rilparencel, an autologous cellular therapy for chronic kidney disease (CKD) and type 2 diabetes, is acceptable. The FDA confirmed that eGFR slope can serve as a surrogate endpoint for a Biologics License Application (BLA). The ongoing Phase 3 PROACT 1 study will be used for both accelerated and full approval. Topline data for accelerated approval is anticipated in Q2 2027, with nearly half of the required patients already enrolled. The FDA also confirmed that the PROACT 1 study can serve as the confirmatory study for full approval.

Key Highlights

  • FDA confirmed alignment on accelerated approval pathway for rilparencel.
  • eGFR slope accepted as surrogate endpoint for accelerated approval.
  • Phase 3 PROACT 1 study will support both accelerated and full approval.
  • Topline data for accelerated approval anticipated in Q2 2027.

Economic Burden

Economic Burden of Treating Chronic Kidney Disease in USA and Europe

The economic burden of CKD is substantial and growing, with projections indicating a 9.3% increase from $372.0 billion to $406.7 billion between 2022 and 2027 across 31 countries/regions according to the 2024 Inside CKD study.

United States

In the North America and Caribbean region, patients have better access to kidney replacement therapy and CKD-related services than in much of the world, with a 2-fold higher than global median prevalence of dialysis and transplantation. The region also has a 3-fold higher than global median prevalence of dialysis centers compared to global figures.

For the US specifically, the total economic burden of RRT for the year 2003 was estimated at 304.773 million euros. For individual patients with type 1 diabetes in the US, early treatment with captopril showed an incremental cost of USD 27,143 per QALY. When the age at onset of diabetes is 20 years and glycosylated hemoglobin (HbA1c) level is 9%, the cost-effectiveness ratio is USD 13,814 per QALY. When the age at onset is 25 years and HbA1c level is 7%, the cost-effectiveness ratio is USD 39,530 per QALY.

In 2019, the annual excess costs associated with ESA (erythropoiesis-stimulating agents) administration for patients with anemia and non-dialysis-dependent (NDD) CKD were estimated at $2.5 billion in the United States. Of this amount, 94.4% ($2.4 billion) came from in-clinic ESA administration, with Medicare-insured patients accounting for 79.4% ($2.0 billion) of total annual excess costs.

Europe

In Greece (2005 data), the total health-sector cost for hemodialysis exceeds 171 million Euros, or 182 Euros per session and 229 Euros per inpatient day. Providing hemodialysis care for 0.05 percent of the population suffering from ESRD absorbs approximately 2 percent of total health expenditure in Greece.

The productivity costs in Greece are also significant, with 2,046 years lost due to mortality, with potential productivity cost estimated at 9.9 million Euros according to the human capital approach. Total morbidity cost due to absence from work and early retirement was estimated at more than 273 million Euros according to the human capital approach.

In Portugal (1993 data), it was noted that dialysis and transplantation should be in balance, with a particular stress on transplantation because it is much less expensive.

Global Trends

Annual KRT-associated costs are projected to increase by 10.0% from $169.6 billion to $186.6 billion between 2022 and 2027. By 2027, patients receiving kidney replacement therapy (KRT) are projected to constitute only 5.3% of the diagnosed CKD population but contribute a disproportionate 45.9% of the total costs.

Earlier diagnosis and proactive management could slow disease progression, potentially alleviating the substantial costs associated with later CKD stages. The data presented can be used to inform healthcare resource allocation and shape future policy.

Drug used in other indications

Rilparencel Clinical Trials Beyond Chronic Kidney Disease

There is currently no available information in the provided sources regarding clinical trials of rilparencel (GFH018) for indications other than Chronic kidney disease. The data needed to identify additional clinical indications and their corresponding intervention models is not present in the reference materials.

Without access to complete clinical trial data, it's not possible to detail the additional indications, intervention protocols, dosing regimens, or trial methodologies that might exist for this pharmaceutical compound beyond its application in renal disease.

For comprehensive information about rilparencel's current clinical development program, consulting official clinical trial registries such as ClinicalTrials.gov, pharmaceutical company pipelines, or recent medical literature would be necessary.