AstraZeneca Plc received two significant regulatory updates from the U.S. Food and Drug Administration, with its cancer therapy Truqap gaining approval for a subset of prostate cancer patients and its kidney disease treatment Ultomiris receiving Priority Review for a new indication. The approvals strengthen the company's portfolio in oncology and rare diseases, addressing critical needs for patients with specific genetic profiles and conditions with limited treatment options.

Truqap Becomes First Targeted Option For PTEN-Deficient Prostate Cancer

The FDA approved Truqap (capivasertib) in combination with abiraterone and prednisone for adults with PTEN-deficient metastatic androgen pathway modulation-naïve or sensitive (mAPMN/S) prostate cancer. This approval makes the regimen the first and only targeted treatment available for this patient population when identified through an FDA-authorized test. The decision addresses a critical need for patients with this specific genetic profile, offering a new therapeutic option where previously none existed.

The FDA decision was supported by findings from the Phase III CAPItello-281 trial. Results showed a 19% reduction in the risk of radiographic disease progression or death compared with abiraterone and androgen deprivation therapy alone. Patients receiving the Truqap combination achieved a median radiographic progression-free survival (rPFS) of 33.2 months, versus 25.7 months in the comparator arm, representing an improvement of 7.5 months. The trial will continue to assess overall survival as a key secondary endpoint.

The safety profile of Truqap in combination with abiraterone and androgen deprivation therapy in CAPItello-281 was broadly consistent with the known profile of each medicine. Grade 3 or higher adverse events occurred in 67% of patients treated with the Truqap combination, with rash (12.3%) and hyperglycemia (10.3%) the most frequently reported. Concurrently with this approval, the FDA also approved a companion diagnostic test to detect PTEN deficiency in tumors of patients with prostate adenocarcinoma.

Ultomiris Receives Priority Review In IgA Nephropathy

Separately, the FDA accepted and granted Priority Review to a supplemental Biologics License Application for Ultomiris (ravulizumab) from AstraZeneca's Alexion rare disease division for the treatment of adults with immunoglobulin A nephropathy (IgAN). Priority Review is reserved for applications that may provide meaningful improvements over existing treatment options. The FDA's decision is expected during the fourth quarter of 2026.

The filing is based on interim Phase 3 I CAN trial data presented at the 2026 European Renal Association Congress. Ultomiris reduced the 24-hour urine protein creatinine ratio by 46.6% from baseline at week 34, compared with 5.6% for placebo, producing a placebo-adjusted treatment effect of 43.4%. Proteinuria reductions appeared as early as week 10 and were maintained through week 34. The treatment was generally well tolerated, with no new safety concerns reported.

AstraZeneca Plc. announced that the US FDA has accepted and granted Priority Review to its supplemental Biologics License Application (sBLA) for Ultomiris (ravulizumab) for adults with immunoglobulin A nephropathy (IgAN). The FDA has set a Prescription Drug User Fee Act (PDUFA) action date during the fourth quarter of 2026. This regulatory milestone follows the Phase 3 I CAN trial, which met a prespecified interim endpoint demonstrating a statistically significant and clinically meaningful reduction in proteinuria compared with placebo at week 34.

Ultomiris Reduces Proteinuria

The interim analysis revealed that patients treated with Ultomiris achieved a 46.6% reduction in 24-hour urine protein creatinine ratio (UPCR) from baseline. In contrast, the placebo group recorded a 5.6% reduction. The placebo-adjusted treatment effect was 43.4%, reaching statistical significance (p<0.0001).

Early and Sustained Response

The reduction in proteinuria emerged as early as week 10 and remained consistent through week 34. At week 10, Ultomiris-treated patients showed a 36.7% reduction in proteinuria, compared with 8.5% for those on placebo. The benefit was observed across multiple patient subgroups, including individuals with varying demographic characteristics and disease severity.

Jonathan Barratt, Mayer Professor of Renal Medicine at the University of Leicester and an investigator in the I CAN trial, noted that terminal complement activation plays a significant role in inflammation and kidney function decline in IgAN. He stated that the interim findings suggest targeting this pathway with Ultomiris may provide a disease-modifying approach.

Safety Profile and Regulatory Impact

Gianluca Pirozzi, Senior Vice President and Head of Development, Regulatory and Safety at Alexion, AstraZeneca Rare Disease, emphasized the early and meaningful reduction in proteinuria, particularly among patients at higher risk of disease progression. He added that the findings support the role of terminal complement inhibition in IgAN and will help advance regulatory filings in key markets. The safety profile was consistent with the known profile of Ultomiris, with no new safety concerns identified.