'Grand Slam' Pancreatic Cancer Drug Wins FDA Approval

The FDA recently granted approval to a groundbreaking pancreatic cancer drug, daraxonrasib, also known as Rasonque. This first-in-class RAS inhibitor targets multiple isoforms of RAS, a crucial driver mutation in most pancreatic ductal adenocarcinoma (PDAC) cases. Approved for use in adults with previously treated metastatic PDAC or those ineligible for multiagent systemic therapy, the oral drug marked a significant milestone in cancer treatment.

Analysts praised the approval, with FDA Oncology Center of Excellence Director Angelo de Claro highlighting the drug's potential given the high unmet need in pancreatic cancer treatment. The drug received principal support from the RASolute 302 trial, which demonstrated that second-line treatment with Rasonque doubled median overall survival (OS) versus standard chemotherapy in patients with RAS G12 mutations (13.2 vs 6.7 months, P 0.001).

Patients also experienced a more than twofold improvement in progression-free survival (PFS) (7.2 vs 3.6 months, P 0.001).

OS and PFS benefits remained consistent across the overall study population, including those with less common RAS mutations. The drug's approval, announced six and a half months before the user fee deadline, underscored the FDA's commitment to accelerating new cancer treatments for serious conditions.

The most common side effect of daraxonrasib was a skin rash, experienced by 85.5% of patients. Other frequent grade ≥3 adverse events included stomatitis (12%). Treatment discontinuation occurred in a minimal proportion of patients - 1.2% in the daraxonrasib group and 11.2% in the chemotherapy group. The positive clinical outcomes of the RASolute 302 study, described as a "grand slam" by ASM's Chief Medical Officer Julie Gralow, provided proof of principle that targeting the RAS signaling pathway is critical in pancreatic cancer treatment.