On August 26, the U.S. Food and Drug Administration (FDA) approved daraxonrasib for the treatment of pancreatic ductal adenocarcinoma (PDAC), the most common type of pancreatic cancer in adults. The approval covers patients whose cancer has metastasized to other parts of the body and for whom other treatments have failed. The drug is marketed by Revolution Medicines under the brand name Rasonque.
Daraxonrasib is the first targeted therapy approved specifically for pancreatic ductal adenocarcinoma. It inhibits a protein called RAS, which acts like a molecular switch that can become stuck in the "on" position, driving cancer cell growth. Daraxonrasib disables this molecular switch, thereby inhibiting cancer cell growth. More than 90% of pancreatic ductal adenocarcinoma patients carry gene mutations that keep the RAS protein in the "on" state, leading physicians to expect the drug to benefit many patients.
The FDA based its approval on clinical trial results presented at the American Society of Clinical Oncology (ASCO) annual meeting. The results showed that daraxonrasib provided a significant survival benefit compared with chemotherapy. In this pivotal study, the median overall survival was 13.2 months for patients receiving daraxonrasib, compared with 6.7 months for those receiving chemotherapy. When the results were announced, more than 9,000 attendees rose to their feet in applause, and the data were simultaneously published in the New England Journal of Medicine.
In terms of safety, 11.2% of patients in the chemotherapy group had to discontinue treatment due to severe side effects, compared with only 1.2% in the daraxonrasib group, suggesting the drug may impose a smaller physical burden than conventional chemotherapy. Rasonque has been filed for marketing approval in China. It is priced at $39,800 per month, with an annual treatment cost of approximately $477,600 (about 3.45 million RMB). More promisingly, RAS mutations are also found in many other cancers, and the drug is expected to benefit patients with those cancers as well, with multiple related clinical trials already underway.

Source: 36Kr / Forbes | https://36kr.com/p/3964626720497153