The Food and Drug Administration (FDA), the U.S. regulatory agency, granted approval this Wednesday, the 26th, to an innovative drug intended for the treatment of pancreatic cancer, a condition classified as one of the most lethal and difficult to treat among all types of neoplasms.
This new treatment has the capacity to slow the progression of the disease and double patients' life expectancy. The drug in question is a tablet called daraxonrasib, marketed under the name Rasonque, and it should be taken daily. Revolution Medicines, the company responsible for manufacturing, reported that the monthly cost of this treatment will be approximately US$ 39,800.
The FDA's endorsement occurred less than three months after the efficacy data of the drug was published in The New England Journal of Medicine. The study included 500 individuals diagnosed with pancreatic ductal adenocarcinoma, which represents the most common type of pancreatic cancer, accounting for over 90% of cases.
The results showed that patients who received daraxonrasib achieved a median survival of 13.2 months, nearly double that of those who underwent only chemotherapy. Furthermore, an improvement in quality of life and a decrease in pain perception were observed.
When these findings were presented during a meeting of the American Society of Clinical Oncology (ASCO) before thousands of healthcare professionals, the news was met with a standing ovation, and some oncologists became so emotional they cried.
Pancreatic cancer is considered extremely fatal for two main reasons. Firstly, it tends to be asymptomatic; when symptoms appear, such as jaundice (yellowing of the skin) or abdominal pain, the disease has often already spread to other organs.
Secondly, more than 90% of diagnoses of this cancer are associated with a genetic alteration in the KRAS gene, which codes for a protein with the same name. Under normal conditions, KRAS proteins stimulate cell division. However, a mutation in this gene can trigger a serious scenario: uncontrolled cell proliferation and, consequently, tumor formation.
For several decades, this mutated protein was considered unattainable by the pharmaceutical industry. This was because its surface was very smooth, lacking a suitable indentation for a molecule to fit into and inhibit its function. Until then, all approaches to create a direct inhibitor failed.
The difference of daraxonrasib lies precisely in this. Instead of seeking a direct binding to KRAS, it first interacts with a molecule called cyclophilin A, which is involved in the protein synthesis process. It is the combined interaction of the drug with this molecule that manages to neutralize KRAS activity.
Although the drug presented adverse effects, such as skin rashes, diarrhea, and vomiting, these were reported as less frequent and milder compared to the side effects observed in chemotherapy.