In the United States green light for the drug daraxonrasib. In the phase 3 trial, median survival increased from 6.7 to 13.2 months. An important result for one of the most difficult tumors to treat.
The news comes from the United States and concerns pancreatic cancer, one of the most difficult to treat. There The Food and Drug Administration approved daraxonrasib on August 26 (trade name Rasonque), a new oral therapy targeted against RAS (a type of protein that works like a molecular switch), for adults with metastatic pancreatic adenocarcinoma already subjected to at least one previous systemic treatment or not eligible for combined chemotherapy. What convinced the American authority were above all the results of the phase 3 study RASolute 302: la Median overall survival was 13.2 months with daraxonrasib versus 6.7 months with chemotherapy chosen by the doctor. A result which, in a disease with a still extremely severe prognosis, marks an important step in the search for new therapies. The study involved 500 patients. Progression-free survival was 7.2 months with the new drug versus 3.6 months in the control group, while the objective response rate reached 30 percent versus 11 percent. The risk of death during the observed period was 60 percent lower in the daraxonrasib group. Important numbers, but which must be read correctly: median survival it doesn’t mean that every patient lives twice as longbut indicates the point at which half of the patients in the study population are still alive and half are not. It is therefore a population figure, not an individual prediction. The significance of the novelty goes beyond the single drug. RAS is a family of proteins that functions as a molecular switchtransmitting signals to cells that regulate growth and proliferation. When the KRAS gene is altered by a mutation, this switch can remain continuously active, encouraging the uncontrolled growth of tumor cells. In pancreatic cancer, KRAS mutations are extremely frequent and represent one of the main biological drivers of the disease. For decades, however, RAS has been considered one of the most difficult targets to target with a drug. Daraxonrasib belongs to the new generation of therapies that attempt to intervene precisely on this mechanism.
When the tumor has a target
This is, perhaps, the most interesting aspect of the story: it is not simply a new chemotherapy, but one targeted therapy built around tumor biology. In the trial, more than 90 percent of participants had a RAS G12 mutation. The drug is therefore not intended for all patients with pancreatic cancer without distinction, but for a population selected on the basis of the characteristics of the disease. The starting picture explains why the result attracted attention. In the world the pancreatic cancer it causes over 490 thousand deaths every year compared to more than 530 thousand new diagnoses. In Italy, second Cancer numbers in Italy 2025in 2024 were estimated 13,585 new diagnoses. Five-year net survival remains around 11-12 percent, among the lowest recorded for cancers. One of the main problems is late diagnosis: when the disease is identified, it is often no longer possible to intervene surgically, which remains the treatment with curative potential. Daraxonrasib, of course, is also not without side effects. Among those most frequently reported are: rash, diarrhea, stomatitis, nausea, tiredness, vomiting, abdominal pain and decreased appetite. In the trial, grade 3 or higher adverse events were observed in 61.8 percent of patients treated with daraxonrasib and in 69.6 percent of those receiving chemotherapy.
The next match is played in Europe
The American green light does not mean that the drug is already available in Italy. The EMA has started an accelerated review of daraxonrasibbased on data from the phase 3 study. The medicine had already obtained orphan drug designation in the European Union and inclusion in the Cancer Medicines Pathfinder, the path intended for therapies considered of particular interest in oncology. Before reaching Italian patients, the European evaluation and the subsequent national process relating to access and reimbursement must therefore be completed. The regulatory road is still open, but the American result indicates that the strategy to target RAS is no longer just a promise of research. In Italy, meanwhile, the challenge also remains that of early diagnosis and concentration of treatment in specialized centres. The Ministry of Health has defined the standards for a national network of Pancreas Units, with the aim of strengthening the multidisciplinary management of patients. Daraxonrasib is therefore not a cure for pancreatic cancer and does not, on its own, change the history of all patients. But it carries a precise scientific message: a molecular target that for decades seemed nearly impossible to hit may become vulnerable.
