Europe gives the green light to the drug that increases life expectancy in pancreatic cancer. The European Medicines Agency (EMA) recommends marketing authorization for daraxonrasib (Rasonque), a daily pill to slow down metastatic pancreatic adenocarcinoma. In the U.S., it was approved last August through the fast track due to proven benefits.
Patients treated with daraxonrasib experienced a median survival of 12.4 months, compared to 6.7 months for those who received chemotherapy. Additionally, the time during which patients lived without their disease worsening was 7.2 months with the pill, compared to 3.6 months with chemo. The most frequently reported side effects were diarrhea, rash, stomatitis (inflammation of the mouth mucosa), nausea, and vomiting.
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This drug is aimed at patients with metastatic pancreatic adenocarcinoma whose disease has worsened after previous treatment, have limited therapeutic options, and have an unfavorable prognosis. Given the severity of the disease, the limited efficacy, and considerable toxicity of standard chemotherapy, there is a need for better treatment options for these patients.
In Europe, an estimated 79,000 to 146,000 new cases of pancreatic cancer are diagnosed annually, of which approximately 50% to 52% are diagnosed at a metastatic stage (at the time of initial detection or due to advanced spread). In our country, there are nearly 10,000 detections per year. Less than 20% of patients are eligible for surgery at the time of diagnosis, given the advanced stage of the disease. After surgery, survival is usually between 10 and 20 months.
The secret of the drug against the key gene of pancreatic cancer
Daraxonrasib was evaluated in a multinational, phase 3, open-label study involving 500 patients with metastatic pancreatic adenocarcinoma whose disease had worsened after previous systemic treatment. Of these patients, 92 had a confirmed RAS G12 mutation, including the most common RAS mutations associated with this disease.
Our country participated through four hospitals (Clínica Universidad de Navarra, Hospital 12 de Octubre in Madrid, Hospital Vall d'Hebron in Barcelona, and Clínico in Valencia), allowing around twenty Spanish patients to test the benefits of this treatment.
The RAS pathway mutation means that the therapy is aimed at cutting off any communication of the tumor cell with the environment that supplies it with nutrients. "Unlike first-generation inhibitors that only targeted very specific mutations (such as the KRAS G12C, rare in the pancreas), daraxonrasib acts broadly against any RAS pathway mutation," detailed Mariano Ponz, an oncologist at the Liver and Pancreas Cancer Area of the Cancer Center Clínica Universidad de Navarra (CUN), in a recent interview with a patient who had benefited from the therapy through clinical trials. "This allows it to benefit 90-95% of pancreatic cancer patients, compared to the previous 2-3%," he added.
In May, at the global medical oncology conference held in Chicago (ASCO), the latest data were presented with simultaneous publication in The New England Journal of Medicine: in patients pretreated with standard chemotherapy, the drug doubled overall survival, increasing from a median of six months to extended survivals. Previous studies reported an overall survival of 14.5 months and 7.6 months progression-free survival.
An emergency approval due to clinical need
The EMA's recommendation is the result of a staged evaluation of data on daraxonrasib, a drug identified within the framework of the EMA's Cancer Medicines Pathfinder project as a high-priority medicine capable of addressing this unmet medical need.
In the future, the EMA, in agreement with the CHMP, will consider the possibility of subjecting other drugs in the development phase to a gradual evaluation process, provided that this approach is feasible and is expected to accelerate the evaluation. This decision will be made on a case-by-case basis, assessing whether the drug is likely to address an unmet medical need and whether it is expected to be of great interest from a public health perspective, especially regarding therapeutic innovation.