Adnkronos (Adnkronos) – The Italian Association of Medical Oncology (AIOM) is asking Revolution Medicines "to initiate a compassionate use program for daraxonrasib, thus making the drug available free of charge, prior to regulatory approval, to patients with metastatic pancreatic cancer already treated with chemotherapy." This is "a targeted therapy that, although it has so far produced results only in terms of prolonging survival and not cure, represents unquestionable progress for these patients," AIOM states in a statement. "In fact, in the pivotal RASolute 302 clinical trial, survival doubled to 13,2 months, compared to 6,6 months with traditional chemotherapy."
These results led to approval by the U.S. Food and Drug Administration (FDA) at the end of August 2026. In Italy, approximately 13.600 new pancreatic cancer diagnoses were estimated in 2024.
“Last June in Chicago, at the American Congress of Clinical Oncology, the results of the RASolute 302 study were greeted with a standing ovation by the oncologists who attended the presentation of the data,” explains Massimo Di Maio, president of AIOM. “For decades, there had been no significant progress in this difficult-to-treat neoplasm.
Now there's the issue of access to the treatment, which is not available outside the United States. The pharmaceutical company that produces it, Revolution Medicines, had launched an expanded access program in the US, thus ensuring the treatment's availability before regulatory approval by the FDA.
In Europe, however, no compassionate use program has been opened, and there is no news of the company submitting a registration dossier to the EMA, the European regulatory agency, for approval of the drug. But patients—he emphasizes—can't wait.
EMA has already designated daraxonrasib as an orphan drug, indicating its scientific importance and clinical benefits in pancreatic cancer. This designation also provides a preferential pathway for subsequent regulatory evaluation. Furthermore, the statement continues, EMA has initiated a rolling review program (progressive review of data), which expedites the regulatory process before receiving formal submission of the dossier from the pharmaceutical company.
"A critical point at this stage is Revolution Medicines' failure to activate an expanded access program, which would allow early access to the drug," notes Chiara Cremolini, member of the national board of directors of AIOM and director of the Clinical Trials Program at the Pisa University Hospital, one of the four Italian centers involved in the RASolute 302 study. "Italian oncologists are therefore unable to use a potentially effective therapy. Not only that," she points out, "Revolution Medicines has activated a procedure, defined as global nominal use, which in reality is not an early access program, but rather a mechanism to facilitate the importation of the drug, at the price agreed upon in the United States, which corresponds to approximately $39,000 per month. Therefore, daraxonrasib, which is not yet available in Italy, can currently be imported into our country through hospitals at patients' expense at an exorbitant cost. This mechanism is ethically unacceptable and creates serious inequities to the detriment of patients. AIOM has always "We emphasize the importance of protecting the National Health Service, which is universal and guarantees care for all patients," Cremolini continues. "We are also aware of the need to ensure the system's sustainability, which would be jeopardized by the use of another early access tool, namely Law 648/1996, which allows for full reimbursement by the National Health Service. We have discussed these issues with AIFA (the Italian Medicines Agency), "with whom we believe it is essential to continue the dialogue."
"We are asking the company," Di Maio reiterated, "to create a compassionate use program so that daraxonrasib can be made available to Italian patients free of charge, pending regulatory approval, as was already the case in the United States prior to the FDA's approval. The scientific community has repeatedly urged the company to do so, but so far the response has been tentative. Oncologists are deeply concerned that this is the first clear application of the principle established by the US administration of 'most favored nation' pricing, which establishes that the cost of drugs in the United States must be equal to or lower than the lowest among countries with a comparable per capita GDP, including Italy. Indeed, at the end of the negotiations, the price of the drug in our country will likely be lower than that set in the United States. Hence," he concluded, "the company's limited interest in submitting the registration dossier to the EMA."
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