The U.S. Food and Drug Administration (FDA) has approved the first drug shown to substantially extend the lives of people with advanced pancreatic cancer, nearly doubling survival time in a key clinical trial.
The pill, Rasonque (daraxonrasib), was cleared Wednesday for adults whose pancreatic cancer has spread and who have already had at least one other drug treatment, or who cannot take combination chemotherapy.
In a trial of 500 adults, people who took Rasonque lived a median of 13.2 months (half lived longer and half for a shorter time), compared with 6.7 months for those on standard chemotherapy, the FDA said.
The once-daily tablet blocks RAS, a cell protein that fuels tumor growth in most pancreatic cancers. It is the first drug of its kind, reports The New York Times.
About 67,000 people in the United States are diagnosed with pancreatic cancer each year. Most of those cases are pancreatic adenocarcinoma, which starts in the cells lining the ducts of the pancreas, according to the National Cancer Institute.
The disease is often caught late and has had few effective treatments, so it accounts for an outsized share of cancer deaths.
“This drug showed unprecedented results in an area of high unmet need,” said Dr. Angelo de Claro, director of the FDA’s Oncology Center of Excellence.
Acting FDA Commissioner Kyle Diamantas said the approval gives patients a critical new option for a historically hard-to-treat cancer and credited agency scientists with the speed of the review.
Dr. Brian Wolpin of the Dana-Farber Cancer Institute in Boston, who led the key study, told The Times he was stunned by the results.
“I have not seen anything like that before in trials we have run in pancreatic cancer,” he said.
The drug is not a cure, and it can cause harsh side effects. The most common are rash, diarrhea, mouth sores, nausea, fatigue, vomiting, belly pain, swelling, loss of appetite and bleeding.
Maker Revolution Medicines Inc. set a list price of $39,800 a month — about $478,000 for a year of treatment — though most of that is expected to be covered by insurance, with out-of-pocket costs varying, according to The Times.
More than 2,000 patients have already received the drug free through an early access program that began in May. The company said they will move to insurance coverage.
Former U.S. Sen. Ben Sasse of Nebraska, who said in December that he had metastatic pancreatic cancer, has credited the drug with extending his life after receiving it in a clinical trial, according to The Times.
Black Americans are disproportionately burdened by pancreatic cancer, facing higher incidence rates and poorer outcomes than other racial and ethnic groups. Because pancreatic cancer is often diagnosed at an advanced stage, new treatment options for metastatic disease are especially important. However, a major therapeutic advance does not automatically equate to equitable outcomes for all patients.
While daraxonrasib approval is a major breakthrough for metastatic pancreatic cancer, which has historically had limited treatment options, ensuring that its benefits reach Black patients will require attention to the persistent disparities that shape pancreatic cancer diagnosis, treatment, and survival.
Daraxonrasib is a RAS-targeted therapy, reflecting the growing importance of understanding a patient’s tumor’s molecular characteristics. The recent FDA approval further reinforces the role of biomarker and molecular testing in planning pancreatic cancer treatment.
For clinicians, ensuring patients across practice settings — particularly community oncology settings serving historically underserved populations — have access to appropriate testing and specialist referrals.
The approval may have implications for treatment sequencing in metastatic pancreatic cancer. While Rasonque is approved for patients who have received at least one prior systemic therapy, its role in clinical practice may extend beyond a traditional second-line setting for some patients.
An FDA spokesperson told BlackDoctor Pro that, “In accordance with its approved indication, oncologists will likely position Rasonque as a standard second-line therapy. However, it may also serve as a frontline option for patients who are not candidates for existing standard-of-care regimens.”
In accordance with its approved indication, oncologists will likely position Rasonque as a standard second-line therapy. However, it may also serve as a frontline option for patients who are not candidates for existing standard-of-care regimens.
FDA Spokesperson
This distinction could be especially important for patients whose performance status, comorbidities or other clinical factors limit their ability to tolerate existing multiagent regimens. As oncologists begin incorporating Rasonque into practice, treatment sequencing, patient selection, and access will likely become important considerations.
A new FDA-approved drug for advanced pancreatic cancer is only the first step. Coverage, affordability, oncology infrastructure, and referral pathways will influence which patients receive treatment. Oral therapies like daraxonrasib can potentially reduce some treatment burdens associated with chemotherapy, but they can also introduce new challenges involving pharmacy access, adherence, and out-of-pocket costs.
Clinicians will also need to proactively identify financial, transportation, and care coordination barriers that could prevent eligible patients from starting or remaining on treatment.
Additionally, understanding whether disparities in access to high-volume cancer centers and clinical trials could also affect adoption of emerging targeted therapies.
While breakthroughs in cancer treatment are significant, in many instances, their impact can only go so far when access is limited to patients most affected by the disease.
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