A daily oral medication that has demonstrated the ability to double survival time for patients with advanced pancreatic cancer has received approval from the U.S. Food and Drug Administration (FDA), sparking immediate calls for its expedited introduction within the British National Health Service (NHS).
The drug, known as daraxonrasib and to be marketed in the U.S. as Rasonque, is intended for individuals with metastatic pancreatic cancer—meaning the cancer has spread—who have previously undergone chemotherapy. In a major clinical trial, patients taking daraxonrasib lived an average of 13.2 months, a substantial increase compared to the 6.7 months survival rate observed with standard chemotherapy.
This development has been recognized as one of the most critical breakthroughs in decades for pancreatic cancer treatment. However, the drug has not yet been approved for use in the United Kingdom. Pancreatic Cancer UK, a leading charity, has urged British regulators to expedite the assessment process to make the medication accessible to NHS patients.
"This exciting new drug is one of the most significant breakthroughs for pancreatic cancer in decades," stated Diana Jupp, chief executive of Pancreatic Cancer UK. "Its approval for patients in America is a source of real hope for people with pancreatic cancer and their loved ones worldwide."
Pancreatic cancer is a particularly lethal disease, with approximately 11,500 diagnoses annually in the UK and around 10,200 deaths. It ranks as the fifth leading cause of cancer death in the UK, despite accounting for only about three percent of all new cancer cases. Survival rates are starkly low, with fewer than six percent of patients alive five years after diagnosis.
Pancreatic cancer's aggressive nature and often late-stage diagnosis contribute to its difficulty in treatment. "Pancreatic cancer is uniquely devastating, half of people with the disease die within just three months of diagnosis," Jupp noted. "That is simply not long enough to make precious memories."
The disease frequently presents with vague symptoms until it has already metastasized, making early intervention challenging. Symptoms can include upper abdominal or back pain, unexplained weight loss, indigestion, changes in bowel habits, and jaundice. By the time the cancer is advanced, surgical options, the only potential curative treatment, are typically no longer feasible.
Daraxonrasib targets a common driver of pancreatic cancers: mutations in the KRAS gene. This gene, normally involved in cell growth regulation, can become permanently activated in cancer, leading to uncontrolled cell proliferation. For years, KRAS mutations were considered a significant hurdle in cancer drug development, often referred to as 'undruggable'.
Daraxonrasib functions by inhibiting the aberrant signaling pathways triggered by mutated KRAS and related proteins, thereby slowing the progression of cancer cells.
The FDA's approval was based on a trial involving 500 patients across North America, Europe, and Asia, with an average age of 66. These patients had metastatic pancreatic cancer and had previously undergone treatment. The trial compared daraxonrasib to standard chemotherapy, showing not only improved survival but also a longer period before disease progression. Patients on daraxonrasib experienced a median of 7.2 months before their cancer worsened, compared to 3.6 months for those on chemotherapy. Additionally, tumor shrinkage was observed in 30 percent of patients receiving daraxonrasib, versus 11 percent in the chemotherapy group. Side effects were also generally less severe with daraxonrasib, primarily consisting of rash, diarrhea, fatigue, and nausea.
While daraxonrasib is not a cure and cancer progression eventually occurred in trial participants, the results are considered highly significant given the historical lack of effective treatments for advanced pancreatic cancer. Clinical trials for daraxonrasib and other KRAS-targeting drugs are ongoing in the UK.
Pancreatic Cancer UK is now appealing to the Medicines and Healthcare products Regulatory Agency (MHRA), the National Institute for Health and Care Excellence (NICE), and other UK assessment bodies to expedite their review of daraxonrasib. "It is imperative that regulators here in the UK now respond with the same urgency," Jupp stated. "We must see the MHRA, NICE and assessment bodies across the UK enact fast-track approval processes for daraxonrasib. People with pancreatic cancer have been overlooked and left behind for too long. We must do everything possible to ensure the most promising new treatments are available on the NHS as soon as possible."