The U.S. Food and Drug Administration has approved a new once-daily pill for certain patients with metastatic pancreatic cancer after a clinical trial showed that the treatment nearly doubled median overall survival compared with chemotherapy.
The drug, daraxonrasib, marketed as Rasonque by Revolution Medicines, was approved Wednesday for adults with metastatic pancreatic adenocarcinoma who have previously received at least one systemic treatment or are not candidates for certain combination therapies.
The approval represents a significant development for pancreatic cancer, one of the most difficult cancers to detect and treat. Researchers have spent years attempting to develop therapies capable of effectively targeting RAS proteins, which play a major role in driving tumor growth.
Survival Nearly Doubled in Major Trial
The FDA's decision was based largely on results from the phase 3 RASolute 302 clinical trial, which enrolled 500 patients with previously treated metastatic pancreatic cancer.
Patients receiving daraxonrasib had a median overall survival of 13.2 months, compared with 6.7 months among patients who received chemotherapy.
That difference represents nearly a doubling of median survival and has been described by researchers as a landmark advance for patients with advanced pancreatic cancer.
The treatment is particularly notable because it targets the underlying biological pathway that drives many pancreatic tumors rather than functioning as traditional chemotherapy.
How the New Treatment Works
Rasonque is taken orally once a day and targets multiple forms of RAS, a group of proteins involved in signals that can cause cancer cells to grow and survive.
Daraxonrasib is described as a multi-selective RAS(ON) inhibitor. Researchers say it works with another protein called cyclophilin A to block RAS signaling. More than 90% of pancreatic cancers contain cancer-driving mutations in the KRAS gene, which belongs to the RAS family.
The ability to directly interfere with this pathway has been a major goal of cancer researchers for decades.
A New Option for Advanced Pancreatic Cancer
Pancreatic adenocarcinoma is particularly challenging because it frequently goes undetected until it has already spread.
According to data cited by the Washington Post, only about 14% of people diagnosed with pancreatic cancer survive for five years or longer. By the time many patients are diagnosed with metastatic disease, treatment options can be limited.
The FDA accelerated the drug's review through several programs, including priority review, orphan-drug status and breakthrough-therapy designation. The agency also allowed expanded access to the treatment before its formal approval.
Daily Pill Could Reduce Treatment Burden
Another potential advantage is the method of administration.
Unlike intravenous chemotherapy, which can require patients to spend hours receiving treatment in a clinical setting, daraxonrasib is taken as a tablet.
Researchers involved in the clinical trial have said the drug generally produces fewer burdensome side effects than traditional IV chemotherapy, although it is not without risks.
Reported adverse effects include rash, diarrhea, mouth sores, nausea, fatigue, vomiting, abdominal pain, swelling, reduced appetite and bleeding.
Patients and physicians will therefore need to weigh the drug's potential survival benefit against its side effects and individual medical circumstances.
Approval Could Open Door to More RAS Treatments
Scientists believe the significance of the approval could extend beyond pancreatic cancer.
Because RAS and KRAS mutations are involved in several types of cancer, researchers are investigating whether similar approaches could eventually help patients with other tumors.
A clinical trial of daraxonrasib in lung cancer is already underway, according to researchers involved in the pancreatic cancer program.
For patients with metastatic pancreatic cancer, however, the immediate significance is more direct: the FDA-approved treatment provides a new targeted option in a disease where effective therapies have historically been difficult to develop.
The nearly doubled median survival seen in the pivotal trial does not mean every patient will experience the same benefit, but researchers say the results mark an important step forward in the fight against one of the deadliest forms of cancer.
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