The U.S. Food and Drug Administration has approved a groundbreaking new drug for advanced pancreatic cancer, giving patients a new treatment option for one of the deadliest forms of cancer.
The FDA granted expedited approval on Wednesday to daraxonrasib, a first-of-its-kind daily pill designed to target mutated proteins that help pancreatic tumors grow.
Revolution Medicines will sell the drug under the brand name Rasonque. According to the company, a one-month supply will cost about $39,800.
New Pancreatic Cancer Drug Nearly Doubles Survival
The approval follows results from a clinical trial involving about 500 patients with metastatic pancreatic cancer. These patients had cancers that stopped responding to previous treatments.
Researchers compared patients who received daraxonrasib with those who received additional chemotherapy.
The results showed a significant difference in survival. Patients who took the new drug lived for a median of 13.2 months, compared with 6.7 months for patients who received chemotherapy.
In other words, the new treatment nearly doubled median survival in the study.
However, doctors stressed that daraxonrasib does not cure pancreatic cancer.
“This medicine is not a cure but a significant improvement,” said Dr. Andrew Coveler of the Fred Hutch Cancer Center.
FDA Approves Drug Ahead of Target Date
The FDA approved daraxonrasib more than six months ahead of its original target date.
FDA acting commissioner Kyle Diamantas said the agency has a responsibility to make important treatments available to patients as quickly as possible.
The approval could provide new hope for people with advanced pancreatic cancer who have already exhausted other treatment options.
How Daraxonrasib Targets Pancreatic Cancer
Daraxonrasib focuses on mutations in the RAS gene family, which normally helps regulate cell growth.
Among these mutations, KRAS plays an especially important role in pancreatic cancer. The mutated protein can continuously send signals that encourage cancer cells to grow.
For decades, scientists struggled to develop medicines that could effectively target KRAS. The structure of the mutated protein made it difficult for drugs to attach to it, leading researchers to describe KRAS as an “undruggable” cancer target.
Daraxonrasib takes a different approach. The drug acts like a molecular glue, allowing it to bind with multiple KRAS subtypes and interfere with the signals that drive tumor growth.
Side Effects Reported in Clinical Trial
Patients in the study experienced several side effects while taking daraxonrasib.
Reported problems included skin rashes, mouth sores, diarrhea and other digestive issues.
Despite these effects, the trial showed fewer severe side effects compared with some chemotherapy treatments, according to the reported study results.
Doctors say the balance between effectiveness and side effects could make the new drug an important addition to pancreatic cancer treatment.
Pancreatic Cancer Remains Difficult to Treat
Pancreatic cancer remains one of the most challenging cancers to diagnose and treat.
The disease often produces few noticeable symptoms during its early stages. As a result, doctors frequently detect pancreatic cancer after it has already spread to other parts of the body.
The American Cancer Society estimates that around 67,000 people in the United States will receive a pancreatic cancer diagnosis this year, while more than 52,000 people could die from the disease.
The five-year overall survival rate remains around 13%, highlighting the urgent need for better treatments.
Ben Sasse Helped Raise Awareness
Daraxonrasib gained wider public attention earlier this year when former U.S. Sen. Ben Sasse discussed his experience with pancreatic cancer on CBS’ 60 Minutes.
Sasse said the drug had helped reduce his pain.
Growing public interest eventually prompted the FDA to allow expanded access to the treatment before its formal approval. That program allowed certain eligible patients to receive the drug while regulators continued reviewing the application.
Drug Could Open Doors for Other Cancer Treatments
Doctors and researchers hope the approval will lead to more advances in treatments that target KRAS and other cancer-driving mutations.
Revolution Medicines is already studying its technology in several other cancers, including lung cancer.
Dr. Pashtoon Kasi of City of Hope Orange County said the development could encourage other companies to explore similar approaches.
The FDA’s approval therefore represents more than a new option for pancreatic cancer patients. It could also mark an important step toward developing targeted treatments for cancers that scientists have struggled to treat for decades.
