An experimental drug that could be a breakthrough in the treatment of one of the deadliest known cancers is driving demand at clinics while awaiting full government approval.
As USA Today reports, Daraxonrasib, a drug that targets the gene mutation behind most pancreatic cancer diagnoses, was given the green light to begin an early access program for some patients on April 30 by the Food and Drug Administration (FDA).
What is Daraxonrasib, and How Does it Work?
Daraxonrasib is a targeted therapy made by Revolution Medicines. It is a once-daily tablet that has been shown to extend survival times for people who have pancreatic cancer that has metastasized, or spread to other parts of the body.
The drug works by targeting the most common gene mutation behind pancreatic cancer. Over 90% of patients diagnosed with the cancer have a mutation in a gene called KRAS, which fuels tumor growth. Daraxonrasib is designed to block these mutated proteins. Honing in on the gene itself allows the drug to target the mechanism behind the cancer while potentially limiting the side effects that come with broader treatments.
“The drug binds to the activating pocket of (the gene mutation) and shuts it down. It’s almost like if you have a bullhorn and you cover it up so no sound can escape,” says Dr. Christopher Lieu, an oncologist and professor at the University of Colorado Anschutz Medical School’s Department of Medicine. “There’s a possibility that this targeted therapy for pancreatic cancer could work more effectively than chemotherapy, meaning it could be a treatment with potentially less toxicity.”
Described by some as a “miracle drug,” the medication has been shown in clinical trials to extend the lifespans of some pancreatic cancer patients by nearly double. In clinical trials, the daily pill nearly doubled overall survival times to over 13 months, compared to less than 7 months for standard chemotherapy. The drug was also shown to reduce the risk of death by 60% in trials.
The FDA Approval Process
The drug is not yet fully FDA-approved, but the FDA has granted early expanded access and is expected to conduct an expedited priority review to speed up availability. This green light has allowed the drugmaker to offer the medication for free to some patients who have previously been treated for pancreatic cancer that has since spread to other parts of the body.
Under the FDA’s new, expedited drug review protocol, Daraxonrasib could get full approval as quickly as a month or two after a formal application is filed – much quicker than the typical 10 to 12 months. Revolution Medicines has yet to file the application but said in an early May call that there is a “full-throttle effort” to do so, reported Reuters.
The expanded access program was approved by the FDA specifically for patients who have previously undergone treatment for pancreatic cancer that has since metastasized. In order to access it, a licensed treating physician must submit a request to Revolution Medicines, where an institutional review board then looks it over. The drugmaker expects to respond to these requests within two business days of receipt, it told Reuters.
If the company decides the drug is a good fit for the patient, those details are then submitted to the FDA. Patients will then have to be followed by hospital monitoring boards and serious side effects or other issues will have to be reported to both Revolution Medicines and the FDA.
Pancreatic Cancer is One of the Deadliest
Pancreatic cancer has one of the highest mortality rates of all known cancers, with only 3% of people surviving five years after diagnosis if it has spread to distant parts of the body, according to the National Cancer Institute. Around 67,000 people in the United States will be diagnosed with pancreatic cancer this year, and 53,000 will die of the disease, according to the American Cancer Society.
Former Republican Senator Ben Sasse told “60 Minutes” host Scott Pelley in April that Daraxonrasib is a “miracle drug” that has helped him manage his pancreatic cancer, which spread to his lung and liver. “I have much, much less pain than I had four months ago when I was diagnosed, and I have a massive 76% reduction in tumor volume over the last four months,” Sasse said during the interview. “So maybe I’m going to crank and live a year instead of a handful of months.”
While the early access program has provided a potential avenue for patients with cancer that has spread, clinics are already struggling to keep up with demand. “The public caught wind of the FDA announcement… which has triggered a deluge of patient requests,” Dr. Daniel King, medical oncologist at the Zuckerberg Cancer Center of Northwell Health, told Reuters. “Cancer centers are all figuring out how to engage with our own institutions, opening up the protocols to provide access.”
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