An experimental drug that could be a breakthrough in the treatment of one of the deadliest known cancers is gaining demand in clinics while awaiting full government approval.
Daraxonrasib, a drug that targets the gene mutation behind most pancreatic cancer diagnoses, was given the green light by the Food and Drug Administration on April 30 to begin an early access program for some patients.
Described by some as a “miracle drug”, the drug almost doubled the life span of some pancreatic cancer patients in clinical trials. While the Early Access program has provided a potential opportunity for patients suffering from cancer, clinics are already struggling to meet demand.
Dr. Daniel King, a medical oncologist at Northwell Health’s Zuckerberg Cancer Center, told Reuters, “The public took the FDA’s announcement for granted … which has led to a flood of requests from patients.” “Cancer centers are figuring out how to connect with our own institutions, opening up protocols to provide access.”
According to the National Cancer Institute, pancreatic cancer has the highest mortality rate 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 American Cancer Society, approximately 67,000 people in the United States will be diagnosed with pancreatic cancer this year, and 53,000 will die from the disease.
Here’s what to know about the drug, how it works and when it will be available to the public.
What is Darxonrasib?
Daraxonrasib is an experimental pancreatic cancer drug made by Revolution Medicines. It is a once-daily pill that prolongs survival for people who have pancreatic cancer that has metastasized, or spread, to other parts of the body.
Clinical trial results released late in April found that patients who took daraxonrasib lived an average of 13.2 months, nearly double the average of 6.7 months for those who received standard chemotherapy.
How does daraxonrasib work?
This drug works by targeting the most common gene mutations behind pancreatic cancer.
More than 90% of patients with cancer have a mutation in a gene called KRAS, Dr. Christopher Liu, oncologist and professor in the department of medicine at the University of Colorado Anschutz Medical School, previously told USA TODAY.
Researching genes allows medicine to target the mechanisms behind cancer, while potentially limiting the side effects that come with extensive treatment.
“The drug binds to the activating pocket (of the gene mutation) and turns it off. It’s almost like you have a bullhorn and you cover it so no sound can come out,” Liu said. “There is a possibility that this targeted therapy for pancreatic cancer could work more effectively than chemotherapy, meaning it could potentially be a treatment with less toxicity.”
Is Darxonrasib FDA-approved? Is it available to the public?
Daraxonrasib is currently undergoing FDA review for full approval. The agency had already given Revolution Medicines permission to begin a limited rollout as part of an expanded access program in late April.
This green light has allowed the drugmaker to offer the drug for free to some patients who have previously been treated for pancreatic cancer that has later spread to other parts of the body.
Under the FDA’s new, accelerated drug review protocol, daraxonrasib could receive full approval just a month or two after a formal application is filed — much sooner than the usual 10 to 12 months.
Revolution Medicines has not yet filed the application, but said in a call in early May that it was “making every effort” to do so, Reuters reported.
Who can receive Daraxonrasib?
News of the expanded access program has prompted a flood of cancer clinics with requests to join, Reuters reported on May 14. Demand for the drug is already high and is expected to increase, doctors told the news outlet.
The expanded access program was approved by the FDA specifically for patients who have previously been treated for pancreatic cancer that has later metastasized. To access it, a licensed treating physician must submit a request to Revolution Medicines, where an institutional review board looks at it. She told Reuters the drugmaker expects to respond to these requests within two business days of receipt.
If the company decides the drug is suitable for the patient, those details are submitted to the FDA. Patients would then have to be monitored by hospital monitoring boards and notify both Revolution Medicines and the FDA about serious side effects or other issues.
While the expedited voucher program allowing this early access was seen as an accomplishment of former FDA Commissioner Dr. Marty Macri, who resigned on May 12, no intended changes to the program have been reported.
Former senator claims ‘miracle drug’
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 had spread to his lungs and liver.
“I have much less pain than I did four months ago when I was diagnosed, and my tumor volume has decreased by a massive 76% in the last four months,” Sasse said during the interview. “So maybe I’ll go and live for a year instead of a few months.”
While doctors initially gave him three to four months to live when he was diagnosed in December, he said he now has more time thanks to “providence, prayer and a miracle drug.”
Contributing: Ken Altaker, Selene Martin, USA TODAY; reuters