Oncologists say newly approved pancreatic cancer drug is a breakthrough for treatment

Oncologists are hailing a recently approved drug as a game changer for pancreatic cancer treatment.

The FDA on Aug. 26 approved Revolution Medicines’s daraxonrasib, which will be sold under the brand name Rasonque, to treat metastatic pancreatic adenocarcinoma in patients who’ve undergone prior chemotherapy treatment.

In Phase 3 clinical trials, daraxonrasib increased patients’ median overall survival time to 13.2 months compared to 6.7 months for patients taking standard chemotherapy treatment when measured from the time of randomization in the trial.

That kind of success is “like climbing Mount Everest for the first time,” Mitesh Borad, an oncologist and professor of medicine at Mayo Clinic in Arizona, told Healthcare Brew. Borad served as principal investigator for the three Phase 3 trial sites located at the Mayo Clinic’s Minnesota, Arizona, and Florida campuses. He was also a consultant for Revolution Medicines prior to the clinical trial.

The trial was conducted at 59 sites in six countries.

When initial results from the trial were released in April, the drugmaker’s stock shot up 58% in two days from $96.43 to $152.54. Since then, the stock has continued to grow gradually. It reached $208.04 as of Sept. 2.

How it works. Pancreatic cancer is known to be very hard to treat, largely due to a lack of early detection tests, meaning that by the time the cancer is found, it’s often advanced. It also grows rapidly, Borad said. The five-year survival rate is just 13%, according to the American Cancer Society.

Daraxonrasib, which is taken as a once-daily pill, is the first treatment to target a mutation on the KRAS gene, which is found in most pancreatic cancer patients. Scientists have been trying to target the gene for decades with little success.

The KRAS gene is one of three in the RAS family of genes. KRAS helps regulate cell growth by working as a kind of on-off switch. The mutation seen in pancreatic cancer patients causes the gene to get stuck in the “on” position, therefore causing uncontrolled cell growth, according to MD Anderson.

Daraxonrasib essentially throws “a wrench in the machine” and stops the mutation from fueling that growth, according to Borad.

The benefits. Beyond extending their lifespan, daraxonrasib was found to improve quality of life for patients in the trial, too.

The IV chemotherapies that have been standard of care for pancreatic cancer tend to come with significant side effects, according to Vincent Chung, a medical oncologist specializing in pancreatic and gastrointestinal cancers at City of Hope, a comprehensive cancer center in Los Angeles. Chung served as principal investigator for the Phase 3 trial site at City of Hope.

While daraxonrasib does come with side effects such as rash that in some cases can be severe, the pill’s side effects are generally “pretty well managed” and not as intense as what some patients experience with chemo, Chung said.

Daraxonrasib may also allow patients to stay on optimal doses of chemo for their disease for longer periods rather than having to lower their dosage to mitigate hematological side effects, Chung added.

In traditional pancreatic cancer treatment, patients go from one kind of IV chemo to another. Because the treatments are given sequentially, the side effects tend to be cumulative, according to Chung. Those side effects can sometimes make a patient’s blood counts drop so low their oncologist has to reduce the chemo dosage—or stop it altogether—and wait for the blood counts to recover before they can continue treatment.

Daraxonrasib, however, doesn’t cause the same drop in blood counts, and “you’re able to potentially select out a different population of cells that now are more responsive to IV chemotherapy again when you have to go back to it after daraxonrasib,” Chung said.

Future potential. While the treatment is currently only authorized for late-stage patients who’ve already undergone chemotherapy, studies are ongoing to determine whether daraxonrasib would be effective prior to or in combination with chemo, Borad said.

It’s also being studied to see if it could prevent recurrence in patients without metastatic disease who’ve undergone surgery and chemo. Between 65% to 75% of patients with pancreatic ductal adenocarcinoma, the most common form of pancreatic cancer, see their disease recur within five years post-treatment, according to a 2023 study.

“I think this is just the beginning, not just for daraxonrasib, but for the field at large going after RAS as the target,” Borad said.

Scientists are also studying whether daraxonrasib could work for other cancers that involve RAS mutations, such as lung, biliary tract, uterine, and some types of ovarian cancers, he added.

It’s unclear how many patients are currently eligible for daraxonrasib under the FDA’s approval, but “it will be a large number” since around 90% of pancreatic cancer patients have a RAS mutation, Chung said. Roughly 67,530 people in the US will be diagnosed with pancreatic cancer this year, according to the American Cancer Society.

Anthony Mancini, Revolution Medicines’s chief global commercialization officer, said in a statement emailed to Healthcare Brew that the company’s “manufacturing scale is sufficient to support US launch needs.”

Access to the treatment may be a challenge, as daraxonrasib comes with a monthly list price of $39,800 before insurance, according to an SEC filing. It’s so far unclear how much insurance will cover. Comparatively, folfirinox, a combination chemotherapy regimen commonly used to treat metastatic pancreatic cancer, was estimated to have a monthly acquisition cost of about $5,501 per month, per a 2024 analysis presented at the American Society of Clinical Oncology’s annual meeting.

Mancini said the price “reflects its differentiation and the meaningful benefit it provides for patients,” and that Revolution has a “comprehensive patient support program” to help patients gain access.

“It’s just an exciting time,” Chung said. “It’s a wonderful time in terms of being able to do research in this field.”