The FDA has approved a first-of-its-kind oral medication for advanced metastatic pancreatic cancer. Experts regard the daily pill, daraxonrasib (Rasonque), as a groundbreaking new therapy for patients facing an exceptionally challenging illness.
“This drug showed unprecedented results in an area of high unmet need,” said Angelo de Claro, MD, director of the FDA’s Oncology Center of Excellent, in a statement.
“There is new hope for pancreatic cancer,” says the trial’s principal investigator, Andrew Hendifar, MD, medical director of the Cancer Clinical Trials Office and the Gastrointestinal Oncology Disease Research Group at Cedars-Sinai in Los Angeles.
“There is a medication that can substantially improve the life of patients with pancreatic cancer for the first time,” he says. “With additional research and progress, the future is brighter.”
New Drug Nearly Doubled Pancreatic Cancer Survival Time
In the trial, researchers randomly assigned 500 patients with previously treated metastatic pancreatic ductal adenocarcinoma (mPDAC) to receive a daily daraxonrasib pill or chemotherapy. This specific type of pancreatic cancer accounts for more than 90 percent of all pancreatic-cancer cases.
The median survival time was 13.2 months for adults who took daraxonrasib, compared with 6.7 months for those who got chemotherapy.
“The medicine is highly effective and it’s a pill — the first oral medication for pancreatic cancers generally,” Dr. Hendifar says.
About 62 percent of participants reported severe side effects with daraxonrasib, compared with 70 percent with chemotherapy. Daraxonrasib side effects reported in the phase 1 and 2 trials included rash, diarrhea, mouth and throat inflammation, and fatigue.
How the New Drug Works
Daraxonrasib targets mutations in the KRAS gene, which is responsible for 92 percent of pancreatic cancer cases, Hendifar says. It’s also responsible for some cases of colon and lung cancer, among others, he adds.
When mutated KRAS genes prompt cells to divide and grow uncontrollably, it can cause cancer to form.
Daraxonrasib blocks the KRAS signal. “Until now, it has been undruggable,” Hendifar says.
“All of this science took time, but we have now figured this out and likely, just like anything else, once you figure something out, you can stand on the shoulders and make it better and better,” he says.
The new drug could lead to treatments for other types of cancer with KRAS mutations, Hong says. “It’s going to be an exciting time to see where this all plays out.”
Why Pancreatic Cancer Is Difficult to Treat
Symptoms of pancreatic cancer often don’t develop right away, and when they do, they’re fairly nonspecific (for example bloating, gas, and nausea). For that reason, pancreatic cancer is typically diagnosed when it’s already metastatic, says Marc Kahn, MD, a hematologist-oncologist and chief of hematology at the University of Nevada in Las Vegas. Dr. Kahn was not involved with the clinical trials.
“We’ve not been able to develop chemotherapy drugs that have effectively impacted the rate of progression of pancreatic cancer,” he says. “This has been one of the most difficult cancers to treat.”
The daraxonrasib clinical trial results are a “substantial development,” Kahn says.
The study results also point the way to other pancreatic cancer treatments, Hong adds. “This will lead us down a path where increasingly more effective drugs will be developed. And maybe someday this may lead to the cure for pancreatic cancer.”
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