FDA Approves New Drug That Could Change How Doctors Treat Pancreatic Cancer

The U.S. Food and Drug Administration has approved a new drug to treat advanced pancreatic cancer — one of the deadliest forms of cancer known to doctors. The drug, called daraxonrasib, will be sold under the brand name Rasonque. It was made by a biotech company called Revolution Medicines. The approval is a major milestone because Rasonque is the first drug to attack a genetic cause of pancreatic cancer, rather than just trying to slow the disease with chemotherapy.

In a clinical trial, patients with advanced pancreatic cancer who took Rasonque as a second treatment lived a median of 13.2 months. That is almost double the 6.7 months seen in patients who received standard chemotherapy. A 'median' survival means that half the patients lived longer than that number and half lived shorter. Doctors say this difference is a big deal for a cancer that has been so hard to treat for so long.

Pancreatic cancer has one of the lowest survival rates of any cancer. Only about 8 out of every 100 patients are alive five years after being diagnosed. For decades, researchers tried to find better treatments, but new drugs made very little difference. That made pancreatic cancer one of the most frustrating challenges in all of medicine.

Scientists found that a faulty gene called KRAS is linked to more than 90 percent of pancreatic cancers. A gene mutation is a change in the instructions inside a cell that can cause it to grow out of control. Researchers believed that if they could target this mutation with a drug, they might be able to stop the cancer. The problem was that the KRAS protein was extremely hard to attack — scientists even called it an 'undruggable greasy ball' because tiny drug molecules couldn't stick to its surface.

A scientist named Gregory Verdine found a way around this problem using a concept called 'molecular glues.' His idea was to use a small molecule that first latches onto a different protein, changing its shape so that it then sticks to the KRAS protein. This creates a three-part complex — the drug and two proteins locked together — which blocks the cancer-driving signal. That discovery became the foundation for Rasonque, which can target both mutated and normal forms of the RAS protein family.

Because the drug affects both cancerous and healthy cells, it can cause side effects. The most common side effect is a rash, which showed up in 85 percent of patients in the study, with about 14 percent of those cases being severe. More than half of patients also experienced stomatitis, which means painful sores and swelling in the mouth and throat. Doctors say they will need to learn the best ways to help patients manage these side effects.

Revolution Medicines CEO Mark Goldsmith said the approval is a game-changer for patients. Before Rasonque, doctors treating pancreatic cancer that had stopped responding to chemotherapy had very few choices. 'More chemo. Something that wasn't working. They gave you more of it,' he said. Now, patients have a real new option.

More than 2,000 pancreatic cancer patients had already started taking Rasonque through a special early-access program before it was officially approved. The drug is expected to sell very well, making Revolution Medicines one of the most valuable biotech companies around. However, Revolution has not yet announced how much the drug will cost.

Rasonque is not a cure, and the cancer can eventually find ways to resist the drug. But scientists are hopeful that it may work even better when combined with other treatments, like immunotherapy. There are already about 80 other drugs being tested in clinical trials that also target the RAS gene family, not just in pancreatic cancer but also in lung, colon, and other cancers. Researchers believe this approval will speed up progress across all of those efforts.

Revolution Medicines is already running new clinical trials to test Rasonque in combination with other drugs and as a first treatment instead of a second one. Cancer researcher Channing Der said the success of Rasonque is exciting because it is pushing the entire field forward at a faster pace. The hope is that future studies will push survival times even further and bring scientists one step closer to a cure.