Revolution Medicines ships experimental pancreatic cancer drug

Revolution Medicines has begun shipping its experimental pancreatic cancer drug daraxonrasib to doctors and patients under an early access program authorized by the Food and Drug Administration, the company’s CEO, Mark Goldsmith, said Friday evening at an event hosted by the outlet held alongside the annual meeting of the American Society of Clinical Oncology.

“We are now shipping the drug,” Goldsmith said. The drug is not yet approved. But patients with pancreatic cancer have been pressing for it since mid-April, when the company reported striking results from a Phase 3 clinical trial. Those results showed patients treated with daraxonrasib lived nearly twice as long as those who received standard chemotherapy, an outcome described as unprecedented in the field.

Pancreatic cancer is one of the deadliest cancers. The five-year survival rate hovers around 12%, according to the National Cancer Institute. Treatment options are limited, and most patients are diagnosed at an advanced stage. That backdrop explains why any sign of a better drug gets immediate attention.

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The early access program, sometimes called expanded access or compassionate use, allows patients with serious or life-threatening conditions to try a drug that hasn’t received full FDA approval. The firm handles the logistics — manufacturing, shipping, and monitoring — without charging patients, though insurers may cover some costs. The FDA generally requires that the company is actively pursuing approval and that there’s no comparable therapy available.

The company’s daraxonrasib targets a specific mutation in the KRAS gene, a driver of many pancreatic cancers. The protein it codes for, RAS, has been considered undruggable for decades until recent advances. The drug belongs to a class called RAS inhibitors. For patients whose tumors carry the G12X mutation, it appears to shrink tumors and slow progression.

But early access comes with caveats. Some oncologists point out that Phase 3 data, while promising, is still a single trial. Broader safety and efficacy will only be confirmed after more patients are treated and the FDA reviews the full application. “It’s encouraging, but we need to see longer follow-up and real-world outcomes,” one academic oncologist told a medical news outlet. The firm has said it plans to submit a new drug application later this year.

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The ASCO meeting, where the announcement was made, is the largest gathering of cancer specialists in the world. Thousands of researchers, doctors, and drug company executives attend. The setting gave the company a high-profile platform to announce the shipping start. For many in the audience, the news was a reminder of how quickly the landscape for pancreatic cancer treatment is shifting — even if the final regulatory verdict is still months away.

It did not disclose how many patients have received the drug so far under the program. It said shipments are going to physicians who have submitted requests and met the program’s eligibility criteria. Supply is expected to be limited initially; the company is manufacturing the drug at its own facilities and relying on contract manufacturers for additional capacity.

Patients or doctors interested in the early access program can find details on Revolution Medicines’ website. It has set up a dedicated team to handle inquiries. For a patient population that has faced decades of few options, the arrival of a drug that might extend life — even if not yet approved — is a tangible step forward.

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Still, the drug is not for everyone. Only patients with the specific KRAS mutation that daraxonrasib targets are eligible. Testing for that mutation is standard in many cancer centers, but not all patients have access. The firm has said it is working to broaden awareness and testing.

The pancreatic cancer community is watching closely. If the FDA ultimately approves daraxonrasib, it would become only the second targeted therapy for pancreatic cancer, after a drug called olaparib for a different genetic subset. The approval process typically takes several months after a submission.

Whether the early access program will provide enough real-world data to support approval — or reveal unexpected side effects — remains to be seen. For now, the drug is moving from clinical trial vials into the hands of patients who have little else to try.