Pancreatic cancer breakthrough as US approves drug that nearly doubles survival time

A new treatment for advanced pancreatic cancer has been approved in the United States after a major clinical trial found patients receiving the drug survived almost twice as long as those given standard chemotherapy.

The US Food and Drug Administration (FDA) approved daraxonrasib, marketed as Rasonque, on 26 August for certain adults with metastatic pancreatic adenocarcinoma – the most common form of pancreatic cancer.

The once-daily oral treatment is the first approved drug designed to inhibit multiple forms of RAS, a family of proteins which play a major role in driving pancreatic cancer.

In a Phase 3 trial involving around 500 patients whose metastatic pancreatic cancer had previously been treated, median overall survival among those receiving daraxonrasib was 13.2 months.

For patients receiving standard chemotherapy, median survival was 6.7 months.

The results represent one of the most significant improvements in survival seen for advanced pancreatic cancer in decades.

Daraxonrasib works by targeting abnormal RAS proteins responsible for signalling cancer cells to grow. Mutations affecting RAS are found in the vast majority of pancreatic cancers, with mutations in the KRAS gene particularly common.

For decades, RAS proteins were considered extremely difficult to target effectively with medicines, making the development of treatments capable of blocking them a major focus of cancer research.

The FDA approved daraxonrasib for adults with metastatic pancreatic adenocarcinoma who have received at least one previous systemic therapy, or who are not suitable candidates for treatment involving multiple systemic drugs.

The treatment is therefore not currently a cure for pancreatic cancer or a replacement for first-line treatment for everyone diagnosed with the disease.

However, specialists have described the development as potentially transformative for a cancer which continues to have one of the poorest survival rates of any major cancer.

The FDA had previously granted daraxonrasib Breakthrough Therapy and Orphan Drug designations and allowed eligible patients access to the investigational treatment before its formal approval.

Its eventual approval also came through the FDA’s Commissioner’s National Priority Voucher programme, designed to dramatically shorten the review period for medicines considered capable of addressing major unmet medical needs.

Researchers are now studying whether daraxonrasib could benefit patients at other stages of pancreatic cancer and whether drugs targeting RAS could be effective against other cancers driven by similar mutations.

The development could have implications far beyond the United States.

Regulatory authorities elsewhere are considering daraxonrasib, raising the possibility that patients in other countries could eventually gain access to the treatment if further approvals are granted.

For pancreatic cancer – where improvements in survival have historically been difficult to achieve – the findings represent an important step towards treatments that directly target the genetic mechanisms driving the disease rather than relying solely on conventional chemotherapy.

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