US approves first drug of new class against pancreatic cancer

The U.S. Food and Drug Administration (FDA) has approved Rasonque (daraxonrasib) - the first drug of a new class of targeted therapy for the treatment of metastatic pancreatic cancer. In a clinical trial, the median overall survival of patients on the new drug was 13.2 months versus 6.7 months with standard chemotherapy.

This was reported by the FDA. The drug was developed by the American biotechnology company Revolution Medicines.

Rasonque is an inhibitor of the RAS family of proteins, which play a key role in tumor growth. Mutations and abnormal RAS activity are found in most cases of pancreatic adenocarcinoma, the most common form of this cancer.

The drug is intended for adult patients with metastatic pancreatic adenocarcinoma who have already received at least one line of systemic therapy or who cannot undergo multicomponent systemic treatment.

Survival nearly doubled

The approval was based on the results of the international randomized trial RASolute 302 involving 500 adults with metastatic pancreatic cancer whose disease had progressed after previous treatment.

Half of the participants received daraxonrasib, while the other half received standard chemotherapy of the physician's choice.

The median overall survival in the new drug group was 13.2 months versus 6.7 months in the chemotherapy group. According to the researchers' estimate, the risk of death with daraxonrasib was approximately 60% lower.

The median progression-free survival also doubled - 7.2 months versus 3.6 months.

In addition, an objective tumor response to treatment was observed in 30% of patients receiving daraxonrasib, compared with 11% in the standard chemotherapy group.

The drug blocks one of the main mechanisms of tumor growth

Daraxonrasib is an oral RAS inhibitor that blocks the active form of the signaling protein RAS. This mechanism is involved in transmitting signals that stimulate the growth and proliferation of cancer cells.

According to the FDA, approximately 90-95% of the about 67,000 new cases of pancreatic cancer diagnosed annually in the United States are adenocarcinoma.

Although pancreatic cancer accounts for only a small fraction of all cancer diagnoses, it remains one of the most dangerous due to late detection, aggressive course, and limited number of effective treatments.

The FDA called the approval of Rasonque the emergence of a critically important new option for patients with a disease that has historically been difficult to treat.

FDA accelerated approval by half a year

Rasonque is taken as a tablet once a day. The recommended dose is 300 mg until disease progression or unacceptable toxicity.

The drug had previously received breakthrough therapy and orphan drug designations from the FDA, and its application was reviewed under an accelerated procedure. The regulator approved the therapy approximately 6.5 months ahead of the scheduled date.

Among the most common side effects, the FDA lists rash, diarrhea, inflammation of the oral mucosa, nausea, fatigue, vomiting, abdominal pain, swelling, decreased appetite, and bleeding.

The FDA approval applies to the use of the drug in the United States. Applications for daraxonrasib are also being considered by other regulators: Canada participated in the evaluation process, while the European Medicines Agency and the Japanese regulator acted as observers.

Source: FDA