FDA approves once-daily Rasonque pill after trial nearly doubles pancreatic cancer survival

The Food and Drug Administration on Wednesday approved Rasonque (daraxonrasib), a once-daily tablet for adults with metastatic pancreatic adenocarcinoma who already tried systemic therapy or cannot take multiagent chemotherapy.

The drug, made by California-based Revolution Medicines, blocks multiple forms of the RAS protein that helps drive tumor growth in most pancreatic cancers. FDA materials call it a first-in-class RAS inhibitor for this disease. Acting FDA Commissioner Kyle Diamantas said the agency moved to deliver meaningful treatments faster for a historically hard-to-treat cancer.

A randomized, open-label trial of 500 adults with previously treated metastatic disease found median overall survival of 13.2 months on Rasonque versus 6.7 months on standard chemotherapy. Progression-free survival was also longer, and the objective response rate was higher in the pill arm, according to FDA summaries covered by Fox News and Reuters.

Angelo de Claro, M.D., director of the FDA Oncology Center of Excellence, called the results unprecedented in an area of high unmet need. The agency approved the drug about 6.5 months before its user-fee deadline and had granted Breakthrough Therapy, Orphan Drug and Priority Review designations. An expanded-access path was already open earlier this year.

The National Cancer Institute figures cited by the FDA put new U.S. pancreatic cancer cases near 67,000 a year, with adenocarcinoma accounting for roughly 90% to 95% of them. The disease causes a disproportionate share of cancer deaths because it is often found late.

Recommended dosing is 300 mg by mouth once daily until progression or unacceptable toxicity. Common side effects listed by the FDA include rash, diarrhea, mouth inflammation, nausea, fatigue, vomiting, abdominal pain, swelling, decreased appetite and bleeding. Revolution said the U.S. list price is about $39,800 for a 30-day supply before insurance and patient-assistance programs.

Doctors quoted in coverage stressed that the pill is not a cure, but that longer survival and a daily oral option change the conversation for many second-line patients.