BS BULLETIN:
- The FDA has approved Rasonque, the first therapy to directly target RAS in the most common form of pancreatic cancer.
- In a 500-patient trial, median survival jumped from 6.7 months with standard chemotherapy to 13.2 months with the once-daily pill.
- The FDA approved the drug roughly 6½ months ahead of its deadline, calling the results “unprecedented.”
Every once in a while, some genuinely great news comes across the wire.
This qualifies.
The FDA on Wednesday approved a new once-a-day pill for patients battling metastatic pancreatic cancer — one of the most aggressive and difficult cancers doctors face.
And the results behind it are remarkable.
Patients receiving Rasonque, also known as daraxonrasib, survived a median 13.2 months in a 500-patient clinical trial.
Patients receiving standard chemotherapy survived 6.7 months.
That’s nearly double.
“This drug showed unprecedented results in an area of high unmet need,” Dr. Angelo de Claro, director of the FDA’s Oncology Center of Excellence, said in the agency’s announcement.
Rasonque attacks something scientists have struggled for decades to target effectively: RAS, a family of proteins that can drive cancer growth.
RAS mutations are present in the overwhelming majority of pancreatic adenocarcinomas. The new drug blocks active RAS signaling and is the first RAS-targeted therapy approved for pancreatic cancer.
And survival wasn’t the only improvement.
Patients taking Rasonque had median progression-free survival of 7.2 months versus 3.6 months with chemotherapy. Tumors responded to treatment in 30% of Rasonque patients, compared with 11% receiving standard chemotherapy.
UCLA, which co-led the international phase-three study, said the drug reduced the risk of death by 60% compared with standard chemotherapy.
That’s a big deal for a disease where big victories have been painfully difficult to come by.
About 67,000 Americans are diagnosed with pancreatic cancer annually, and roughly 90% to 95% of those cases are pancreatic adenocarcinoma. Because the disease is frequently discovered late and spreads aggressively, treatment options have historically been limited.
This isn’t a cure, and the approval isn’t for every pancreatic cancer patient. It’s specifically for adults with metastatic pancreatic adenocarcinoma who have already received at least one systemic treatment or aren’t candidates for multi-drug systemic therapy.
There are also potentially serious side effects, including gastrointestinal problems, lung inflammation and skin and soft-tissue toxicity.
But the results were strong enough that regulators didn’t make patients wait.
The FDA gave Rasonque Priority Review, Breakthrough Therapy and Orphan Drug designations and ultimately approved it approximately six and a half months ahead of its target date.
DBS WIRE SOURCES
- FDA approves once-daily pill for deadly cancer after patients’ survival rates nearly double — Fox News
- FDA Approves First in Class Targeted Therapy for Metastatic Pancreatic Cancer — FDA
- FDA approves daraxonrasib for metastatic pancreatic adenocarcinoma — FDA
