Newly approved pancreatic cancer drug wins breakthrough therapy status
FDA designation backs med's use with chemo as first treatment in PDAC
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The U.S. Food and Drug Administration (FDA) has awarded its breakthrough therapy designation — which aims to speed the development of promising new medications — to the newly approved oral therapy Rasonque (daraxonrasib) for use as part of a chemotherapy regimen in people with pancreatic cancer who have not yet received any treatment.
This status backs the new drug’s use alongside chemo to treat pancreatic adenocarcinoma (PDAC), the most common form of this cancer type. Specifically, the FDA designation is for the use of Rasonque in combination with gemcitabine and nab-paclitaxel (GnP), a common chemo regimen, for people with previously untreated PDAC that is metastatic, meaning the cancer has spread beyond the pancreas to other organs.
The new designation comes just a few weeks after the FDA approved Rasonque as a monotherapy — one used on its own — for certain adults with PDAC who had previously received treatment. Among such patients, the drug extended both the time people lived before their disease worsened and overall survival.
The FDA grants breakthrough status to experimental therapies that aim to address unmet medical needs in the management of serious diseases and that have shown promise in early clinical testing. The Rasonque-chemo combo is now being assessed in a clinical trial involving more than 1,100 people with PDAC.
“This Breakthrough Therapy Designation underscores the significant unmet need among patients with previously untreated metastatic pancreatic adenocarcinoma and recognizes the importance of advancing new treatment options earlier in their treatment journey,” Alan Sandler, MD, chief development officer at Revolution Medicines, the therapy’s developer, said in a company press release announcing the award.
The FDA granted the designation based on data from an ongoing Phase 1/2 study (NCT06445062) that’s testing Rasonque plus GnP in people with various solid tumor types. That study is still enrolling at more than 30 sites across the U.S.
Early data from the study have shown that the combo demonstrated “encouraging preliminary antitumor activity and a manageable safety profile” in people with newly diagnosed metastatic PDAC, per Revolution.
Rasonque now being tested alongside chemo in large trial
According to Sandler, “data from the [Phase 1/2] study have now shown the therapeutic potential of Rasonque in combination with GnP, a commonly used multiagent systemic therapy, in treatment-naïve patients.”
Revolution is now running a Phase 3 trial called RASolute 303 (NCT07491445) to further test this regimen in adults with previously untreated PDAC. The study aims to enroll approximately 900 participants; recruitment is ongoing at sites worldwide.
Participants will be randomly assigned to treatment with Rasonque only, GnP only, or Rasonque in combination with GnP. The study’s main goal is to evaluate the effects of Rasonque on disease progression and survival outcomes.
Rasonque works by blocking the activity of RAS proteins, a group of proteins that drive cell growth and are often mutated in pancreatic cancer. The RASolute 303 study is open to patients irrespective of RAS mutation status.
Data from the [Phase 1/2] study have now shown the therapeutic potential of Rasonque in combination with GnP, a commonly used multiagent systemic therapy, in treatment-naïve patients.
Under its recent FDA approval, Rasonque is indicated to treat adults with metastatic PDAC who have received at least one prior line of systemic treatment or who are not candidates for systemic therapy with multiple medications.
Its approval was based on data from a prior Phase 3 study called RASolute 302 (NCT06625320) that involved 500 patients. In that trial, treatment with Rasonque nearly doubled overall survival compared with standard chemo used alone.
“Rasonque monotherapy demonstrated compelling clinical benefit in the RASolute 302 trial, leading to FDA approval for patients with previously treated metastatic pancreatic adenocarcinoma or those who are not candidates for multiagent systemic therapy,” Sandler said. “Together with our other ongoing studies, these findings reflect our deep commitment to advancing a broad [RAS-inhibiting] approach for patients with some of the most difficult-to-treat cancers.
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