FDA approves oral pancreatic cancer drug that extends survival
Treatment, aimed at those whose cancer has spread, could 'transform' care
Written by |
The U.S. Food and Drug Administration (FDA) has approved an oral drug for adults with pancreatic adenocarcinoma, the most common form of pancreatic cancer, whose disease has spread to other parts of the body.
Revolution Medicines Rasonque (daraxonrasib) is indicated for use in patients who have received at least one prior line of systemic treatment or who are not candidates for systemic therapy with multiple medications. Trial results showed that it extended both the time patients lived before their disease worsened and overall survival time.
“The FDA approval of RASONQUE is a monumental step forward for patients with pancreatic cancer and for the oncology field,” Mark A. Goldsmith, MD, PhD, chairman and CEO of Revolution Medicines, said in a company press release. “For the first time, patients have an approved targeted medicine designed to directly inhibit the main cause of pancreatic cancer. We continue to engage with global regulatory authorities with the goal of expanding and accelerating the reach of RASONQUE.”
The therapy was reviewed under the Commissioner’s National Priority Voucher pilot program, which offers an expedited evaluation process for therapies that aim to tackle public health priorities. According to the FDA, the approval was granted months ahead of the scheduled date for the regulatory decision.
“This drug showed unprecedented results in an area of high unmet need,” Angelo de Claro, MD, director of the FDA’s Oncology Center of Excellence, said in a press release from the FDA. “The approval was granted 6.5 months before the user fee deadline, demonstrating the FDA’s commitment to accelerating the approval of new cancer treatments for patients with serious and life-threatening conditions.”
‘More time with loved ones’
Physicians in the U.S. can now prescribe Rasonque, which comes as oral tablets to be taken once daily, with or without food. Revolution Medicines has also launched a support program, (ON)Path, meant to help patients navigate insurance, receive financial assistance, and learn about treatment.
“I believe this drug will transform how pancreatic cancer is treated, giving people the opportunity for more time with loved ones, the possibility of a better quality of life and optimism that continued research may lead to even greater advances,” said Anna Berkenblit, MD, chief scientific and medical officer of the Pancreatic Cancer Action Network (PanCAN).
Berkenblit said the fact that the drug comes in pill form can provide “a less burdensome treatment experience than standard intravenous chemotherapy.”
“PanCAN will continue to empower patients and caregivers with the resources and knowledge they need to advocate for the care they deserve now that RASONQUE is available for doctors to prescribe,” Berkenblit said.
The vast majority of pancreatic tumors harbor mutations in the RAS gene family, which encode signaling proteins that act as on/off switches to control cell growth. Rasonque works to block the active, or on state, of mutant and wild-type (normal) RAS proteins, thereby inhibiting tumor growth.
The FDA’s approval was based on data from a Phase 3 clinical trial called RASolute 302 (NCT06625320). The trial enrolled 500 adults with previously treated metastatic pancreatic adenocarcinoma who were randomly assigned to treatment with Rasonque or standard chemotherapy regimens.
Results showed that median overall survival nearly doubled with Rasonque relative to standard chemo (13.2 months vs. 6.7 months), corresponding to a 60% lower risk of death.
Rasonque also extended the time patients lived before their disease worsened when compared with chemo (median progression-free survival of 7.2 months vs. 3.6 months), representing a 51% lower risk of progression.
Rasonque also outperformed chemo in preserving quality of life and limiting pain.
Serious safety issues were also less common with Rasonque than with chemo. The most common side effects associated with Rasonque, per the therapy’s prescribing information, include rash, diarrhea, stomatitis (inflammation of the lining of the mouth), nausea, fatigue, vomiting, abdominal pain, swelling, decreased appetite, and hemorrhage.
“The unprecedented results of the global Phase 3 trial position RASONQUE to become the new standard of care for patients with metastatic pancreatic cancer under an approved label that supports real-world clinical decision-making,” Goldsmith said.
A few months ago, the FDA authorized an expanded access program that allowed some patients to access Rasonque outside of clinical trials before the therapy was formally approved. The FDA has also granted the therapy designations, including breakthrough therapy and orphan drug designations, aiming to speed the development and regulatory review of treatments for serious or rare diseases
“Today’s approval provides a critical new option for patients facing an extraordinarily difficult and historically hard-to-treat cancer,” said Kyle Diamantas, acting FDA commissioner. “It is our fundamental duty to deliver more cures and meaningful treatments to patients as quickly as possible. I am immensely proud of the dedicated FDA scientists whose fast, thorough review and relentless commitment made this groundbreaking milestone a reality.”
Leave a comment
Fill in the required fields to post. Your email address will not be published.