The US Food and Drug Administration on Wednesday approved a new once-daily pill for certain patients with advanced pancreatic cancer after a major clinical trial found that patients receiving the treatment had a median survival of 13.2 months, nearly twice the 6.7 months recorded among patients receiving standard chemotherapy.
The drug, Rasonque, is manufactured by California-based Revolution Medicines and is designed to treat the most common form of pancreatic cancer. Taken once a day, the medication inhibits several forms of the RAS protein, which plays a central role in driving the growth of pancreatic tumors.
Announcing the decision, the FDA described Rasonque as a “new treatment option for patients with advanced pancreatic cancer.” The agency granted approval more than six months before the deadline originally set for completing its review.
“Today’s approval provides a critical new option for patients facing an extraordinarily difficult and historically hard-to-treat cancer. It is our fundamental duty to deliver more cures and meaningful treatments to patients as quickly as possible,” acting FDA Commissioner Kyle Diamantas said in a press release.
“I am immensely proud of the dedicated FDA scientists whose fast, thorough review and relentless commitment made this groundbreaking milestone a reality.”
The approval applies to adults with metastatic pancreatic cancer whose disease has failed to respond to at least one previous treatment, as well as patients who are unable to receive certain combinations of cancer medications.
Pancreatic adenocarcinoma is among the deadliest cancers in the United States and accounts for as much as 95% of the approximately 67,000 pancreatic cancer cases diagnosed annually, according to National Cancer Institute figures.
Although pancreatic cancer represents only about 3.2% of all cancer diagnoses, it is responsible for a disproportionately large share of cancer deaths. The disease is frequently discovered only after it has reached an advanced stage and is known for its ability to spread rapidly.
The FDA’s approval was based on results from a randomized, open-label study involving 500 patients with metastatic pancreatic cancer who had already received treatment. Median overall survival among those who received Rasonque reached 13.2 months, compared with 6.7 months among patients in the standard chemotherapy control group.
“This drug showed unprecedented results in an area of high unmet need,” said Angelo de Claro, M.D., director of the FDA’s Oncology Center of Excellence.
Rasonque reached approval through several expedited regulatory pathways. The FDA granted the treatment priority review, orphan drug status and breakthrough therapy designation, allowing the agency to accelerate its evaluation of the medication.
The application was also reviewed through the FDA commissioner’s National Priority Voucher pilot program, an initiative intended to speed the availability of treatments considered important to public health.
Even before Wednesday’s formal approval, some patients had been given access to Rasonque. In May, the FDA issued a “safe to proceed” letter permitting an expanded-access protocol through which eligible patients could receive the drug while its final review remained underway.
Clinical-trial safety data identified the most commonly reported side effects as rash, diarrhea, mouth sores, nausea, fatigue, vomiting, abdominal pain, fluid retention, loss of appetite and bleeding.
“Today’s landmark approval is the most significant advance we have seen in the fight against pancreatic cancer, a devastating disease,” said Anna Berkenblit, M.D., chief scientific and medical officer of the Pancreatic Cancer Action Network (PanCAN).
“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.”
{Matzav.com}
