The Food and Drug Administration has approved a new targeted treatment for advanced pancreatic cancer after a clinical trial found that patients receiving the drug survived about twice as long as those receiving chemotherapy.
Rasonque, also known as daraxonrasib, was developed by Revolution Medicines. The daily pill is approved for patients with advanced pancreatic cancer who have already received another treatment, such as chemotherapy.
In a late-stage trial involving 500 patients, those treated with Rasonque lived an average of 13.2 months. Cancer specialists described the results as unprecedented for a disease that remains among the deadliest and most difficult cancers to treat.
Rasonque targets mutations in the KRAS gene, which are found in nearly all pancreatic cancers and also occur in some lung, colorectal, ovarian, and other cancers.
The KRAS gene normally helps regulate cell growth by acting like an on-and-off switch. A mutation can leave that switch permanently activated, allowing cancer cells to grow uncontrollably. Scientists have spent years attempting to develop treatments capable of effectively targeting the mutation.
More than 67,000 Americans are expected to be diagnosed with pancreatic cancer this year, according to the American Cancer Society. Pancreatic cancer is the country’s third-leading cause of cancer death.
The disease is particularly dangerous because there is no routine screening test and its early symptoms can be vague. Many patients are not diagnosed until their cancer has reached an advanced stage, when treatment is more difficult. The five-year survival rate for pancreatic adenocarcinoma, the most common form of the disease, is about 8%.
Rasonque can cause significant side effects, including blistering rashes, sores in the mouth and throat, vomiting and diarrhea. Patients and physicians will need to weigh those risks against its potential benefits.
More than 2,000 patients have already received the drug through an expanded-access program that Revolution Medicines began in May.
The company said the list price before insurance coverage, discounts or financial assistance will be $39,800 for a one-month supply. Actual patient costs will vary depending on insurance and assistance programs.
Researchers believe the approval could also accelerate development of additional treatments targeting KRAS mutations across several types of cancer.
Source: NBC