Bladder Cancer Drug That Received Accelerated Approval Shows Even Stronger Findings in Later Study

According to a publication from Medscape, the first ever drug targeted for bladder cancer has shown even more impressive results in post-approval study. Erdafitinib was approved on an accelerated basis by the Food and Drug Administration back in April. Accelerated approval is a system used by the FDA to bring promising drugs with no existing alternative to market faster than they would otherwise.

Though accelerated approvals can result in the marketing of drugs that are later found to be ineffective, in this case, it seems to have worked just as intended.

About Bladder Cancer

Bladder cancer, while technically a rare disease, is one of the most common cancers in the United States — affecting an estimated 68,000 adults each year.

It mostly occurs in older men, though it can occur in both men or women, and at any age. Typically, the cancer begins in the urothelial cells that line the inside of the bladder — however, it can occur almost anywhere along the urinary tract since urothelial cells form the lining of much of the system.

Because the symptoms of bladder cancer are uncomfortable (such as pain during urination, pelvic pain, and blood in the urine), many cases (about 70%) are diagnosed early — when the cancer is at its most treatable. Prognosis highly dependent on the location and stage of the cancer, though five, ten, and fifteen-year survival rates are all fairly robust (77%, 70%, and 65%, respectively). However, even early-stage bladder cancers may be recurring, meaning follow-up examination is required to ensure long-term health and survival.

About Accelerated Approval and Erdafitinib

Accelerated approval is offered by the FDA on a circumstantial basis to bring drugs to market without the usual degree of study. Drugs approved on this basis often treat dangerous illnesses with few or no alternate treatments. The general thinking is that these illnesses are more dangerous than the potential side-effects of the drug in question, so the degree of certainty of a drug’s effectiveness is of lesser concern.

The results can be mixed. Some drugs approved on an accelerated basis are later found to offer little, if any, clinical benefit — these drugs subsequently lose their market approval, though not necessarily without netting millions before getting pulled. However, other, actually effective drugs might receive approval years before they would otherwise.

When erdafitinib first received approval, it yielded a 32.3% overall response rate (ORR). The full results of the phase 2 study, however, reveal an overall response rate of closer to 40%. In the subgroup of participants who had received prior immunotherapy, the ORR was almost 60% (59%).

Checkpoint inhibitors, a type of cancer immunotherapy, yield an ORR of only around 20%. Though erdafitinib is designed for use primarily in patients who have previously received immunotherapy, future studies will likely be conducted to test the efficacy of the drug in treatment-naive patients.


What do you think of drugs that receive accelerated approval? Do you think the program is good or bad for the patient community? Share your thoughts with Patient Worthy!

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