FDA Approves New Non-Muscle Invasive Bladder Cancer Treatment

For the first time in 20 years, the US Food and Drug Administration (FDA) has approved a new treatment for non-muscle invasive bladder cancer (NMIBC). The drug pembrolizumab is marketed as Keytruda and manufactured by Merck.^1-4 It is an immunotherapy drug, which means that works with your immune system to help fight certain cancers.

A new way to treat non-invasive bladder cancer

80,000 people develop bladder cancer each year in the United States. 75 percent of all cases are non-muscle invasive.² Keytruda (pembrolizumab) is a new way to treat patients with high-risk, non-muscle invasive bladder cancer that is Bacillus Calmette-Guerin (BCG)-unresponsive.² BCG is another bladder cancer treatment, and it is currently considered the "gold-standard” treatment for non-invasive bladder cancer. However, BCG doesn’t work for everyone, so pembrolizumab is a new option for those patients.

How does immunotherapy work?

Immunotherapy is a way to help the body’s immune system find and fight tumor cells.³ This new treatment offers an option that may be able to keep your bladder working as it should, all while fighting cancer. The hope is that this new therapy could lead to new combinations of drugs used to treat this aggressive bladder cancer that has not spread. Continued advances will hopefully create new options for others with bladder cancer.²

A new way to use an existing cancer drug

In 2014, Keytruda received its first FDA approval for the treatment of melanoma, a type of skin cancer. Since then, it has been studied in many clinical trials for use in the treatment of many different kinds of cancer.⁴

Pembrolizumab is used for the treatment of people with BCG-unresponsive, high-risk, non-muscle invasive bladder cancer, meaning bladder cancer that has not grown into the bladder wall nor spread outside of the bladder. It is also an option for those who cannot have or don’t want to have their bladder removed.^1,2

Pembrolizumab was shown to be very good at reducing the number of tumors in a person’s bladder. It was also shown to help people stay tumor-free for a long time. It might need more clinical trials in the future to learn more about the drug.

Drug trial findings

The clinical trial that studied the use of pembrolizumab took place in New York at the NYU Langone Health's Perlmutter Cancer Center.² 148 patients with high-risk non-muscle-invasive bladder cancer participated in the clinical trial. 96 of those participants had bladder cancer that did not respond (or improve) to BCG. After being given pembrolizumab, 4 in 10 of those 96 patients had a complete response to the treatment, meaning there was no evidence of cancer in any of their tests. 2 in 10 remained cancer-free after 2 years.

What are the possible side effects?

Some people in the drug had side effects. The most common ones were:^1,2

• Fatigue
• Diarrhea
• Rash
• Itching
• Muscle and joint pain
• Cough
• Nausea
• Constipation
• Blood in the urine
• Urinary tract infection
• Swelling
• Cold

BCG alternatives due to a shortage

BCG is a common treatment for high-risk, non-muscle invasive bladder cancer. Unfortunately, there have been major BCG supply shortages over the last 10 years, including the most recent shortage in 2019 that has continued into 2020. Due to the shortage, not all patients will be able to get BCG. The approval of Keytruda (pembrolizumab) for BCG-unresponsive non-muscle invasive bladder cancer is a big step forward. Future research could lead to the use of pembrolizumab as a BCG alternative for those who are unable to receive BCG due to the shortage.

The full prescribing information for pembrolizumab can be found here.