Advances in the treatments for bladder cancer have led to increased survival rates each year. Although surgery is often the main treatment for bladder cancer, it may not be effective for some people with advanced bladder cancer. This includes people with large tumors or metastatic bladder cancer (cancer that’s spread to other parts of the body).

Cancer researchers have been looking into new medicines to help people with advanced and metastatic bladder cancer, who often have fewer treatment options and worse outcomes. This article will cover some of the latest medications approved to treat bladder cancer.

1. Immune Checkpoint Inhibitors

Immune checkpoint inhibitors (ICIs) are a type of immunotherapy and have become important in treating several types of cancer. Immunotherapy uses medicines to boost the body’s immune system to identify and kill cancer cells.

As of December 2024, there are three immune checkpoint inhibitors approved by the U.S. Food and Drug Administration (FDA) to treat bladder cancer:

• Avelumab (Bavencio)
• Nivolumab (Opdivo)
• Pembrolizumab (Keytruda)

Avelumab and pembrolizumab were FDA-approved in 2020, and nivolumab was approved in 2024.

How Do Immune Checkpoint Inhibitors Work?

Immune checkpoint inhibitors work by activating the body’s immune cells to attack cancer cells.

Immune checkpoints are a natural part of the immune system, designed to regulate immune responses and prevent immune cells from attacking healthy tissues. Immune checkpoint proteins are found on immune cells called T cells and other cells in the body. When immune checkpoint proteins on a T cell bind with their counterparts on a normal cell, they send an “off” signal that prevents the T cell from attacking. Some cancer cells exploit this mechanism by producing checkpoint proteins themselves, effectively "hiding" from the immune system.

ICIs block immune proteins from binding and sending the off signal. This allows T cells to attack and kill cancer cells. The immune checkpoint inhibitors used for bladder cancer can target the immune checkpoint proteins on T cells or cancer cells. Avelumab targets an immune checkpoint protein called PD-L1 that’s found on cancer cells. Nivolumab and pembrolizumab target PD-1, which is found on T cells.

When Are Immune Checkpoint Inhibitors Used for Bladder Cancer?

Immune checkpoint inhibitors are a treatment option for some people with advanced, metastatic, or recurrent (cancer that comes back after treatment) bladder cancer.

Avelumab can be used as maintenance therapy after chemotherapy to prevent cancer from progressing in people whose condition has responded well to treatment. It can also be used as a second option to treat advanced or metastatic bladder cancer that progressed while using platinum-containing chemo (such as cisplatin).

Nivolumab is approved as an adjuvant treatment for people with muscle-invasive bladder cancer (MIBC) with a high risk of recurrence after a radical cystectomy (bladder removal surgery). It is also a first-line maintenance treatment for people with advanced or metastatic bladder cancer who have responded to initial chemotherapy with cisplatin and gemcitabine but whose cancer cannot be surgically removed.

Pembrolizumab may be used on its own to treat advanced or metastatic bladder cancer in people who haven’t responded to or can’t take platinum-containing chemo. It can be used in combination with other treatments, such as antibody-drug conjugates (ADCs), as first-line therapy options.

Pembrolizumab is the only immune checkpoint inhibitor that’s also approved to treat some types of non-muscle-invasive bladder cancer (NMIBC) in people who haven’t responded to intravesical immunotherapy with Bacillus Calmette–Guérin (Tice BCG).

2. Fibroblast Growth Factor Receptor Inhibitors

Fibroblast growth factor receptor (FGFR) inhibitors are a type of targeted therapy used for bladder cancer. Targeted therapies are drugs that target a specific protein or substance found in cancer cells to find and destroy them. Erdafitinib (Balversa) is an FGFR inhibitor for bladder cancer that was approved by the FDA in 2024.

How Do FGFR Inhibitors Work?

FGFRs are proteins that signal cancer cells to grow and multiply. Some cancer cells have a mutation (change) in the FGFR gene that controls how much FGFR proteins are made. FGFR inhibitors target and block cancer cells with mutations in the FGFR3 gene to help slow or stop the growth of cancer cells.

About 20 percent of people with bladder cancer have a mutation in the FGFR gene.

Erdafitinib is most effective in people with a mutation in the FGFR gene. About 20 percent of people with bladder cancer have this mutation. Your cancer care team will test your cancer cells for this genetic mutation to find out if erdafitinib will work for you.

When Are FGFR Inhibitors Used for Bladder Cancer?

Erdafitinib can be used to treat people with advanced or metastatic bladder cancer that hasn’t responded to at least one other type of treatment, such as chemotherapy or immunotherapy. This drug isn’t recommended for people who are eligible for immune checkpoint inhibitors but haven’t tried them yet.

To make sure erdafitinib will work well, cancer cells are tested for the FGFR3 mutation before starting this drug.

Unlike the other new treatments for bladder cancer, erdafitinib is given as a once-daily pill you swallow by mouth. This means you don’t have to go to a clinic or health care provider’s office regularly to receive this medication.

3. Antibody-Drug Conjugates

ADCs are made of a chemotherapy drug connected to a monoclonal antibody (laboratory-made immune protein). This type of treatment can be classified as a type of chemotherapy, immunotherapy, and targeted therapy.

Enfortumab vedotin (Padcev) was FDA-approved for bladder cancer in 2020.

How Do ADCs Work?

Antibody-drug conjugates combine a monoclonal antibody with a chemotherapy drug. The monoclonal antibody is designed to target specific proteins on cancer cells, delivering the chemotherapy directly to where it’s needed. In enfortumab vedotin, the antibody targets a protein called Nectin-4, which is commonly found on the surface of bladder cancer cells. Once the antibody binds to Nectin-4, the attached chemotherapy drug is activated to kill the cancer cells.

Delivering chemotherapy drugs directly to cancer cells can help reduce side effects, as fewer healthy cells are exposed to the toxic effects of the treatment.

When Are ADCs Used for Bladder Cancer?

Enfortumab vedotin is approved to treat adults with advanced or metastatic bladder cancer when it’s combined with pembrolizumab as first-line therapy regardless of cisplatin eligibility.

Enfortumab vedotin may also be used by itself in people who meet the following criteria:

• Have advanced or metastatic bladder cancer
• Have already been treated with an immune checkpoint inhibitor drug and chemotherapy with a platinum-containing drug (such as cisplatin)
• Can’t take a cisplatin-containing treatment and have already tried another type of treatment

Clinical Trials for Bladder Cancer

Before securing FDA approval, each of these drugs underwent extensive testing in large groups of people with bladder cancer. Clinical trials are studies that help cancer researchers learn whether new treatments are safe and effective. People with advanced, metastatic, or recurrent bladder cancer are encouraged to join a clinical trial where newer treatments are available.

Before securing FDA approval, each of these drugs underwent extensive testing in large groups of people with bladder cancer.

As cancer researchers learn more about what causes bladder cancer and how it spreads, new treatments may become available. There are several ongoing clinical trials for bladder cancer studying new treatments or new combinations of treatments. Talk to your cancer care team to learn more about which clinical trials you may be eligible to join. The Bladder Cancer Advocacy Network also has a list of featured clinical trials for bladder cancer that you can search.

Talk With Others Who Understand

MyBladderCancerTeam is the social network for people with bladder cancer and their loved ones. On MyBladderCancerTeam, members come together to ask questions, give advice, and share their stories with others who understand life with bladder cancer.

Have you tried any of these newer bladder cancer treatments? How did it work for you? Share your experience in the comments below, or start a conversation by posting on your Activities page.