FDA approves ImmunityBio's bladder cancer treatment

A sign outside the Food and Drug Administration's offices.

Published April 22, 2024 Updated April 23, 2024 8:27 PM PT

The Food and Drug Administration has approved a new treatment for patients with bladder cancer that does not respond well to standard therapy.

The treatment, which will be sold under the brand name Anktiva, is an immunotherapy agent designed to prompt the body’s immune system to step up its fight against cancer cells in the bladder. In a clinical trial led by UCLA, patients who responded to the treatment were much less likely to require surgery to remove all or part of their bladders than were patients who weren’t helped by the drug.

The drug was approved Monday and is being developed by ImmunityBio of Culver City after its initial development by Altor BioScience of Miramar, Fla. ImmunityBio designed and sponsored the clinical trial that led to the FDA’s action.

Dr. Patrick Soon-Shiong, whose family owns the Los Angeles Times, is executive chairman of ImmunityBio.

In a statement, Soon-Shiong heralded the FDA action and called Anktiva “a next-generation immunotherapy.”

The drug itself is designed to mimic a type of cell that causes the immune system to churn out so-called memory killer T cells that recognize and attack bladder cancer cells.

Last May, the FDA had declined to approve the new therapy because of deficiencies related to a pre-license inspection of third-party manufacturing facilities, according to a Securities and Exchange Commission filing. The FDA provided recommendations on how to resolve the issues, according to the filing.

The National Cancer Institute estimates that 83,190 people in the United States will be diagnosed with bladder cancer this year, and that 16,840 will die of the disease. Those figures account for 4.2% of all new cancer cases and 2.8% of cancer deaths.

Most diagnoses are in people age 65 and older, while men are up to four times as likely as women to get the disease, according to the University of Texas MD Anderson Cancer Center.

The first-line treatment for bladder cancer is a surgical procedure to remove visible tumors from the tissue that lines the inner surface of the bladder, according to the American Cancer Society. If the tumors haven’t spread to the wall of the bladder, doctors also use a catheter to administer a bacterial drug called Bacille Calmette-Guerin that revs up the immune system to fight the cancer.

However, about 20% of bladder cancer patients do not respond to BCG, and the usual course of action for them is to remove the bladder altogether, said Dr. Kyle Richard, a urologic oncologist and associate professor at the University of Wisconsin-Madison.

It’s also common for tumors to return. If the cancer invades the muscle of the bladder wall, “then you’re looking at surgery, which is a really, really brutal operation,” said Mark Story, a spokesman for the Bladder Cancer Advocacy Network, a nonprofit that supports patients with the disease.

In an attempt to head off that surgery, doctors may consider another immunotherapy treatment. Current options include Keytruda, which is administered through an IV, and Adstiladrin, which is delivered directly to the bladder like BCG, the American Cancer Society says. Anktiva will represent a third choice.

Treatments such as Anktiva should give patients hope that “they can spare or save their bladder without their cancer progressing,” said Richard, who was not involved in the drug’s development.

Anktiva goes straight to the bladder and is meant to be used in conjunction with BCG, the FDA said.

In the clinical trial, patients who were treated with Anktiva along with BCG fared much better than a smaller group of patients who received Anktiva alone.

Out of 10 patients in the latter group, the treatment appeared to have worked for two of them after three months, and one was able to stay that way for six months. Meanwhile, among 82 patients who got both drugs, 55% had a successful response at three months, as did 56% at the six-month mark and 45% after a full year.

In one group of 72 patients with a certain type of bladder cancer that received both Anktiva and BCG, 55.4% of the patients had a successful response at 12 months. By 24 months, that had slipped to 48%. Overall, the median length of time that patients remained cancer-free after treatment was 19.3 months, investigators reported in the journal NEJM Evidence. (Soon-Shiong was the senior author.)

The most common side effects seen during the trial were pain or discomfort while urinating, having to urinate frequently, and finding blood in the urine. One patient in the trial died of a cardiac arrest.

Dr. Karim Chamie, an associate professor of urology at UCLA’s David Geffen School of Medicine, who led the trial, said in a statement that Anktiva offers “a compelling alternative for patients who have exhausted conventional treatment options.”

The treatment provided “a long-lasting response,” Chamie added. “Overall, the treatment was safer and more effective than other options” for patients who didn’t respond to BCG.

Dr. Ashish Kamat, an endowed professor of urologic oncology and cancer research at MD Anderson, who was not involved in the trial, said the development of a new immunotherapy that builds on “knowledge and experience with BCG as an immune stimulant is exciting to see.”

However, Richard noted that there’s a worldwide shortage of BCG, which “could limit the number of patients that could receive” Anktiva.

“We also need more research on patient-reported outcomes and quality of life with this new agent,” he added.

Anktiva is expected to be available by the middle of next month, according to ImmunityBio. No price was announced, but the research firm Evaluate has estimated that the drug would rack up $878 million in annual sales by 2028, according to a report in Fierce Pharma.

Shares of ImmunityBio rose 55 cents to $5.49 in Nasdaq trading Tuesday, a gain of 11%.