First Treatment for Upper Tract Urothelial Cancer Approved by FDA

The US Food and Drug Administration (FDA) has approved JelmytoTM (mitomycin gel) as the first therapy for treatment of low-grade upper tract urothelial cancer (UTUC).1-2

What is low-grade upper tract urothelial cancer (UTUC)?

Urothelial cancer is a cancer that develops in the lining of the urinary system, mostly commonly the bladder.1 UTUC is a type of urothelial cancer which actually grows in the lining of the kidney or the ureter. The ureter is the long, thin tube that connects that kidney to the bladder. When UTUC grows, it can block the ureter or kidney. This can cause swelling, infection, and interfere with normal kidney function.1-2

How common is UTUC?

Low-grade UTUC is rare. There are around 6,000-8,000 new cases in the US each year.1 80,000 people develop bladder cancer each year in the United States. Less than 10 percent of all cases are UTUC.1 UTUC low-grade tumors are not invasive. They rarely spread, but they do grow back.

Why is this treatment important for people with UTUC?

Jelmyto is a first of its kind, non-surgical approach for treating people with low-grade UTUC. Previously, people with UTUC would usually have to undergo a surgery called a nephroureterectomy. A nephroureterectomy is a radical surgery to remove the diseased kidney, ureter, and bladder cuff. Early clinical results using Jelmyto suggest this treatment may help some people avoid a nephroureterectomy.1

How the FDA designated Jelmyto

The FDA has specific categories or “designations” that it uses to help speed up the availability of treatments for serious diseases like bladder cancer. For Jelmyto, the FDA issued the following designations:1-3

  • Priority Review– This means that the FDA will take action on a new drug application within 6 months
  • Breakthrough Therapy– This also means the application will go through a faster review process, but it is designed specifically for drugs that may be substantially better than what is currently available based on early clinical study results
  • Fast Track– This is also a faster review process, but it is designed to support development of drugs needed to fill an unmet medical need for serious conditions (like bladder cancer)
  • Orphan Drug – This is an FDA incentive to foster the development of drugs for rare diseases (like UTUC)

People with cancer remain a priority for the FDA

Approval of Jelmyto is an important step in continuing product development to fight cancer. “Although our nation’s emphasis is on the need to combat COVID-19, patients with cancer and their unique needs continue to be a top priority for the FDA,” said Dr. Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. 1

How does Jelmyto work?

Jelmyto is a gel formulation of the chemotherapy drug mitomycin.2 It is an alternative treatment option for low-grade UTUC.1 It interferes with how proteins are made, and it stops cancer cells from multiplying.1 This represents a significant new non-surgical alternative for low-grade UTUC. 1-2

Clinical trial findings

A Phase III clinical trial enrolled 71 people with low-grade UTUC. To be eligible, the participants had to either: 1

  • Never had any treatment
  • Have a recurring condition with at least one tumor with its stem attached to the inner lining of an organ

Jelmyto was given once a week for 6 weeks using sustained-release RTGel™ technology. This technology allows for the mitomycin treatment to be in contact with the affected area for a longer time.2 For people who achieved a complete response (no sign of a tumor), the drug was continued monthly for up to 11 more months.1-2

There was a 58% complete response rate in the 71 people who were treated after receiving weekly treatment with Jelmyto for 6 weeks. A complete response means having negative results: no evidence of cancer cells in any screening tests. After a year, 19 people (46%) who were tumor-free at 3 months were still responding to treatment.1-2

What are the possible side effects of Jelmyto?

Some people in the clinical trial experienced side effects. The most common side effects were: 1-2

  • A back-up of urine
  • Pain on the side of the body
  • Urinary tract infection
  • Blood in the urine
  • Fatigue
  • Nausea
  • Stomach pain
  • Painful urination

More serious side effects include obstruction of the ureter, pain, and bacteria that enter the bloodstream from a urinary tract infection. Some ureter obstructions may require stents to relieve the obstruction. 1

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This article represents the opinions, thoughts, and experiences of the author; none of this content has been paid for by any advertiser. The team does not recommend or endorse any products or treatments discussed herein. Learn more about how we maintain editorial integrity here.

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