Jelmyto

Indications

Jelmyto is specifically indicated for the treatment of low-grade upper tract urothelial cancer. This condition involves the growth of abnormal cells in the lining of the upper urinary system, which includes the kidneys and the ureters.

Targeted Treatment

This medication is used when the cancer is classified as low-grade, meaning the cancer cells appear more like normal cells and typically grow and spread more slowly than high-grade cancer cells. It is prescribed for patients who have tumors located in the:

• Renal pelvis: The part of the kidney where urine collects before it enters the ureter.
• Ureter: The tube that transports urine from the kidney to the bladder.

Jelmyto is designed to be administered directly into the upper urinary tract by a healthcare professional. The medication is formulated as a gel that allows the active ingredient to remain in contact with the lining of the urinary tract for an extended period, targeting the cancer cells directly at the site of the tumor.

Mechanism of Action

Jelmyto contains mitomycin, a type of chemotherapy known as an alkylating agent. It works by binding to the DNA inside cancer cells, a process called cross-linking. This damage prevents the cancer cells from copying their genetic material, which stops them from dividing and growing. Eventually, these damaged cells die off.

Reverse-Thermal Gel Technology

This medication uses a unique delivery system called reverse-thermal gel technology. This technology changes the physical state of the medicine based on temperature:

• Liquid state: The medication is a liquid when it is chilled, which allows a healthcare provider to instill it into the upper urinary tract through a catheter.
• Gel state: Once the medication reaches body temperature inside the kidney or ureter, it turns into a semi-solid gel.
• Prolonged exposure: The gel formulation allows the mitomycin to stay in contact with the lining of the urinary tract for a longer period than a standard liquid would. As urine flows through the area, the gel slowly dissolves and is eventually washed out of the body.

Common Side Effects

Many people receiving Jelmyto experience side effects related to the urinary tract or general physical well-being. Common side effects include:

• Pain in the side and back (flank pain)
• Urinary tract infections
• Blood in the urine
• Nausea and vomiting
• Fatigue or unusual tiredness
• Painful or frequent urination
• Abdominal pain

Serious Side Effects

Some side effects can be severe and may require medical intervention. Contact a healthcare provider immediately if you experience:

• Ureteral obstruction: Narrowing or blockage of the tube that carries urine from the kidney to the bladder. This may require the placement of a stent or surgery to correct.
• Kidney problems: Changes in kidney function or signs of kidney failure.
• Bone marrow suppression: Low levels of white blood cells, red blood cells, or platelets, which can increase the risk of infection, anemia, or easy bruising and bleeding.
• Severe infection: Signs of a systemic infection or sepsis.

This is not a complete list of side effects. Patients should discuss all potential risks with their doctor before starting treatment.

Ureteric Obstruction

Jelmyto can cause narrowing or blockage of the ureter, the tube that carries urine from the kidney to the bladder. This condition, known as ureteric obstruction, may be serious and might require the placement of a temporary stent or other medical procedures. Healthcare providers typically monitor kidney function and check for signs of blockage throughout the course of treatment.

Bone Marrow Suppression

This medication can lead to a decrease in the number of blood cells produced by the bone marrow. This may result in:

• Increased risk of infection: Due to low white blood cell counts.
• Bleeding or bruising: Due to low platelet counts.
• Fatigue: Due to low red blood cell counts (anemia).

Blood tests are usually performed before each dose to monitor these levels. Treatment may be delayed or adjusted if blood counts become too low.

Embryo-Fetal Toxicity

Jelmyto can cause harm to an unborn baby. It is not recommended for use during pregnancy. The following precautions are generally advised:

• Females who can become pregnant should use effective birth control during treatment and for 6 months after the last dose.
• Males with female partners who can become pregnant should use effective birth control during treatment and for 3 months after the last dose.

Contraindications

Jelmyto should not be used in patients who have a perforation (a hole or tear) in the bladder or the upper urinary tract.

Pregnancy and Contraception

Jelmyto can cause fetal harm when administered to a pregnant person. It is important to inform a healthcare provider if you are pregnant or planning to become pregnant before starting this treatment. Healthcare providers typically confirm pregnancy status with a test before beginning Jelmyto therapy.

Due to the risk of birth defects or pregnancy loss, the following contraception guidelines are recommended:

• Females: Use effective birth control during treatment and for at least 6 months after the final dose.
• Males: If you have a female partner who can become pregnant, use effective birth control during treatment and for at least 3 months after the final dose.

Breastfeeding

It is currently unknown if the medication in Jelmyto passes into human breast milk or how it might affect a nursing infant. Because of the potential for serious adverse reactions in a breastfed child, it is recommended that individuals do not breastfeed during treatment and for at least one week after the last dose.

Fertility

Based on findings from animal studies, Jelmyto may have an impact on fertility in both males and females. If you have concerns about future fertility, discuss these with your healthcare provider before starting treatment.

Available Forms

Jelmyto is supplied as a kit for pyelocalyceal use. The kit contains two components that must be mixed before administration:

• A vial of mitomycin powder.
• A vial of sterile hydrogel.

Once prepared, the medication is a chilled gel. This formulation is liquid when cold and becomes a semi-solid gel at body temperature. It is administered by a healthcare provider through a ureteral catheter or a nephrostomy tube.

FDA Approval History

The U.S. Food and Drug Administration (FDA) approved Jelmyto (mitomycin) for pyelocalyceal use on April 15, 2020. This approval marked the first time a drug was specifically authorized to treat low-grade upper tract urothelial cancer (LG-UTUC).

Approved Indications

Jelmyto is indicated for the treatment of adult patients with low-grade upper tract urothelial cancer. It is a formulation of mitomycin, an alkylating drug, delivered as a gel that is administered directly into the urinary tract by a healthcare professional.

Regulatory Background

The FDA granted Jelmyto several designations during its development to expedite the review process, including:

• Fast Track designation
• Orphan Drug designation
• Breakthrough Therapy designation
• Priority Review

The approval was based on results from the OLYMPUS clinical trial, which evaluated the drug's effectiveness in achieving a complete response in patients with LG-UTUC. Because it was approved under the priority review pathway, the FDA determined that the drug had the potential to provide a significant improvement in the safety or effectiveness of the treatment of a serious condition.

Administration and Billing

Jelmyto is a specialized medication administered by a healthcare professional in a clinical or hospital setting. Because it is not a drug that patients fill at a retail pharmacy, the total cost is typically billed through a medical benefit rather than a standard prescription drug plan. The overall expense usually includes:

• The cost of the mitomycin gel itself.
• Professional fees for the healthcare provider performing the administration.
• Facility fees for the hospital or outpatient center where the procedure takes place.

Insurance Coverage

Most health insurance plans, including Medicare, may provide coverage for Jelmyto when it is used for its FDA-approved indication. However, the specific out-of-pocket costs, such as deductibles, coinsurance, or copayments, will vary significantly based on the individual's insurance policy and the setting of care. Patients are encouraged to contact their insurance provider to confirm coverage details and any prior authorization requirements before beginning treatment.