UroGen Pharma Receives U.S. FDA Expedited Approval for Jelmyto™, the First and Only Non-Surgical Treatment for Patients with Low-Grade Upper Tract Urothelial Cancer
- Approval Based on Phase 3 Trial Results Showing a Complete Response Rate of 58%
- Median Duration of Response Has Not Been Reached
Therapy Provides an Effective, Kidney-Sparing Option for Patients With This Rare and Difficult-To-
- First-in-Class Approval Validates UroGen’s Innovative Technology and Future Opportunity Across its Specialty Cancers and Urologic Diseases Portfolio
Company to Host Conference Call on
Thursday, April 16at 8:30 AM Eastern Time
Jelmyto consists of mitomycin, an established chemotherapy, and sterile hydrogel, using UroGen’s proprietary sustained release RTGel™ technology. It has been designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means.
“UroGen was founded on the vision to improve lives by challenging the current standard of care. Jelmyto, which leverages our innovative technology and expertise in specialty cancers and urologic diseases, is just the beginning as we build a company focused on bringing novel solutions to patients,” said
LG UTUC is a rare cancer that develops in the lining of the upper urinary tract, ureters and kidneys. In the
“Jelmyto offers a new, non-surgical treatment approach for patients who otherwise may require treatment by radical nephroureterectomy, which is associated with declining kidney function and other complications,” said Dr.
The FDA approval is based on results from the Phase 3 OLYMPUS trial showing Jelmyto achieved clinically significant disease eradication in adults with LG UTUC.Findings include:
- Complete response (CR) (primary endpoint) of 58% in the intent-to-treat population and in the sub-population of patients who were deemed not capable of surgical removal at diagnosis.
- At the 12-month time point for assessment of durability, 19 patients remained in CR, seven had experienced recurrence of disease and nine patients continued to be followed for the 12-month duration of response.
- Kaplan-Meier analysis estimated 12-month durability at 84%2 (based on interim data).
- The most commonly reported adverse events (≥ 20%) were ureteric obstruction, flank pain, urinary tract infection, hematuria, renal dysfunction, fatigue, nausea, abdominal pain, dysuria, and vomiting. Most adverse events were mild to moderate and manageable using well established treatments. No treatment-related deaths occurred.
“There has been little progress for decades in the treatment of low-grade upper tract urothelial cancer, and this new option is paradigm-shifting for patients who often face recurrence and major surgery to remove their kidney,” said
UroGen is committed to helping patients access Jelmyto. UroGen Support may help identify appropriate financial assistance programs for patients with commercial, Medicare or Medicaid coverage, as well as those with no insurance coverage. These programs are for eligible patients who have been prescribed Jelmyto and who need help managing the cost of treatment. The appropriate program will depend on the patient’s coverage. Visit http://www.Jelmyto.com or contact UroGen Support at 855-JELMYTO for additional information.
The FDA evaluated Jelmyto under Priority Review, which is reserved for medicines that may represent significant improvements in safety or efficacy in treating serious conditions. Jelmyto was also granted Breakthrough Therapy designation by the FDA, which was created to expedite the development and review of drugs developed for serious or life-threatening conditions with high unmet need.
Conference Call & Webcast Information
Members of UroGen’s management team will host a live conference call and webcast on
About the Phase 3 OLYMPUS Trial
OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) is an open-label, single-arm Phase 3 clinical trial of Jelmyto™ (mitomycin) for pyelocalyceal solution to evaluate the safety, tolerability and tumor ablative effect of Jelmyto in patients with low-grade upper tract urothelial cancer (UTUC). The trial enrolled 74 patients at clinical sites across the
About Jelmyto™ (mitomycin) for pyelocalyceal solution
Jelmyto™ (mitomycin) for pyelocalyceal solution is a drug formulation of mitomycin for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel™ technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, Jelmyto is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. Jelmyto is delivered to patients using standard ureteral catheters. The
IMPORTANT SAFETY INFORMATION
You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.
Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:
are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO.
Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose.
Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
- are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
- Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?
- Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
- You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
- JELMYTO is given to your kidney through a tube called a catheter.
- During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:
- JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
- To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
- Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:
- Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
- Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: side pain, urinary tract infection, blood in your urine, kidney problems, tiredness, nausea, stomach pain, trouble with urination, vomiting, low red blood cell count, frequent urination, itching, chills, and fever.
Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.
About Upper Tract Urothelial Cancer (UTUC)
Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the
UroGen is a biopharmaceutical company dedicated to building novel solutions that treat specialty cancers and urologic diseases because patients deserve better options. UroGen has developed RTGel™ reverse-thermal hydrogel, a proprietary sustained release, hydrogel-based platform technology that has the potential to improve therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. UroGen’s approved Jelmyto™ (mitomycin) for pyelocalyceal solution, and pipeline treatment UGN-102 (mitomycin) for intravesical solution are designed to ablate tumors by non-surgical means and to treat several forms of non-muscle invasive urothelial cancer, including low-grade upper tract urothelial cancer and low-grade non-muscle invasive bladder cancer, respectively. UroGen is headquartered in
COVID-19 Pandemic Potential Impact
UroGen continues to gather information in this very fluid and rapidly-evolving environment regarding the potential impact of the COVID-19 pandemic on our Company, however, we are not currently able to quantify or predict with any certainty the overall scope of impact on UroGen, or any resulting delays in the availability of Jelmyto™ (mitomycin) for pyelocalyceal solution. Our primary focus is on the health and well-being of patients, caregivers, and UroGen employees at this critical juncture.
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation: the potential of Jelmyto™ to transform the treatment of LG UTUC; the potential of UroGen’s proprietary RTGel™ technology platform to improve therapeutic profiles of existing drugs; the potential of UGN-102 for LG NMIBC; and the potential impact of the COVID-19 pandemic on UroGen’s business. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: the timing and success of clinical trials and potential safety and other complications thereof; the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to maintain regulatory approval; complications associated with achieving commercial readiness for the launch of a new product; the labeling for any approved product; the scope, progress and expansion of developing and commercializing UroGen’s product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; and UroGen’s ability to attract or retain key management, members of the board of directors and personnel; and any negative effects on UroGen’s business and product development plans caused by or associated with COVID-19. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Form 10-K filed with the
- Browne BM, Stensland KD, Moynihan MJ, Canes D. An Analysis of Staging and Treatment Trends for Upper Tract Urothelial Carcinoma in the National Cancer Database. Clin Genitourin Cancer 2018;16:e743-e50.
Lerner, Seth. Primary Chemoablation for the treatment of Low-Grade Upper Tract Urothelial Carcinoma: The Olympus Trial. 2020 by
American Urological Association Education and Research, Inc.
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