UroGen Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has quickly approved Jelmyto (mitomycin, mitomycin) pyelonephr calyces gel preparation, which is a first-in-class drug, used For the treatment of adult low-grade upper urothelial carcinoma (LG-UTUC). Jelmyto was approved through a priority review process and was previously granted a Breakthrough Drug Qualification (BTD). It is worth mentioning that Jelmyto is the first and only non-surgical treatment option approved for the treatment of LG-UTUC.
Jelmyto consists of mitomycin (a chemotherapeutic drug) and a sterile gel, developed using UroGen's proprietary slow-release RTGel ™ technology. The drug is designed to expose urinary tract tissue to mitomycin for a long time, making it possible to treat tumors by non-surgical means.
This milestone approval is based on the positive results of the phase III OLYMPUS test. The trial showed that Jelmyto achieved clinically significant disease eradication in adult LG-UTUC patients: the complete response rate was as high as 58%, and the median duration of response (DOR) had not yet been reached. These data suggest that for a rare and difficult-to-treat cancer patient such as LG-UTUC, Jelmyto will provide an effective, kidney-retaining treatment option.
The results of this study include: (1) In the intention-to-treat population and the subgroup of people who were deemed inoperable at diagnosis, the complete response rate (CR, primary endpoint) was 58%. (2) At the 12-month time point for evaluating the durability of remission, 19 patients were still in CR status, 7 patients had disease recurrence, and 9 patients continued to follow up to evaluate 12-month remission data. (3) The sustained rate of 12-month remission assessed by Kaplan-Meier analysis was 84% (based on interim data). (4) The most common adverse reactions (> 20%) are ureteral obstruction, low back pain, urinary tract infection, hematuria, renal insufficiency, fatigue, nausea, abdominal pain, dysuria, and vomiting. Most adverse events are mild to moderate. It can be controlled using existing treatment methods and no treatment-related deaths have occurred.
LG-UTUC is a rare cancer that occurs in the upper urinary tract, ureters, and kidneys. In the United States, there are approximately 6000-7000 new or relapsed LG-UTUC patients each year. The anatomical structure of the urinary system is complex, and the treatment is difficult. Current standards of care include multiple operations, and most patients require radical nephroureterectomy, including removal of the pelvis, kidney, ureter, and bladder cuff. Complicating treatment is that LG-UTUC is most commonly seen in patients over 70 years of age. These patients may already have impaired renal function and may suffer further complications due to major surgery.
Seth Lerner, MD, chief investigator of the OLYMPUS trial and professor of urology at Baylor College of Medicine in Texas, said: "Radical nephroureterectomy is associated with decreased kidney function and other complications. Jelmyto will be responsible for those who may require Patients treated with ureterectomy provide a new non-surgical treatment. This novel, minimally invasive, kidney-retaining treatment may change the treatment of low-grade upper urothelial cancer, helping patients avoid surgery and Long-term complications related to kidney loss. "
Andrea Maddox-Smith, CEO of the Bladder Cancer Advocacy Network, said: "For decades, the treatment of low-grade upper urothelial carcinoma (LG-UTUC) has made little progress, and this new option will be for those Patients who often face relapses and major surgery to remove their kidneys bring a change in treatment model. We applaud FDA approval for Jelmyto. LG-UTUC seriously affects elderly patients, many of whom have been fighting the disease and have always wanted to have a There is no option for surgical treatment. "