"Our new study is the first post-market analysis to see how doctors are using this drug in routine practice and what their patient outcomes are,” said Soloman Woldu, MD.
Investigators across 15 different institutions have completed the largest review of post-approval utilization of UGN-101 (Jelmyto) in real-world practice for patients with upper tract urothelial carcinoma (UTUC).1
The mitomycin-containing reverse thermal gel was approved by the FDA in 2020 for the treatment of low-grade UTUC based on data collected from OLYMPUS, a single-arm, phase 3 trial (NCT02793128).
“This drug, UGN-101, was approved based on a trial with a limited number of patients. Our new study is the first post-market analysis to see how doctors are using this drug in routine practice and what their patient outcomes are,” said Soloman Woldu, MD, a co-leader of the study, in a news release on the findings.2 Woldu is an assistant professor of urology at UT Southwestern Medical Center in Dallas, Texas.
The study included 132 patients being treated for 136 different cancers, with the majority of patients having low-grade UTUC confirmed with a biopsy. Approximately 43% of patients had previously ablated tumors and were seeking UGN-101 to prevent recurrence.
UGN-101 was administered to the patients via different methods, although the most common was antegrade instillation via a percutaneous nephrostomy. 74% of patients received the full 6 doses of the induction course of UGN-101, and 27% of patients received monthly maintenance doses.
Findings showed that approximately 69% of patients in the adjuvant setting had no recurrence of disease at a 3-month follow-up. In the chemoablative setting, 37% of patients were endoscopically clear at the 3-month follow-up.
Investigators also found that patients with a smaller tumor size had higher rates of complete response. In tumors that had a volume of less than 1 cm, residual disease had a complete response rate of 70%. In patients who had tumors with a volume greater than 3 cm, tumors disappeared in about a quarter of cases.
Complications reported included development of urinary tract infections during treatment in approximately 15% of patients, as well ureteral stenosis in approximately 25% of patients. Five patients required kidney removal.
These findings represent the largest amount of patient data collected on the utilization of UGN-101 in a real-world setting, which can inform treatment for the drug moving forward.
“Patients with upper tract urothelial cancers are often left with the choice of lifelong dialysis or untreated cancers. This drug has enormous potential to save them from either of these fates,” said Woldu in the news release.
The investigators are in the process of developing a prospective registry (uTRACT) to further investigate these findings on a larger scale and test their durability.
The OLYMPUS trial was an initial single-arm phase 3 trial to test the safety, efficacy, and tolerability of UGN-101.3 The primary end point of the study was complete response based on ureteroscopy and cytology following a 6-week induction course of UGN-101.
The study cohort consisted of patients 18 years or older with primary or recurrent low-grade UTUC as confirmed by a biopsy. Each patient received 6 once-weekly instillations of UGN-101 via retrograde catheter to the renal pelvis and calyces.
Investigators found a complete response rate of 59% in patients with at least 1 dose of the thermal gel at the time of the primary disease evaluation visit, which was 4 to 6 weeks following the initial therapy. The durability of response (DOR) at 12 months was 82%. Rollover data from the OLYMPUS trial showed a median DOR of 28.9 months.
1. Woldu SL, Labbate C, Murray KS, et al. Early experience with UGN-101 for the treatment of upper tract urothelial cancer – a multicenter evaluation of practice patterns and outcomes. Urol Oncol. 2022;S1078-1439(22)00429-X. doi:10.1016/j.urolonc.2022.10.029
2. UTSW-led study shows promise for drug to treat upper urinary tract cancers. UT Southwestern Medical Center. News release. Published online January 4, 2023. Accessed January 18, 2023. https://www.newswise.com/articles/utsw-led-study-shows-promise-for-drug-to-treat-upper-urinary-tract-cancers?sc=mwhr&xy=10016681
3. Matin SF, Pierorazio PM, Kleinmann N, et al. Durability of response to primary chemoablation of low-grade upper tract urothelial carcinoma using UGN-101, a mitomycin-containing reverse thermal gel: OLYMPUS trial final report. J Urol. 2022;207(4):779-788. doi:10.1097/JU.0000000000002350