The Food and Drug Administration (FDA) has granted expedited approval of Jelmyto (mitomycin for pyelocalyceal solution; UroGen Pharma) for the treatment of adult patients with low-grade upper tract urothelial cancer (LG-UTUC).
The approval was based on data from the ongoing open-label, single-arm, phase 3 OLYMPUS study that assessed the efficacy of Jelmyto in 71 adults with treatment-naïve or recurrent noninvasive LG-UTUC with at least 1 measurable papillary tumor 5 to ≤15mm located above the ureteropelvic junction. Patients received Jelmyto 4mg/mL once weekly via ureteral catheter or nephrostomy tube for a total of 6 instillations. The primary end point was complete response, defined as the complete absence of tumor lesions at 3 months after initiation of therapy. A key secondary end point was the long-term durability of complete response at 12 months.
Results showed that 41 of the 71 patients achieved complete response (58%; 95% CI, 45-69). At the 12-month assessment of durability, 19 patients (46%) continued to have complete response; 7 patients experienced a recurrence of disease and 9 patients continued to be followed for the 12-month duration of response. The durability of complete response at 12 months was estimated to be 84% by Kaplan-Meier analysis, based on interim data.
With regard to safety, the most common adverse reactions (≥20%) reported were ureteric obstruction, flank pain, urinary tract infection, hematuria, renal dysfunction, fatigue, nausea, abdominal pain, dysuria, and vomiting. Most adverse events were found to be mild to moderate and manageable. No treatment-related deaths occurred.
“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 Seth Lerner, MD, FACS, Professor of Urology at Baylor College of Medicine in Houston, TX and Principal Investigator of the OLYMPUS trial. “This novel, minimally invasive, kidney-sparing treatment has the potential to transform the way low-grade upper tract urothelial cancer is treated and help patients avoid long-term complications associated with surgery and the loss of their kidney.”
Jelmyto will be supplied as a carton containing two 40mg single-dose vials of mitomycin for pyelocalyceal solution and one 20mL single-dose vial of sterile hydrogel. The product is expected to be available June 1, 2020.
For more information visit jelmyto.com.