UroGen Pharma announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for UGN-102 (mitomycin) intravesical solution for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of June 13, 2025.
UGN-102 Clinical Trial Data
The NDA is supported by data from a comprehensive development program, including the Phase 3 ENVISION trial. The ENVISION trial demonstrated a clinically meaningful complete response (CR) rate of 79.6% (95% CI, 73.9%, 84.5%) at three months following the first instillation of UGN-102. Durability data from the ENVISION trial showed an 82.3% (95% CI, 75.9%, 87.1%) 12-month duration of response (DOR) by Kaplan-Meier estimate in patients who achieved a complete response at three months.
According to UroGen, this is the highest DOR ever reported in this patient population. The ENVISION trial also demonstrated an acceptable safety profile for UGN-102, consistent with previous clinical trials.
UroGen's Perspective
"The recent FDA acceptance of our New Drug Application for UGN-102 marks a significant milestone in our mission to deliver breakthrough treatments to patients," said Liz Barrett, President and Chief Executive Officer of UroGen. "Supported by robust clinical evidence, we believe UGN-102 has the potential to redefine the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer and provide longer treatment-free intervals for patients that currently face the burden of repetitive surgeries under general anesthesia. We look forward to a potential FDA approval by the PDUFA target action date of June 13, 2025. If approved, we believe UGN-102 will represent a paradigm shift in care, addressing a total market opportunity of over $5 billion. Our team is fully focused on preparing for a successful commercial launch of UGN-102, if approved, in 2025."
JELMYTO Performance
UroGen also reported that JELMYTO (mitomycin) for pyelocalyceal solution, used in low-grade upper tract urothelial cancer (LG-UTUC), generated net product revenue of $25.2 million in the third quarter of 2024, compared to $20.9 million in the third quarter of 2023.
Next-Generation Products
The first patient has been dosed in the Phase 3 UTOPIA clinical trial of investigational drug UGN-103 (mitomycin) for intravesical solution in patients with LG-IR-NMIBC. UroGen plans to initiate a Phase 3 study early next year to explore the safety and efficacy of UGN-104, its next generation product for the treatment of LG-UTUC.
Financial Position
As of September 30, 2024, UroGen's cash, cash equivalents, and marketable securities totaled $254.2 million.
