On the 16th, the US FDA announced that it approved the listing of Jelmyto (mitomycin gel) developed by UroGen Pharma for the treatment of low-grade upper urothelial carcinoma (UTUC). The press release states that this is the first approved drug therapy for this specific patient group.
Most urothelial cancers occur in the bladder, while UTUC occurs in the kidney or ureter. It can block the ureter or kidney, causing swelling, infection, and kidney damage in some patients. UTUCs can be classified as low-grade malignant tumors or high-grade malignant tumors. In general, low-grade malignant tumors are not aggressive and rarely spread from the kidney or ureter. However, they often relapse, and the usual treatment goal is to treat visible tumors and try to preserve the urinary tract.
Jelmyto is an alkylating drug that can inhibit the transcription of DNA into RNA, stop protein synthesis and deprive cancer cells of their proliferative ability. It has obtained the fast-track qualification, orphan drug qualification, priority review qualification and breakthrough therapy certification granted by the FDA.
FDA approval is based on the results of a clinical trial involving 71 patients with low-grade UTUC. The results of the trial showed that 41 of the 71 patients (58%) had complete remission. Within one year after starting treatment, 19 patients (46%) who achieved complete remission continued to achieve complete remission at 12 months.
"Although the current national focus is to combat the COVID-19 epidemic, cancer patients and their unique needs remain the top priority of the FDA," Dr. Richard Pazdur, director of the FDA Center for Cancer Excellence and acting director of the Office of Oncology and Diseases at the Center for Drug Evaluation and Research "We will continue to accelerate the development of oncology products at this critical moment. Our staff is continuing to meet with drug developers, academic researchers, and patient advocates to advance the coordinated review of cancer drugs, biological products, and devices."
Dr. Pazdur added: “This is the first approved treatment for patients with low-grade UTUC. Many patients previously required radical surgery, which meant complete removal of the affected kidney, ureter and bladder cuff. Jelmyto provided these patients for the first time An alternative treatment option. "