FDA Approves First Therapy for Treatment of Low-Grade Upper
U.S. Food and Drug Administration approved Jelmyto (mitomycin gel), the first therapy to treat low-grade upper tract urothelial cancer (UTUC). Urothelial cancer is a cancer of the lining of the urinary system.

Jelmyto is an alkylating drug, meaning it inhibits the transcription of DNA into RNA, stopping protein synthesis and taking away the cancer cell’s ability to multiply.

The FDA approved Jelmyto based on the results of a clinical trial involving 71 patients with low-grade UTUC. These patients had never undergone treatment or had recurrent low-grade non-invasive UTUC with at least one measurable papillary tumor. Patients received Jelmyto once a week for six weeks and, if assessed as a complete response (complete disappearance of the papillary tumor), monthly for up to 11 additional months. Efficacy of Jelmyto was evaluated using urine cytology (a test to look for abnormal cells in a patient’s urine), ureteroscopy (an examination of the upper urinary tract) and biopsy (if warranted) three months following the initiation of therapy.

The primary endpoint was complete response at three months following initiation of therapy. A complete response was found in 41 of the 71 patients (58%) following six treatments of Jelmyto administered weekly. Durability of the effect of Jelmyto in patients with a complete response was also evaluated using urine cytology, ureteroscopy and biopsy (if warranted) every three months for a year following the initiation of therapy. Nineteen patients (46%) who achieved a complete response continued to have a complete response at the 12-month mark.

Common side effects for patients taking Jelmyto were ureteric obstruction (narrowing or blockage of the ureter that may lead to excess fluid in the kidney due to a backup of urine), flank pain (pain occurring on the side of the body), urinary tract infection, hematuria (blood in the urine), renal dysfunction (inability of the kidney to function in its designed capacity), fatigue, nausea, abdominal pain, dysuria (painful or difficult urination) and vomiting.

The FDA granted this application Priority Review and Breakthrough Therapy designation, which expedites the development and review of drugs that are intended to treat a serious condition, when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies.

The FDA granted approval of Jelmyto to UroGen Pharma, Inc.

Source: https://www.medpagetoday.com/hematologyoncology/othercancers/85983
S S and 10 others like this2 shares