According to a recent FDA press release, the agency has given its first approval for a drug developed specifically for adult patients diagnosed with upper tract, low-grade, urothelial cancer.
The alkylating gel, mitomycin (JELMYTO), was developed by UroGen Pharma. It functions by interfering with the DNA in a cell and inhibits the growth of cancer cells.
Mitomycin gel inhibits the transcription of DNA into RNA by copying a gene’s DNA sequence to create an RNA molecule.
If successful, Mitomycin gel can provide an alternative for patients in lieu of radical surgery. There are significant challenges in treating patients with urothelial cancer because of the complexity of the upper urinary track. Patients are faced with removal of the affected ureter, bladder cuff and affected kidney.
About the OLYMPUS Trial
The recent approval of the gel was based on the OLYMPUS trial (NCT02793128). The trial is ongoing. It involves seventy-one patients either newly diagnosed or with one or more papillary tumors situated above the area where the ureter meets the bladder (ureteropelvic junction).
JELMYTO was administered in six week instillations using a ureteral catheter or a nephrostomy tube which is a small tube placed through the lower back into the kidney.
Results of the Trial
If patients showed a complete response after three months, then the instillations would be performed on a monthly basis for eleven additional months.
The researchers were judging the efficacy of JELMYTO according to the number of patients who had a complete response combined with the durability of those responses.
If patients were free of tumors for three months post JELMYTO treatment, they were considered to be in complete recovery. Two tests, ureteroscopy and cytology, were used to make these determinations.
Fifty-eight percent or forty-one patients were in complete recovery at the three month mark. These patients continued to be monitored. Twenty-nine patients were given one or more doses of therapy as maintenance.
The durability of the response was assessed at three-month intervals. There were recurrences in seven patients but nineteen patients were still in complete recovery at the twelve month mark.
Note that the median duration target for response was not reached.
Approximately twenty percent of patients receiving JELMYTO experienced some but not all of the following symptoms:
- Ureteric obstructions
- Urinary tract infection
- Flank pain
- Renal dysfunction
- Fatigue, abdominal pain, and nausea or vomiting
Fifty-eight percent of the JELMYTO patients experienced ureteric obstruction requiring that a ureteral stent be place in eighty-eight percent of these patients.
The FDA granted three designations to JELMYTO:
- Priority review: The FDA reviews the application within 6 months
- Fast track: the FDA attempts to make a decision within 60 days
- Breakthrough Therapy: the FDA grants priority review if the clinical trial shows that the drug offers substantial advantages over similar drugs currently on the market
About UroGen Pharma
UroGen, based in New York, NY, is a biopharmaceutical company dedicated to developing products and solutions intended to revolutionize treatment of urologic diseases and cancer.