Here’s a look at the next two target action dates on the calendar for early April.
Although the U.S. Food and Drug Administration (FDA)’s focus is on the COVID-19 pandemic, PDUFA dates are still on the calendar and drugs are being approved. Here’s a look at the next two target action dates on the calendar for early April.
Bristol Myers Squibb and Acceleron’s Reblozyl for MDS
Bristol Myers Squibb and Acceleron Pharma have a target action date of April 4, 2020 for the companies’ supplemental Biologics License Application (sBLA) for the use of Reblozyl (luspatercept-aamt) in patients with myelodysplastic syndromes (MDS). The sBLA is for adults with very low- to intermediate-risk MDS-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions.
The drug was approved in 2019 for anemia in adults with beta thalassemia who require regular RBC transfusions. The drug is being jointly developed as part of a global collaboration. Ongoing trials include the Phase III COMMANDS trial in erythroid stimulating agent-native, lower-risk MDS patients, the Phase II BEYOND trial in adults with non-transfusion-dependent beta thalassemia, and a Phase II trial in myelofibrosis patients.
On March 26, the companies published results from the BELIEVE Phase III pivotal trial of Reblozyl for anemia in adults with beta thalassemia who require regular RBC transfusions in the New England Journal of Medicine.
The drug was originally developed by Celgene and Acceleron. Celgene is now a Bristol Myers Squibb company.
UroGen’s UGN-101 for Upper Tract Urothelial Cancer
UroGen has a target action date of April 18 for UGN-101 (mitomycin gel) for instillation as a potential treatment for patients with low-grade upper tract urothelial cancer (LG UTUC). If approved, it would be the first non-surgical treatment option for LG UTUC. It was accepted as Priority Review. The drug has previously received Orphan Drug, Fast Track, and Breakthrough Therapy Designations.
The company developed the drug using the RTGel technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation. The drug is designed to provide longer exposure of urinary tract tissue to mitomycin and is delivered via standard ureteral catheters.
Urogen initiated its rolling submission of the UGN-101 New Drug Application (NDA) to the FDA in December 2018.
The NDA was built on positive data from the pivotal Phase III OLYMPUS trial. The final data of the primary endpoint demonstrated a complete response rate of 59% in patients with LG UTUC. The durability of response was estimated as 89% at six months and 84% at 12 months by Kaplan Meier analysis. Median time to recurrence was estimated to be 13 months.
“The FDA filing acceptance and granting of priority review for UGN-101 is an important milestone in our mission to pioneer new treatments to improve patient care in specialty cancers and urologic diseases,” said Liz Barrett, president and chief executive officer of UroGen, in a December 2019 statement. “There is a significant unmet need for a better treatment option for patients with LG UTUC, as the current standard of care involves surgical removal of the kidney or repetitive endoscopic tumor removal.”
LG UTUC is a urothelial cancer that presents in the lining of the upper urinary tract, in the ureters and renal collecting system of the kidneys. It is a rare but critical cancer. It affects about 6,000 to 8,000 new patients in the U.S. annually.
At the company’s March 2 fourth quarter and 2019 financial results, Barrett said, “At UroGen, we are eagerly awaiting potential approval of our lead product candidate, UGN-101, for the treatment of patients with low-grade upper tract urothelial cancer (LG UTUC). The significant progress on key clinical, regulatory and commercial milestones in 2019 places us in a position of strength as we prepare to deliver the first non-surgical therapy for the treatment of LG UTUC. Our experienced commercial team has been working tirelessly to ensure we are prepared for launch, and we look forward to providing these patients with a new treatment option.”
