Jazz Pharmaceuticals completes FDA supplemental BLA for Rylaze for Monday/Wednesday/Friday dosing schedule.

The submission will be reviewed under the Real-Time Oncology Review (RTOR) program, an initiative of FDA's Oncology Center of Excellence designed for efficient review of safe and effective cancer treatments, and follows Rylaze's initial approval under the RTOR program in June 2021.

The sBLA submitted by Jazz is supported by data from the three-cohort intramuscular administration part of the Phase II/III trial of Rylaze in adult and pediatric patients with ALL and LBL who have developed hypersensitivity to an E. coli-derived asparaginase. The trial studied three dosing regimens of Rylaze, with cohort 1a receiving 25 mg/m2 administered M/W/F, cohort 1b receiving 37.5 mg/m2 administered M/W/F and cohort 1c receiving 25 mg/m2 administered Monday and Wednesday and 50 mg/m2 administered on Friday. Initial results showed that in cohort 1c, a dosing regimen of Rylaze administered 25 mg/m2 on Monday and Wednesday and 50 mg/m2 on Friday demonstrated a positive benefit-to-risk profile, showing that Rylaze maintains a clinically meaningful level of nadir serum asparaginase activity (NSAA) greater than 0.1 IU/mL at both 48 and 72 hours (from Friday to Monday). In addition, the safety profile of Rylaze was consistent with the reported safety information for patients with ALL/LBL receiving asparaginase with combination chemotherapy. Initial results from the trial were presented at the 63rd American Society of Hematology (ASH) Annual Meeting in December 2021.

nThe sBLA follows FDA approval of Rylaze in June 2021 under the RTOR program. Rylaze was also granted orphan drug designation for the treatment of ALL/LBL in June 2021 and was added to the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology (NCCN Guidelines®) in July 2021.