Jazz Pharmaceuticals Plc (NASDAQ: JAZZ) has announced that it has finalized the submission of a supplemental Biologics License Application to the US FDA for Monday/Wednesday/Friday intramuscular dosing schedule approval for Rylaze. The company is seeking approval to use Rylaze as a multi-agent chemotherapeutics regimen component for acute lymphoblastic leukaemia and lymphoblastic lymphoma treatment in pediatric and adult patients that have developed hypersensitivity to E. coli-derived asparaginase.
Rylaze was initially approved under the RTOR program.
Rylaze’s initial clearance under the Real-Time Oncology Review (RTOR program in June 2021) will examine this application via the RTOR initiative. RTOR is an FDA Oncology Center of Excellence initiative intended to expedite the review of effective and safe cancer medicines.
The company’s global head of R&D and EVP Rob Iannone said, “We were pleased Rylaze, a much-needed therapeutic option, was approved under the RTOR program while the clinical trial was ongoing. Our science-led and patient-focused development program has enabled us to deliver a clinically significant advancement for patients.”
Patients maintained a clinically relevant level of nadir blood asparaginase activity throughout treatment using a Rylaze dose regimen of 25/25/50 mg/m2 on Monday, Wednesday, and Friday. The company plans to submit two more regulatory submissions this year to ensure that as many patients as possible can access a reliable and greater supply of this crucial therapy, including a regulatory application to the FDA to support the intravenous method of administration and an EU application later this year.
Phase 2/3 study data of Rylaze supported sBLA
Data from the three-cohort intramuscular administration portion of the Phase 2/3 study of Rylaze in paediatric and adult ALL and LBL patients that have developed hypersensitivity to an E. coli derived asparaginase supported the sBLA. The study evaluated three-dose regimens of Rylaze of 25 mg/m2, 37.5 mg/m2, and 25 mg/m2 administered Monday and Wednesday and 50mg/m2 on Friday. Preliminary results showed a positive benefit-to-risk profile, demonstrating that Rylaze maintains a clinically significant nadir serum asparaginase activity level.
The sBLA came following Rylaze’s FDA approval in June 2021 under the RTOR initiative.
