Rylaze is indicated for ALL/LBL in paediatric and adult patients, who have developed hypersensitivity to E. coli-derived asparaginase
Jazz Pharmaceuticals has received the US Food and Drug Administration (FDA) approval for Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) as a component of the treatment regimen for acute lymphoblastic leukaemia (ALL) or lymphoblastic lymphoma (LBL).
The US regulator indicated Rylaze for ALL/LBL in paediatric and adult patients, aged one month and above, who have developed hypersensitivity to E. coli-derived asparaginase.
Rylaze, also known as JZP458, is a recombinant erwinia asparaginase that uses a novel Pseudomonas fluorescens expression platform.
The drug was granted the FDA Fast Track designation in October 2019 and was approved as part of the agency’s Real-Time Oncology Review programme.
The Irish drugmaker claimed that its Rylaze is the only recombinant erwinia asparaginase product that works to maintain the optimal level of asparaginase activity during treatment.
Jazz Pharmaceuticals chairman and CEO Bruce Cozadd said: “We are excited to bring this important new treatment to patients who are in critical need, and we are grateful to FDA for the approval of Rylaze based on its established safety and efficacy profile.
“We are pleased Rylaze was approved before the trial is complete and are diligently working to advance additional clinical trial data.
“We are committed to quickly engaging with FDA to evolve the Rylaze product profile with additional dosing options and an IV route of administration.”
The FDA approval was based on the data from an ongoing Phase 2/3 dose confirmation study evaluating the safety, tolerability and efficacy of Rylaze in 102 participants.
The Phase 2/3 study is designed to be conducted in two parts and to determine the efficacy as measured by serum asparaginase activity (SAA) levels.
Evaluating the intramuscular (IM) route of administration, including a Monday-Wednesday-Friday dosing schedule forms the first part of the study.
The second part aims to further confirm the dose and schedule for the intravenous (IV) route of administration, said the company.
In the study, IM doses of Rylaze 25 mg/m showed the achievement and maintenance of nadir serum asparaginase activity (NSAA) greater than or equal to 0.1 U/mL in 94% of patients.
In July last year, the company received the FDA approval for Xywav oral solution to treat cataplexy or excessive daytime sleepiness (EDS) and narcolepsy.