FDA approves Rylaze for acute lymphoblastic leukaemia and lymphoblastic lymphoma

July 2021 Pharma News Tobias Rawson

Rylaze, a recombinant erwinia asparaginase, has received regulatory approval for use in the Unites States. To be used as part of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukaemia (ALL) and lymphoblastic lymphoma (LBL) in patients one month of age or older, the drug received an accelerated approval and orphan-drug status, via the FDA’s real-time oncology review programme.

Asparagine is a non-essential amino acid synthesized by cells throughout the body. However, some cancer cells, such as those in ALL and LBL, are unable to produce their own asparagine, and must instead rely on circulatory asparagine. The enzyme asparaginase breaks down circulating asparagine, at no detriment to healthy cells, which are still able to produce their own supply intracellularly, whilst starving cancer cells of this amino acid, ultimately resulting in cell death.

Therapeutic levels achieved by 93.6% of patients

Manufactured by Jazz Pharmaceuticals, Rylaze is the only recombinant erwinia asparaginase product that maintains a clinically meaningful level of asparaginase activity throughout the duration of treatment. Approval follows the ongoing review of the phase II/III of the drug in a cohort of 102 ALL and LBL patients, all of which were required to have had a previous allergic reaction to E. coli-derived asparaginase, and who have not previously been treated with asparaginase produced from Erwinia chrysanthemi, known since 2005 as Dickeya dadantii. Data of the first of three cohorts from this study demonstrates both the achievement and subsequent maintenance of a nadir serum asparaginase activity (NSAA) ≥0.1 U/mL. At a dose of 25 mg/m2 given intramuscularly every 48 hours, 93.6% of patients were able to maintain a NSAA ≥ 0.1 U/mL at 48 hours. Occurring in 20% or more, the most common adverse events included abnormal liver values, nausea, musculoskeletal pain, febrile neutropenia, stomatitis, bleeding and hyperglycaemia.

Speaking on the approval, Jazz CEO Bruce Cozadd said “We are pleased Rylaze was approved before the trial is complete and are diligently working to advance additional clinical trial data”. The approval comes in light of a recent retrospective analysis from the Children’s Oncology Group of over 8000 paediatric patients indicating that patients who did not receive the full course of asparaginase treatment due to toxicity had significantly worse survival outcomes, irrespective of if they were high or low risk. By offering an alternative for these patients who have adverse or allergic reaction to E. coli-derived asparaginase, it is hoped that Rylaze will be able to fulfil an unmet clinical need.  

Reference:

Jazz Pharmaceuticals. Jazz Pharmaceuticals Announces U.S FDA Approval of RylazeTM (asparaginase erwinia chrysanthemi (recombinant)-rywn) for the Treatment of Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma. [internet]. 2021. Available from: https://investor.jazzpharma.com/news-releases/news-release-details/jazz-pharmaceuticals-announces-us-fda-approval-rylazetm

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