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Rylaze FDA Approved as Part of a Treatment Regimen for Pediatric Leukemia and Lymphoma

August 2021 Vol 14, Special Issue: Payers' Perspectives in Oncology - FDA Approvals, Leukemia, Lymphoma, Conference Highlights ASCO

On June 30, 2021, the FDA accelerated the approval of asparaginase erwinia chrysanthemi (recombinant)-rywn (Rylaze; Jazz Pharmaceuticals), an asparagine-specific enzyme, as a component of a multidrug chemotherapy regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma in patients aged ≥1 months with hypersensitivity to Escherichia coli–derived asparaginase.

The FDA granted erwinia chrysanthemi an orphan drug designation for this indication. Erwinia chrysanthemi will address the shortages of Erwinia asparaginase (Erwinaze). Rylaze “has the same molecular makeup as Erwinaze. But it’s manufactured differently, and in a more efficient way, so having the option to substitute Rylaze should reduce the likelihood of treatment shortages for these patients in the future,” said Malcolm A. Smith, MD, PhD, Associate Branch Chief for Pediatric Oncology, NCI’s Cancer Therapy Evaluation Program.

Erwinia chrysanthemi can be used as a substitute to Erwinia asparaginase in people with an immune reaction to pegaspargase. These ongoing shortages have been “incredibly stressful to patients and their parents,” said Sumit Gupta, MD, PhD, University of Toronto, and Pediatric Oncologist, Hospital for Sick Children, Toronto, Canada.

The FDA approved erwinia chrysanthemi based on results of an open-label, multicohort, multicenter clinical trial of 102 patients (median age, 10 years; range, 1-24 years) with ALL or lymphoblastic lymphoma and hypersensitivity to asparaginase caused by E coli infection, as part of a multidrug chemotherapy regimen.

The primary end point was achievement and maintenance of nadir serum asparaginase activity (NSAA) >0.1 U/mL. The results of modeling and simulations showed that for a dosage of 25 mg/m2 administered every 48 hours, the proportion of patients maintaining NSAA ≥0.1 U/mL at 48 hours after a dose of erwinia chrysanthemi was 93.6% (95% confidence interval, 92.6%-94.6%).

The most common (>20%) adverse reactions were abnormal liver test, nausea, musculoskeletal pain, fatigue, infection, headache, pyrexia, drug hypersensitivity, febrile neutropenia, decreased appetite, stomatitis, bleeding, and hyperglycemia.

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Last modified: August 30, 2021