Health

FDA OKs Key Component of Acute Leukemia Regimen

WASHINGTON — The FDA approved a recombinant form of a key component of a chemotherapy regimen for acute lymphoblastic leukemia (ALL), the most common childhood cancer.

The approval of recombinant asparaginase erwinia chrysanthemi-rywn (Rylaze) encompasses treatment regimens for adults and pediatric patients with ALL who are allergic to asparaginase products derived from Escherichia coli. A global shortage of the E. coli-derived products has existed since 2016.

“It is extremely disconcerting to patients, families, and providers when there is a lack of access to critical drugs for treatment of a life-threatening but often curable cancer, due to supply issues,” said Gregory Reaman, MD, of the FDA Oncology Center of Excellence, in a statement. “Today’s approval may provide a consistently sourced alternative to a pivotal component of potentially curative therapy for children and adults with this type of leukemia.”

About 5,700 new cases of ALL occur each year, half of them involving children. An estimated 20% of patients are allergic to standard E. coli-derived asparaginase and require alternative products. Recent research in ALL suggests worse survival outcomes for patients unable to complete a full course of asparaginase, regardless of risk status.

Supporting data for approval of the recombinant product came from a study of 102 patients with hypersensitivity to E. coli-derived asparaginase or who developed neutralizing antibodies leading to silent inactivation. The primary outcome was achievement and maintenance of a target level of asparaginase activity. The trial data showed that 94% of patients met the target.

Patients included in the analysis were enrolled in an ongoing phase II/III trial of recombinant asparaginase. They received the treatment by intramuscular injection. A second component of the trial will confirm the dose and schedule of an intravenous formulation of the product.

Side effects occurring in more than 15% of patients were abnormal liver tests, nausea, musculoskeletal pain, fatigue, infection, headache, pyrexia, drug hypersensitivity, febrile neutropenia, decreased appetite, stomatitis, bleeding, and hyperglycemia.

Serious adverse events (SAEs) occurred in 55% of the patients, and one patient died of infection. SAEs occurring in at least 5% of patients consisted of febrile neutropenia, dehydration, pyrexia, stomatitis, diarrhea, drug hypersensitivity, infection, nausea, and viral infection. Adverse events led to permanent discontinuation in 9% of patients in the trial.

The FDA granted the approval to Jazz Pharmaceuticals, which announced that the recombinant asparaginase product will be available by mid-July.

  • Charles Bankhead is senior editor for oncology and also covers urology, dermatology, and ophthalmology. He joined MedPage Today in 2007. Follow

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