FDA approves orphan drug to treat ALL

The FDA has approved asparaginase Erwinia chrysanthemi to treat patients with acute lymphoblastic leukemia who have shown a hypersensitivity to asparaginase and pegaspargase chemotherapy drugs derived from Escherichia coli.

Asparaginase E. chrysanthemi (Erwinaze, EUSA Parma) is injected into the muscle three times a week and breaks down the amino acid asparagine, a protein building block in the blood required for cell growth.

Leukemia cells, which cannot produce asparagines, die after treatment. The ability of healthy cells to produce asparagine is not affected.

“The approval of Erwinaze underscores the FDA’s commitment to the approval of drugs for conditions with limited patient populations with unmet medical needs using novel trial endpoints,” Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release.

The approval was based on a clinical trial involving 58 patients with ALL, as well as on data from the Erwinaze Master Treatment Protocol, in which another 843 patients with ALL were enrolled.

Allergic reactions prevented patients in both studies from continuing to receive E. coli-derived pegaspargase (Oncaspar, Enzon Pharmaceuticals) or asparaginase (Elspar, Merck).

The primary outcome was sustained asparaginase activity levels that indicate better disease control and survival. All evaluable patients achieved the prespecified asparaginase activity threshold at 48 or 72 hours after dosing.

Adverse effects reported in patients who received the treatment included pancreatitis, anaphylaxis, blood clotting, hemorrhaging, abnormal transaminases and bilirubin, hyperglycemia and nausea. For more information please visit the FDA website.

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