Jazz Pharmaceuticals Wins FDA Nod for New ALL and LBL Drug

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Jazz Pharmaceuticals won U.S. regulatory approval for a new leukemia drug aimed at patients who have developed hypersensitivity to E. coli-derived asparaginase, an enzyme that is a component of the chemotherapy regimen used to treat acute lymphoblastic leukemia (ALL). 

Late Thursday, the U.S. Food and Drug Administration (FDA) greenlit Jazz Pharmaceuticals’ Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) for use as a component of a chemotherapy regimen for the treatment of ALL or lymphoblastic lymphoma (LBL) in pediatric and adult patients. Rylaze is the only recombinant erwinia asparaginase manufactured product that provides a clinically meaningful level of asparaginase activity throughout the entire duration of treatment. It was developed by Jazz to address the needs of patients with what was described as “an innovative, high-quality erwinia-derived asparaginase with reliable supply.” 

Up to 30% of ALL and LBL patients have a sensitivity to E. coli-derived asparaginase, including many juvenile patients. Data from a recent analysis of more than 8,000 juvenile patients conducted by Children's Oncology Group found that patients who did not receive a full course of asparaginase treatment due to associated toxicity had significantly lower survival outcomes. In its own announcement about the approval of Rylaze, the FDA noted that the only other drug approved for patients with this sensitivity has been in global shortage for years. 

The company said Rylaze offers a meaningful option for these patients.

Rylaze was approved based on data from the Phase II portion of a Phase II/III study assessing different delivery options for the medication. The Phase II study is investigating the intramuscular (IM) delivery and the Phase III portion will assess an intravenous delivery of the drug. 

The FDA approval of Rylaze was based on data from the first of three IM cohorts, which demonstrated the achievement and maintenance of nadir serum asparaginase activity. The study found that the recommended dosage would provide the target level of asparaginase activity in 94% of patients.

The approval was granted weeks after the FDA granted orphan drug designation for Rylaze. Approval of the formulation was granted under the FDA’s Real-Time Oncology Review (RTOR) program, which was established for the efficient delivery of safe and effective cancer treatments to patients.

Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals, expressed his excitement at bringing a new treatment to patients who are in critical need of a treatment for patients that will not cause a sensitivity issue. 

“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," Cozadd said in a statement. 

Luke Maese, assistant professor at the University of Utah, Primary Children's Hospital and Huntsman Cancer Institute, said the approval of Rylaze is an important step in bringing new treatment options for leukemia patients. 

Before the approval of Rylaze, there was a significant need for an effective asparaginase medicine that would allow patients to start and complete their prescribed treatment program with confidence in supply,” Maese said.

Following approval for this indication, Jazz Pharma said it will continue to work with the FDA and will submit additional data from a completed cohort of patients evaluating the intramuscular dosing of Rylaze, as well as other dosing measures.

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