On July 5, 2021 Jazz Pharmaceuticals reported that it is flying high after hitting two back-to-back regulatory milestones late last month (Press release, Jazz Pharmaceuticals, JUL 5, 2021, View Source [SID1234584747]). The first was obtaining a seven-year Orphan Drug Exclusivity for Xywav, the latest contender from its array of treatments for daytime sleepiness. Then, days later, the Dublin-based biopharma announced notching a speedy FDA approval for its blood cancer drug.
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On June 30th, Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) was greenlighted as a component of a chemotherapy regimen designed for treating acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) in both children and adults. The drug received accelerated approval from the agency under the Real-Time Oncology Review (RTOR) program.
"We are excited to bring this important new treatment to patients who are in critical need, and we are grateful to FDA for the approval of Rylaze based on its established safety and efficacy profile. We are pleased Rylaze was approved before the trial is complete and are diligently working to advance additional clinical trial data," said Bruce Cozadd, Chairman, and CEO of Jazz Pharmaceuticals, in a statement.
"We are committed to quickly engaging with FDA to evolve the Rylaze product profile with additional dosing options and an IV route of administration."
L-Asparignase as an ALL Treatment
ALL is a rare type of blood cancer but still accounts for 30% of all cancers in children. In 1953, a discovery by Dr. John Graydon Kidd led to the widespread use of L-asparaginase as a treatment for ALL. It was found that the deprivation of the amino acid, L-asparagine, sends the leukemic cells into starvation mode, eventually killing them owing to the blockage in DNA, RNA, and protein synthesis.
Over the years, asparaginase derived from Escherichia coli was typically used as first-line therapy, but around two-thirds of patients were found to develop an allergic reaction. Such patients continued treatment with Erwinia chrysanthemi asparaginase.
Encouraging Trial Data
Rylaze is a recombinant erwinia asparaginase produced from a novel Pseudomonas fluorescens expression platform. The FDA approval was based on encouraging data from an ongoing Phase 2/3 trial that assessed the drug’s safety, tolerability, and efficacy. The study evaluated Rylaze in pediatric and adult patients with ALL or LBL who have had an allergic reaction to E. coli-derived asparaginases and have not previously received asparaginase from erwinia chrysanthemi.
Results from the first of three cohorts showed that patients who received 25 mg/m2 of Rylaze through intramuscular injection achieved intended levels of serum asparaginase activity, and nearly 94% of them maintained ≥ 0.1 U/mL of enzyme levels at 48 hours after administration.
"The accelerated development and approval of Rylaze marks an important step in bringing a meaningful new treatment option for many ALL patients – most of whom are children – who cannot tolerate E. coli-derived asparaginase medicine," said Dr. Luke Maese, Assistant Professor at the University of Utah, Primary Children’s Hospital and Huntsman Cancer Institute.
"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," he added.
Jazz reported total revenue of $2.36 Billion in 2020, of which $1,757.0 million was brought in by its combined oxybate business. Meanwhile, its total oncology net product sales accounted for $554.5 million. With the approval of some new oncology drugs, the company would be expected to exceed that number this year.