The FDA Approves Rylaze for Leukemia and Lymphoma

The therapy, a recombinant version, was developed to address supply and manufacturing issues.

The FDA has approved Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) as a component of a chemotherapy regimen to treat acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients who are allergic to the E. coli-derived asparaginase products used most commonly for treatment.

Jazz Pharmaceuticals, the therapy’s developer, expects Rylaze to be commercially available in mid-July.

The only other FDA-approved drug for such patients with allergic reactions has been under global shortage since 2016. Jazz Pharmaceuticals’ Erwinaze (asparaginase erwinia chrysanthemi) has experienced supply and manufacturing issues from the owner and manufacturer of the product, Porton Biopharma Limited. Jazz Pharmaceuticals’ agreement with Porton Biopharma ended in December 2020.

In May 2021, the FDA had authorized Porton Biopharma and its distributor Clinigen to import Erwinase (crisantaspase) into the United States and bring this therapy to physicians.

Rylaze is the only recombinant erwinia asparaginase manufactured product that maintains a clinically meaningful level of asparaginase activity throughout the entire duration of treatment, and it was developed by Jazz to address the needs of patients and health care providers with a reliable supply.

Rylaze’s efficacy was evaluated in a study of 102 patients who either had a hypersensitivity to E. coli-derived asparaginases or experienced silent inactivation. The main measurement was whether patients achieved and maintained a certain level of asparaginase activity. The study found that the recommended dosage would provide the target level of asparaginase activity in 94% of patients.

Jazz Pharmaceuticals is continuing to study Rylaze. The ongoing phase 2/3 trial is being conducted in two parts. The first part is investigating the intramuscular route of administration, including a Monday-Wednesday-Friday dosing schedule. The second part remains active to further confirm the dose and schedule for the intravenous route of administration.

Rylaze was granted orphan drug designation for the treatment of ALL/LBL by FDA in June 2021. The Biologics Licensing Application (BLA) approval followed review under the Real-Time Oncology Review (RTOR) program, an initiative of FDA’s Oncology Center of Excellence designed for efficient delivery of safe and effective cancer treatments to patients.

Acute lymphoblastic leukemia occurs in about 5,700 patients annually, about half of whom are children. It is the most common type of childhood cancer. One component of the chemotherapy regimen is an enzyme called asparaginase that kills cancer cells by depriving them of substances needed to survive. An estimated 20% of patients are allergic to the standard E. coli-derived asparaginase and need an alternative their bodies can tolerate.