FDA approves Rylaze for acute lymphoblastic leukemia, lymphoblastic lymphoma
The FDA approved asparaginase erwinia chrysanthemi (recombinant)-rywn as a component of a chemotherapy regimen to treat certain adults or children with acute lymphoblastic leukemia or lymphoblastic lymphoma.
The approval applies to use of the agent by patients aged 1 month or older who are allergic to E. coli-derived asparaginase products, which are commonly used in treatment.
The other FDA-approved drug indicated for patients with similar allergic reactions has been in shortage for years, according to the FDA.
“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,” Gregory Reaman, MD, associate director for pediatric oncology in FDA’s Oncology Center of Excellence, said in an agency-issued press release. “[This] approval may provide a consistently sourced alternative to a pivotal component of potentially curative therapy for children and adults with this type of leukemia.”
ALL is the most common type of childhood cancer. About half of the 5,700 people diagnosed annually are children.
Asparaginase — an enzyme that kills cancer cells — is one component of the chemotherapy regimen used for treatment. However, approximately 20% of patients are allergic to the standard E. coli-derived asparaginase.
The FDA based approval of erwinia chrysanthemi (recombinant)-rywn (Rylaze, Jazz Pharmaceuticals) on results of an ongoing phase 2/phase 3 study that included 102 patients who had either a hypersensitivity to E. coli-derived asparaginases or experienced silent inactivation.
Results showed the recommended dosage would provide the target level of asparaginase activity for approximately 94% of patients.
The most common adverse events associated with the therapy included hypersensitivity reactions, pancreatic toxicity, blood clots, hemorrhage and liver toxicity.
The FDA previously granted fast track and orphan drug designations to erwinia chrysanthemi (recombinant)-rywn for this indication.
“The accelerated development and approval of Rylaze marks an important step in bringing a meaningful new treatment option for many [patients with ALL] — most of whom are children — who cannot tolerate E. coli-derived asparaginase medicine,” Luke Maese, DO, assistant professor at The University of Utah, Primary Children’s Hospital and Huntsman Cancer Institute, said in a Jazz Pharmaceuticals-issued press release. “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.”