"Today's positive CHMP opinion is welcome news for those in the ALL and LBL community who are unable to be treated with E. coli-derived asparaginase due to hypersensitivity reactions," says Robert Iannone, MD., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "We look forward to receiving the final decision that will help bring us one step closer to delivering a reliable supply of recombinant Erwinia asparaginase to patients in the European Union." The CHMP's recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines in all European Union Member States, Iceland, Norway, and Liechtenstein, and is expected to make a final decision soon.
Asparaginase is a core component of multi-agent chemotherapeutic regimens in ALL,6 however, up to 30% of patients develop hypersensitivity to E. coli-derived asparaginase,7 necessitating treatment discontinuation or a switch to a non-E. coli-derived asparaginase preparation.8 Patients not receiving asparaginase due to hypersensitivities and those not receiving all prescribed doses have been shown to have poor outcomes.9,10 Lymphoblastic Lymphoma (LBL) is a rare, fast-growing, aggressive subtype of non-Hodgkin's lymphoma (NHL), which is very rare in adults and is most often seen in teenagers and young adults under the age of 35.11,12 LBL is a type of high-grade lymphoma – which means the lymphoma grows quickly with early spread to different parts of the body.12 LBL is the second most common type of NHL in childhood and adolescence, accounting for 25-35% of cases.13 Jazz Pharmaceuticals plc (NASDAQ: JAZZ) is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing life-changing medicines for people with serious diseases—often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines and novel product candidates, from early- to late-stage development, in neuroscience and oncology. We are a leader in sleep disorders and epilepsy; further developing in movement disorders and PTSD; and focused on hematologic malignancies and solid tumors. Within these therapeutic areas, we are identifying new options for patients by actively exploring small molecules and biologics, and through innovative delivery technologies and cannabinoid science. Jazz is headquartered in Dublin, Ireland, and has employees around the globe, serving patients in nearly 75 countries. Please visit www.jazzpharmaceuticals.com for more information. Head of Strategic Brand Engagement
Vice President, Head, Investor Relations
1 National Cancer Institute. Adult acute lymphoblastic leukemia treatment (PDQ®)–Patient Version. Cancer.gov. Updated November 19, 2021. Accessed July 02, 2023. https://www.cancer.gov/types/leukemia/patient/child-all-treatment-pdq.
3 Chennamadhavuni A, et al. Leukemia. In: StatPearls [Online]. Treasure Island (FL). StatPearls Publishing. Updated January 17, 2023. Accessed July 02, 2023. https://www.ncbi.nlm.nih.gov/books/NBK560490/. 5 Hoelzer D, et al. Acute lymphoblastic leukaemia in adult patients: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2016;27(suppl 5):v69–v82.
9 Maese L, Rizzari C, Coleman R, et al. Can recombinant technology address asparaginase Erwinia chrysanthemi shortages? Pediatr Blood Cancer. 2021;68(10):e29169. DOI 10.1002/pbc.29169. 11 Leukemia Foundation. Lymphoblastic lymphoma. Leukaemia.org.au. Updated June 18, 2019. Accessed July 02, 2023. https://www.leukaemia.org.au/blood-cancer/lymphoma/non-hodgkin-lymphoma/lymphoblastic-lymphoma/. 12 Macmillan Cancer Support. Cancer information and support – lymphoblastic lymphoma. Macmillan.org.uk. Updated January 31, 2021. Accessed July 02, 2023. https://www.macmillan.org.uk/cancer-information-and-support/lymphoma/non-hodgkin/types/lymphoblastic.
13 Burkhardt B, et al. Lymphoblastic lymphoma in children and adolescents: review of current challenges and future opportunities. Br J Haematol. 2019;185(6):1158–1170. 14 U.S. Food and Drug Administration. Rylaze prescribing information. Accessdata.fda.gov. Updated 2021. Accessed July 02, 2023. https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761179s000lbl.pdf.