Besponsa approved in the EU for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.
Pfizer Inc. announced that the European Commission has approved Besponsa (inotuzumab ozogamicin) as monotherapy for the treatment of adults with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL). This indication includes treatment of adults with Philadelphia chromosome positive (Ph+) as well as Philadelphia chromosome negative (Ph-) relapsed or refractory B-cell precursor ALL.
Adults with Ph+ relapsed or refractory CD22-positive B-cell precursor ALL should have failed treatment with at least one tyrosine kinase inhibitor (TKI). With this approval, Besponsa becomes the first and only antibody drug conjugate (ADC) available for patients with this type of leukemia in the European Union (EU).
“The European Commission’s approval of Besponsa represents an important milestone for patients, the oncology community and Pfizer,” said Andreas Penk, M.D., regional president, Pfizer Oncology. “This is the first approval for Besponsa and provides patients in the EU, who are battling an especially hard-to-treat leukemia, with a new treatment option beyond chemotherapy.”
ALL is an aggressive type of leukemia that can be fatal within a matter of months if left untreated.1 The goal of treatment in relapsed or refractory (resistant) ALL is to achieve complete remission without excessive toxicity so patients may proceed to additional therapeutic intervention, particularly stem cell transplant, which is the most recognized option to prolong patient survival, maintenance therapy or other therapy.2
In adult patients with relapsed or refractory ALL, median overall survival is just three to six months.3,4,5 The current standard of care is intensive chemotherapy6, which is effective in less than 50 percent of relapsed or refractory patients and associated with poor long-term survival, high toxicities, lengthy inpatient stays and continuous infusions.7
“Acute lymphoblastic leukemia that has recurred or is refractory following first-line therapy is a rare and rapidly progressive disease with poor prognosis,” said Professor David Marks, Department of Hematology, University Hospitals Bristol NHS Foundation Trust, Bristol, United Kingdom. “The approval of Besponsa (inotuzumab ozogamicin) provides a much needed treatment option for physicians and patients alike, that may help improve outcomes for some of the most vulnerable leukemia patients in Europe.”
The European Commission’s approval of Besponsa is supported by results from the Phase 3 INO-VATE ALL trial, in which 326 adult patients with relapsed or refractory B-cell precursor ALL were enrolled and which compared Besponsa to standard of care chemotherapy. The INO-VATE ALL study had two primary endpoints, complete response with or without hematologic recovery (CR/CRi) and overall survival (OS). Results from the trial were published in The New England Journal of Medicine in June 2016.
In the U.S., Besponsa received Breakthrough Therapy designation from the Food and Drug Administration (FDA) in October 2015 for ALL. A Biologics License Application (BLA) for Besponsa for the treatment of adult patients with relapsed or refractory B-cell precursor ALL was accepted for filing and granted Priority Review by the FDA in March 2017. The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA is August 2017.
With a growing hematology pipeline, Pfizer is committed to extending therapeutic progress in acute and chronic leukemias that leverage select pathways and mechanism of actions (MOAs). Specifically, our investigational products aim to treat some of the hardest to treat leukemias and lymphomas including, acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), chronic myeloid leukemia (CML) and mantle cell lymphoma (MCL).
1 National Cancer Institute: Adult Acute Lymphoblastic Leukemia Treatment (PDQ®) – General Information About Adult Acute Lymphoblastic Leukemia (ALL). Available at: http://www.cancer.gov/cancertopics/pdq/treatment/adultALL/HealthProfessional/page1. Accessed March 21, 2016.
2 Gokbuget N. et al. Outcome of relapsed adult lymphoblastic leukemia depends on response to salvage chemotherapy, prognostic factors, and performance of stem cell transplantation. Blood. 2012; 120(10): 2032-2041.
3 Advani AS. New immune strategies for the treatment of acute lymphoblastic leukemia: Antibodies and chimeric antigen receptors. Hematology Am Soc Hematol Educ Program. 2013;131-7.
4 Tavernier E et al. Outcome of treatment after first relapse in adults with acute lymphoblastic leukemia initially treated by the LALA-94 trial. Leukemia. 2007 Sep;21(9):1907-14. Epub 2007 Jul 5.
5 Fielding AK et al. Outcome of 609 adults after relapse of acute lymphoblastic leukemia (ALL); an MRC UKALL12/ECOG 2993 study. Blood. 2007 Feb 1;109(3):944-50. Epub 2006 Oct 10.
6 American Cancer Society: Typical treatment of acute lymphocytic leukemia. Available at: http://www.cancer.org/cancer/leukemia-acutelymphocyticallinadults/detailedguide/leukemia-acute-lymphocytic-treating-typical-treatment. Accessed March 21, 2016.
7 Alan K. Burnett. Treatment of acute myeloid leukemia: are we making progress? School of Medicine, Cardiff University, Cardiff, United Kingdom. Hematology 2012.
(Source: Business Wire)