FDA approves Aphexda for mobilization of stem cells before transplant in multiple myeloma
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The FDA approved motixafortide combined with filgrastim for mobilization of hematopoietic stem cells into peripheral blood during collection for subsequent autologous transplantation in individuals with multiple myeloma.
Motixafortide (Aphexda, BioLineRx) — administered by subcutaneous injection — is a high-affinity CXCR4 antagonist with a long receptor occupancy of more than 72 hours.
The agent is indicated in combination with the granulocyte colony-stimulating factor filgrastim (Neupogen, Amgen).
Motixafortide blocks CXCR4 on hematopoietic stem cells from binding to its ligand CXCL12 and remaining in the bone marrow and lymph nodes. CXCR4 blocking allows hematopoietic stem cells to enter the peripheral blood for collection during apheresis.
The FDA based approval on results from the randomized phase 3 GENESIS trial. The double-blind, placebo-controlled study evaluated the safety and efficacy of motixafortide plus filgrastim compared with filgrastim alone for hematopoietic stem cell mobilization.
The study included 122 adults undergoing subsequent autologous hematopoietic stem cell transplant for multiple myeloma.
A central laboratory assessment of CD34+ cells after apheresis showed that motixafortide plus filgrastim allowed significantly more patients to achieve the study’s stem cell collection goal of at least 6 × 106 CD34+ cells/kg within two apheresis sessions compared with those who received filgrastim plus placebo (67.5% vs. 9.5%).
Meanwhile, local laboratory assessment revealed that 92.5% of patients in the motixafortide plus filgrastim group met the study’s stem cell collection goal in up to two apheresis sessions compared with 21.4% in the placebo group.
Treatment-related serious adverse reactions occurred in 5.4% of those who received motixafortide plus filgrastim. The most frequent treatment-related toxicities occurring in more than 20% of study participants included injection site reactions, pruritus, flushing and back pain.
"Greater numbers of patients with multiple myeloma are candidates for autologous stem cell therapy; however, achieving target collection goals can be difficult in some patients given modern barriers, including the treatment of older patients and use of contemporary induction regimens," John DiPersio, MD, PhD, primary investigator for the GENESIS trial and professor of medicine, pathology and immunology, as well as director of the Center for Gene and Cellular Immunotherapy, at Washington University School of Medicine in St. Louis, said in a BioLineRx-issued press release. “Innovation in this area of medicine has been needed, and today's approval of Aphexda addresses the demand for new therapies that can meet today's challenges by delivering more reliability in stem cell mobilization, versus filgrastim alone, with fewer days of apheresis sessions and fewer doses of filgrastim for people living with this cancer."