Site Editors
Shaji K. Kumar, MD
Prashant Kapoor, MD, FACP
Shaji K. Kumar, MD
Prashant Kapoor, MD, FACP
Posted: Monday, September 11, 2023
Today, BioLineRx Ltd announced that the U.S. Food and Drug Administration (FDA) has approved motixafortide (Aphexda) in combination with the colony-stimulating factor filgrastim to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous stem cell transplantation (ASCT) in patients with multiple myeloma. Motixafortide, which targets chemokine (C-X-C motif) receptor 4 (CXCR4), is administered by injection for subcutaneous use.
The FDA approval of this combination is based on results from the two-part, phase III GENESIS trial. This randomized, double-blind, placebo-controlled study evaluated the safety and efficacy of motixafortide plus filgrastim, compared with placebo plus filgrastim, for the mobilization of hematopoietic stem cells for ASCT in patients with multiple myeloma.
The combination of motixafortide plus filgrastim enabled 67.5% of patients to achieve the stem cell collection goal of ≥ 6 × 106 CD34-positive cells/kg within two apheresis sessions, versus 9.5% for the placebo plus filgrastim regimen, as measured by a central laboratory. In addition, 92.5% of patients reached the stem cell collection goal in up to two apheresis sessions in the combination therapy arm and 21.4% in the placebo arm, as measured by local laboratories. Local laboratory data were used for a sensitivity analysis. The data are descriptive and were not statistically powered nor prespecified. The information should be interpreted cautiously.
In the GENESIS trial, safety was evaluated in 92 patients with multiple myeloma who received motixafortide at 1.25 mg/kg subcutaneously plus filgrastim and 42 patients who received placebo plus filgrastim. Serious adverse reactions occurred in 5.4% of patients receiving the combination regimen. These reactions included vomiting, injection-site reaction, hypersensitivity reaction, injection-site cellulitis, hypokalemia, and hypoxia. The most common adverse reactions occurring in the GENESIS trial (incidence > 20%) were injection-site reactions (pain, erythema, and pruritus), pruritus, flushing, and back pain.
