The FDA approval was supported by findings from the two-part, Phase 3 GENESIS trial in which the combination met the primary endpoint with a high degree of statistical significance
A micrograph of myeloma cast nephropathy. (Credit: Nephron from Wikimedia Commons)
Biopharmaceutical company BioLineRx announced that the US Food and Drug Administration (FDA) has approved Aphexda (motixafortide) in combination with filgrastim (G-CSF) for use on multiple myeloma (MM) patients.
Aphexda is a CXCR4 antagonist with long receptor occupancy, more than 72 hours.
The US health regulator has approved the Aphexda combination to mobilise hematopoietic stem cells (HSC) to the peripheral blood for collection and subsequent autologous transplantation in multiple myeloma patients.
The FDA approval was supported by findings from the two-part, Phase 3 GENESIS trial.
According to BioLineRx, the part 1 single centre, lead-in, open-label study involved 12 patients treated with motixafortide plus filgrastim designed to ascertain the dose. The part 2 involved 122 patients who were randomised 2:1 in a double-blind, placebo-controlled, multicenter study.
The study met the primary endpoint with a high degree of statistical significance, the Israel-based biopharmaceutical firm said.
In the trial, the Aphexda combination enabled 67.5% of patients to achieve the stem cell collection goal of ≥ 6 × 106 CD34+ cells/kg within two apheresis sessions, against 9.5% for the placebo plus filgrastim regimen.
In addition, 92.5% of patients achieved the stem cell collection goal in up to two apheresis sessions in the Aphexda arm and 21.4% in the placebo arm, as measured by local laboratories.
BioLineRx CEO Philip Serlin said: “Given the strong efficacy data shown in the GENESIS trial, which included patients who are representative of the current multiple myeloma patient population, we believe Aphexda will play a critical role in addressing unmet needs and introducing a new treatment paradigm for this challenging cancer.”
The GENESIS trial is a randomised, double-blind, placebo-controlled study with the goal of assessing the safety and efficacy of Aphexda plus filgrastim, against placebo plus filgrastim for the mobilisation of HSC for autologous transplantation in MM patients.
The primary endpoint was to evaluate if one dose of Aphexda plus filgrastim is superior to placebo plus filgrastim in the ability to mobilise ≥ 6 million CD34+ cells in up to two apheresis sessions.
The key secondary endpoint was to assess whether one dose of motixafortide plus filgrastim is superior to placebo plus filgrastim in the same ability.