The Food and Drug Administration (FDA) has approved Aphexda (motixafortide) in combination with bone marrow stimulant filgrastim (G-CSF) for the mobilization of hematopoietic stem cells for collection and subsequent autologous transplantation in patients with multiple myeloma, biopharmaceutical company BioLineRx announced on Monday.
BioLineRx, in a news release, reported that one dosage of Aphexda plus filgrastim enabled most patients to reach the collection goal of six million or more hematopoietic stem cells among a contemporary population of patients with multiple myeloma. Aphexda is purported by BioLineRx to be “the first innovation in stem cell mobilization for multiple myeloma to be approved in the U.S. in a decade.”
Aphexda, as BioLineRx explained in the news release, “is a CXCR4 antagonist with long receptor occupancy (greater than 72 hours) that, in combination with filgrastim, enables mobilization of hematopoietic stem cells to the peripheral blood for collection and subsequent autologous stem cell transplantation in patients with multiple myeloma.”
BioLineRx reported that it expects to make Aphexda available later this month.
The approval follows results from the two-part, phase 3, randomized, double-blind, placebo controlled GENESIS trial, which evaluated Aphexda plus filgrastim versus placebo plus filgrastim among patients with multiple myeloma. The study, according to its listing on clinicaltrials.gov, launched in 2018 and is estimated to be completed in December of 2025.
With a median participant age of 63, GENESIS had 12 patients treated at a single center in its first part, followed by 122 patients involved in a multicenter study in its second part. Serious side effects were reported in 5.4% of patients, according to the news release, including vomiting, injection site reactions, hypersensitivity reaction, injection site cellulitis, hypokalemia (low potassium levels) and hypoxia (low oxygen in tissues). The most common side effects among more than 20% of GENESIS participants were injection site reactions, pruritus (itchy skin), flushing and back pain.
Results of the study published in April in the journal Nature Medicine showed that 92.5% of patients treated with Aphexda plus filgrastim reached the six million or more hematopoietic stem cell collection goal after two procedures, compared to 26.2% of patients treated with placebo, and that 88.8% of Aphexda group patients reached that benchmark after one procedure, versus 9.5% of patients in the placebo group.
“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,” said Dr. John DiPersio, primary investigator for the GENESIS trial and professor of medicine, pathology and immunology and director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine in St. Louis, in the news 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.”
Autologous stem cell transplantation (ASCT), BioLineRX noted in the news release, is part of the standard of care treatment regimen for patients with multiple myeloma, and its success “depends on adequate mobilization of stem cells during the treatment process.”
“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 introduce a new treatment paradigm for this challenging cancer,” said Philip Serlin, chief executive officer of BioLineRx, in the news reselase. “The company is working relentlessly to make this important innovation in stem cell mobilization available to appropriate patients, their physicians and transplant teams.”
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