After 10 years of stagnation, a new FDA approval could help more cancer patients become eligible for stem cell transplants.
Monday, Israeli drugmaker BioLineRx said the FDA approved motixafortide—also known as Aphexda—in tandem with the decades-old colony-stimulating factor (CSF) filgrastim to mobilize hematopoietic stem cells to the peripheral blood for collection and autologous stem cell transplantation (ASCT) in patients with multiple myeloma.
Amgen first scored approval for filgrastim way back in 1991. That drug, marketed as Neupogen, has several biosimilar versions approved.
ASCT is part of the standard of care for multiple myeloma, which is the second-most common blood cancer, BioLineRx said in a press release. The success of ASCT depends on adequate mobilization of stem cells during the treatment process, the company explained.
Historically, however—depending on induction regimens and mobilization strategies—up to 47% of patients have had challenges reaching target numbers of hematopoietic stem cells for ASCT after one apheresis session.
Apheresis describes the removal of blood plasma from the body and its separation into plasma and cells, with the cells subsequently reintroduced to the patient.
BioLineRx says Aphexda is the first innovation in stem cell mobilization for multiple myeloma approved in the U.S. in a decade.
In a two-part study, one dose of Aphexda plus filgrastim helped most patients (67.5%) achieve a specific collection goal for hematopoietic stem cells, BioLineRx said in a press release. That compared to just 9.5% in the placebo-plus-filgrastim arm of the trial.
Additionally, 92.5% of patients on Aphexda reached the stem cell collection goal in up to two apheresis sessions, versus 21.4% in the placebo cohort.
Increased age, plus exposure to lenalidomide-containing induction regimens, have been linked to impaired stem cell mobilization, the company said. To account for that fact, the GENESIS study included patients representative of the “typical” multiple myeloma population undergoing ASCT, BioLineRx said.
As far as safety was concerned, 5.4% of patients on Aphexda experienced serious side effects, BioLineRx said. Those reactions included vomiting, injection site reaction, hypersensitivity reaction and injection site cellulitis plus low potassium levels in the blood and hypoxia—a state in which oxygen is unavailable in sufficient amounts at the tissue level to maintain adequate homeostasis.
"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," Philip Serlin, BioLineRx’s CEO, said in a statement.
BioLineRx says it expects to make Aphexda available later this month.