Aphexda approved in US to improve autologous stem cell transplants
Developer BioLineRx to make Aphexda available later this month
The U.S. has approved BioLineRx’s Aphexda (motixafortide) in combination with filgrastim (G-CSF) to improve the process of autologous hematopoietic stem cell transplant (AHSCT) for people with multiple myeloma.
AHSCT’s success depends on efficacious mobilization of healthy blood stem cells into the bloodstream for collection and subsequent transplant. In the Phase 3 GENESIS clinical trial (NCT03246529), the combo was shown to help mobilize blood stem cells in fewer sessions and with fewer doses of medication.
“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 company press release.
BioLineRx expects to make Aphexda available in the U.S. later this month.
“The company is working relentlessly to make this important innovation in stem cell mobilization available to appropriate patients, their physicians and transplant teams,” Serlin added.
Approval marks ‘innovation in stem cell mobilization for multiple myeloma’
The approval by the U.S. Food and Drug Administration comes about 10 months after the agency agreed to review the regulatory application, and marks “the first innovation in stem cell mobilization for multiple myeloma to be approved in the U.S. in a decade,” BioLineRx stated in the release.
Multiple myeloma is a blood cancer arising from abnormal plasma cells, a type of white blood cell made in the bone marrow. These abnormal plasma cells begin to multiply uncontrollably in the bone marrow, affecting the production of blood stem cells, which give rise to all types of blood cells.
A standard myeloma treatment, AHSCT involves collecting a patient’s own healthy blood stem cells using a procedure called apheresis, and eliminating all remaining blood cells in the body with chemotherapy. Then, the stem cells are transplanted back so that the body can be repopulated with new and healthy blood cells.
The procedure’s success depends on how many blood stem cells can be mobilized from the bone marrow. This is a slow process that usually involves daily doses of filgrastim before apheresis to stimulate stem cell mobilization.
The recommended collection target number is 3 million to 5 million stem cells per kilogram (kg) of body weight. But up to 47% of myeloma patients have difficulties in meeting collection target numbers after one apheresis session, the company noted.
“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 John DiPersio, MD, PhD, the GENESIS trial’s principal investigator at Washington University School of Medicine in St. Louis.
Aphexda is expected to help stem cells move from the bone marrow into the bloodstream by binding to and blocking a protein called CXCR4, which normally keeps them in the bone marrow.
With 122 adult myeloma patients enrolled, the second part of the multicenter GENESIS trial evaluated the safety and efficacy of Aphexda plus filgrastim versus filgrastim plus a placebo for the mobilization of stem cells for AHSCT.
The trial’s participants were representative of the current patient population, with a median age of 63 and with about 70% in both groups receiving the anti-cancer medication Revlimid (lenalidomide).
Aphexda was administered as an under-the-skin injection at a dose of 1.25 mg per kg, and the main goal was to collect at least six million stem cells per kg in up to two apheresis sessions.
Collection goal reached in most patients given Aphexda plus filgrastim
The collection goal was reached in most (92.5%) patients in the Aphexda plus filgrastim group, compared with 21.4% of those given filgrastim plus a placebo, as measured by local laboratories.
The superiority of the Aphexda combo over filgrastim alone was also observed when measurements were done by central laboratories, with more than two-thirds (67.5%) of patients meeting the goal versus 9.5% of those in the filgrastim plus placebo group.
Also, patients given Aphexda plus filgrastim needed fewer filgrastim injections (5.26 vs. 8.12) and fewer apheresis procedures (1.23 vs. 3.24) to collect enough stem cells relative to those receiving filgrastim alone.
The combo therapy was generally safe and well tolerated, with the most common adverse event being temporary injection site reactions.
Serious side effects occurred in 5.4% out of the 92 patients who received the Aphexda combo. Those side effects included vomiting, injection site reactions, allergic reactions, low potassium, and low oxygen levels.
“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,” DiPersio said.
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