Aphexda™ (motixafortide) injection received approval from the U.S. Food and Drug Administration (FDA) on Sept. 8, 2023. The long-acting inhibitor of C-X-C motif chemokine receptor 4 (CXCR4) moves hematopoietic stem cells from bone marrow to the blood in outer areas of the body where they can be captured through apheresis for stem cell transplants, which also are called bone marrow transplants. Used along with the granulocyte-colony stimulating factor (G-CSF), filgrastim, Aphexda’s recommended dose is one 1.25mg/kg subcutaneous (SC) injection delivered between 10 hours and 14 hours before apheresis is scheduled. If needed, a second dose can be administered before a third apheresis procedure. One treatment of filgrastim should be given each morning for four consecutive days before Aphexda. Although the launch is expected later in September, the manufacturer, BioLineRx, has not announced cost or distribution plans. Complete prescribing information should be available soon on the FDA’s website (here) or at www.Aphexda.com.
At a Glance
- Brand (Generic) Name: Aphexda (motixafortide)
- Manufacturer: BioLineRx
- Date Approved: Sept. 8, 2023
- Indication: to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients who have multiple myeloma
- Dosage Forms Available: single-dose vials with 62mg of Aphexda powder for reconstitution
- Launch Date: September 2023
- Estimated Annual Cost: Not available
- Multiple myeloma is a progressive, incurable blood cancer that affects plasma cells. A type of white blood cell found in bone marrow, plasma cells are matured B-cells that produce antibodies. When damaged, they rapidly displace normal cells and create tumors in the bone marrow.
- The American Cancer Society (ACS) estimates that about 36,000 cases of multiple myeloma are diagnosed annually in the U.S. Men are somewhat more likely to have it than women. At about 14%, the risk for African Americans is nearly twice as high as for members of other ethnic groups.
- Treatment for multiple myeloma typically consists of chemotherapy (chemo) followed by an autologous stem cell transplant.
- Clinical guidelines recommend between 3X106 and 5X106 CD34+ stem cells/kg for a successful transplant. Up to one-third of candidates for stem cell transplants do not have enough stem cells to harvest and as many as 47% of patients need more than one apheresis session to get enough stem cells for a transplant.
- Aphexda stimulates stem cells to migrate from bone marrow into the blood, so they are easier to collect by apheresis.
- In a two-part clinical study, 63.8% of the patients needed only one apheresis procedure to harvest the goal of 6X106 or more stem cells versus 2.4% of those who got a placebo injection along with filgrastim. With two apheresis sessions, the numbers increased to 67.5% of patients in the Aphexda arm as compared with 9.5% of those in the placebo group.
- About 5% of patients who were treated with Aphexda had serious side effects that included cellulitis at the injection site, hypersensitivity reactions, and low blood levels of potassium or oxygen.
- More common side effects from Aphexda were back pain, flushing, and injection site reactions such as itching, pain, and redness.
Current stem cell mobilization regimens are a G-CSF either by itself, with some off-label chemo drugs, or in combination with Mozobil® (plerixafor/generics). Plerixafor is injected with SC once a day for up to four days.