Motixafortide

Motixafortide is an inhibitor of the C-X-C Motif Chemokine Receptor 4 (CXCR4) and blocks the binding of its cognate ligand, stromal-derived factor-1α (SDF-1α)/C-X-C Motif Chemokine Ligand 12 (CXCL12). SDF-1α and CXCR4 play a role in the trafficking and homing of human hematopoietic stem cells to the marrow compartment.

• APHEXDA is indicated in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma. APHEXDA, a hematopoietic stem cell mobilizer, is indicated in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma.ICD-10: C90.00

• APHEXDA is contraindicated in patients with a history of serious hypersensitivity reactions to motixafortide [see Warnings and Precautions (5.1) ] . History of serious hypersensitivity reaction to APHEXDA.contraindicated

Initiate APHEXDA treatment after filgrastim has been administered daily for 4 days. ( 2.1 ) Recommended dosage is 1.25 mg/kg actual body weight by subcutaneous injection 10 to 14 hours prior to initiation of apheresis. ( 2.2 ) A second dose of APHEXDA can be administered 10 to 14 hours prior to a third apheresis. ( 2.2 ) See Full Prescribing Information for instructions on preparation and administration. 2.1 Important Dosing Information Premedicate all patients before each dose of APHEXDA to reduce the risk of hypersensitivity and injection site reactions: Administer diphenhydramine (12.5 mg intravenously or 25 mg to 50 mg orally, or another H1-antihistamine), an H2 blocker (e.g., famotidine), and a leukotriene inhibitor (e.g., montelukast) approximately 30 to 60 minutes before injection of APHEXDA. The addition of an analgesic medication (e.g., acetaminophen) to the premedication regimen is recommended. Administer filgrastim 10 mcg/kg subcutaneously once daily for 4 days prior to the first dose of APHEXDA and on each day prior to each apheresis. 2.2 Recommended Dosage The recommended dosage of APHEXDA is 1.25 mg/kg administered via slow (approximately 2 minutes) subcutaneous injection 10 to 14 hours prior to the initiation of the first apheresis. Dosing is based on actual body weight. A second dose of APHEXDA can be administered 10 to 14 hours before a third apheresis, if necessary. 2.3 Preparation and Administration Use aseptic technique to prepare and administer APHEXDA: More than one vial may be needed for a full dose.

Anaphylactic Shock and Hypersensitivity Reactions: Premedicate all patients with a combination of an H1-antihistamine, an H2 blocker, and a leukotriene inhibitor prior to each APHEXDA dose. Administer APHEXDA in a setting where personnel and therapies are available for immediate treatment. Observe for signs and symptoms and manage promptly. ( 2.1 , 2.3 , 5.1 ) Injection Site Reactions: The addition of analgesic premedication (e.g., acetaminophen) is recommended. ( 5.2 ) Tumor Cell Mobilization in Patients with Leukemia: APHEXDA may mobilize leukemic cells and should not be used in leukemia patients. ( 5.3 ) Leukocytosis: Increased circulating leukocytes have been observed. Monitor white blood cell counts during APHEXDA use. ( 5.4 ) Potential for Tumor Cell Mobilization: Tumor cells may be released from marrow during HSC mobilization with APHEXDA and filgrastim. Effect of reinfusion of tumor cells is unknown. ( 5.5 ) Embryo-fetal Toxicity: Can cause fetal harm. Advise women of reproductive potential of the potential risk to a fetus and to use effective contraception. ( 5.6 , 8.1 , 8.3 ) 5.1 Anaphylactic Shock and Hypersensitivity Reactions Anaphylactic shock occurred in 0.7% of APHEXDA-treated patients in clinical studies (n=407). The time to anaphylactic shock was between 5 minutes and 30 minutes after drug administration. Hypersensitivity reactions occurred in 7.6% of APHEXDA-treated patients in the GENESIS study. In addition, pruritus, flushing, urticaria, rash, erythema, vomiting, nausea and chills have been reported.

The following clinically significant adverse reactions are discussed in other sections of the labeling: Anaphylactic Shock and Hypersensitivity Reactions [see Warnings and Precautions (5.1) ] Injection Site Reactions [see Warnings and Precautions (5.2) ] Potential for Tumor Cell Mobilization in Patients in Leukemia [see Warnings and Precautions (5.3) ] Leukocytosis [see Warnings and Precautions (5.4) ] Potential for Tumor Cell Mobilization [see Warnings and Precautions (5.5) ] Most common adverse reactions (incidence >20%) are injection site reactions, injection site pain, injection site erythema, injection site pruritus, pruritus, flushing, and back pain. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Gamida Cell at (844) 477-7478 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of APHEXDA was evaluated in the GENESIS study based on data from 92 patients with multiple myeloma who received at least one dose of APHEXDA 1.25 mg/kg subcutaneously and filgrastim and 42 patients who received placebo and filgrastim for mobilization of hematopoietic stem cells for collection and apheresis [see Clinical Studies (14) ] . The premedication regimen changed during the conduct of the trial as evidence of hypersensitivity reactions was noted.

Lactation: Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on its mechanism of action, APHEXDA can cause fetal harm when administered to a pregnant woman [see Clinical Pharmacology (12.1) ] . There are no available data with APHEXDA use in pregnant women informing the risk of embryo-fetal toxicity. Animal models link dysfunction in CXCR4/SDF-1 signaling to adverse outcomes in mammalian embryo-fetal development and suggest risks to normal placental development (see Data ) . No animal studies have been conducted to evaluate the effect of motixafortide on reproduction and fetal development. Advise pregnant women of the potential risk to a fetus. The background risk of major birth defects and miscarriages for the indicated population is unknown. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Data Animal Data Animal reproduction studies have not been conducted with motixafortide to evaluate its effect on reproduction and embryo-fetal development.

Metabolism

The pharmacokinetics of motixafortide were evaluated using both noncompartmental and population PK approaches in various populations, including healthy volunteers, patients with acute myeloid leukemia, and patients with multiple myeloma. Subjects received single or multiple doses ranging between 0.24 mg/kg to 2 mg/kg of APHEXDA administered by subcutaneous injections using a weight-based (mg/kg) dosing strategy as monotherapy or in combination with filgrastim with or without chemotherapy.

• Sep 8, 2023NDANDA217159Ayrmid Pharma

1. 1Injection Site Pain3337%
2. 2Pruritus2225%
3. 3Urticaria1820%
4. 4Flushing1618%
5. 5Rash1213%
6. 6Injection Site Erythema910%
7. 7Paraesthesia910%
8. 8Injection Site Reaction89.0%
9. 9Injection Site Pruritus77.9%
10. 10Discomfort66.7%
11. 11Adverse Drug Reaction55.6%
12. 12Erythema55.6%
13. 13Hypersensitivity55.6%
14. 14Injection Site Necrosis44.5%
15. 15Injection Site Swelling44.5%

The 10 most recently updated of 10 ClinicalTrials.gov registrations naming Motixafortide as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• SCD Stem Cell Mobilization and Apheresis Using Motixafortide
  Recruiting · Phase 1 · Interventional · 15 enrolled · St. Jude Children's Research Hospital
  NCT06442761updated 2026-06-03
• Gene Editing For Sickle Cell Disease
  Recruiting · Phase 1 · Interventional · 25 enrolled · St. Jude Children's Research Hospital
  NCT06506461updated 2026-05-05
• Motixafortide for MRD Sensitization in AML
  Not yet recruiting · Phase 2 · Interventional · 10 enrolled · Washington University School of Medicine
  NCT07392970updated 2026-04-09
• Evaluating Premedication Regimens (Methylprednisolone vs Dexamethasone-based) for the Prevention of Systemic and Injection Site Reactions to Motixafortide in Patients With Multiple Myeloma Undergoing Stem Cell Mobilization, PARADE Trial
  Recruiting · Phase 4 · Interventional · 94 enrolled · Emory University
  NCT07101445updated 2026-02-10
• Mobilization of Stem Cells With Motixafortide (BL-8040) in Combination With G-CSF in Multiple Myeloma Patients
  Recruiting · Phase 3 · Interventional · 60 enrolled · Guangzhou Gloria Biosciences Co., Ltd.
  NCT06514508updated 2025-12-16
• A Pharmacodynamic Study of the Apheresis Product of Multiple Myeloma Patients Undergoing Quad-induction Followed by Motixafortide + G-CSF Mobilization
  Completed · Phase 1 · Interventional · 20 enrolled · Washington University School of Medicine
  NCT06547112updated 2025-08-29
• Motixafortide and Natalizumab to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD)
  Completed · Phase 1 · Interventional · 10 enrolled · Washington University School of Medicine
  NCT05618301updated 2025-08-11
• Chemo4METPANC Combination Chemokine Inhibitor, Immunotherapy, and Chemotherapy in Pancreatic Adenocarcinoma
  Recruiting · Phase 2 · Interventional · 10 enrolled · Gulam Manji
  NCT04543071updated 2025-06-19
• Study to Investigate the Effect of BL-8040 (Motixafortide) on the QTc Interval in Healthy Subjects
  Completed · Phase 1 · Interventional · 38 enrolled · BioLineRx, Ltd.
  NCT05293171updated 2025-03-21
• Safety, Tolerability and Efficacy of the BL-8040 and Atezolizumab for Maintenance Treatment in Subjects With Acute Myeloid Leukemia (AML)
  Terminated · Phase 1 · Phase 2 · Interventional · 1 enrolled · BioLineRx, Ltd.
  NCT03154827updated 2024-09-05

Frequently asked questions

How does Motixafortide work?

Motixafortide is an inhibitor of the C-X-C Motif Chemokine Receptor 4 (CXCR4) and blocks the binding of its cognate ligand, stromal-derived factor-1α (SDF-1α)/C-X-C Motif Chemokine Ligand 12 (CXCL12). SDF-1α and CXCR4 play a role in the trafficking and homing of human hematopoietic stem cells to the marrow compartment.

What is Motixafortide used for?

According to FDA labeling, Motixafortide carries indications including: APHEXDA is indicated in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma. APHEXDA, a hematopoietic stem cell mobilizer, is indicated in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Motixafortide?

Motixafortide is classified as Other immunostimulants, Chemokine Receptor Type 4 Interactions, Hematopoietic Stem Cell Function Alteration, Increased Hematopoietic Stem Cell Mobilization.

What are the brand names for Motixafortide?

Motixafortide is marketed under brand names including Aphexda.

What are the contraindications for Motixafortide?

Motixafortide labeling lists contraindications including: APHEXDA is contraindicated in patients with a history of serious hypersensitivity reactions to motixafortide [see Warnings and Precautions (5.1) ] . History of serious hypersensitivity reaction to APHEXDA.. Always consult the full prescribing information and a clinician.