APHEXDA: A New Frontier in Multiple Myeloma Treatment Approved by the FDA on September 8, 2023

Jul 22, 2024

What is APHEXDA prescribed for? 

APHEXDA is used 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.

What is the name of the drug and what does it do?

APHEXDA , pronounced ah fex’ dah, (generic name: motixafortide) is a newly approved drug designed to work alongside filgrastim (G-CSF) to mobilize hematopoietic stem cells from bone marrow to the bloodstream. 

This process is essential for the collection and subsequent autologous transplantation in patients battling multiple myeloma, a cancer that targets plasma cells.

How does it work?

APHEXDA targets and blocks the CXCR4 receptor, a critical element that normally holds stem cells within the bone marrow. 

By inhibiting this receptor, APHEXDA allows more stem cells to enter the bloodstream, making them accessible for collection and transplantation, enhancing the treatment’s effectiveness.

What did the research discover?

The research on APHEXDA focused on its ability to work with filgrastim, a drug already used to help move stem cells from the bone marrow into the bloodstream. 

APHEXDA enhances this process by targeting a specific pathway in the bone marrow that usually keeps the stem cells inside. 

When APHEXDA is added, it blocks this pathway, allowing more stem cells to move into the bloodstream than with filgrastim alone.

In clinical trials, this combination of APHEXDA and filgrastim proved to be very effective. Patients who received both drugs had a significantly higher number of stem cells in their blood, which is crucial for successful stem cell transplantation. 

This effectiveness means that patients might need to undergo fewer sessions of apheresis—a procedure where blood is drawn and processed to collect stem cells—making the whole process more efficient and less burdensome for the patient.

What are some of the side effects?

The side effects listed are from the product manufacturer. As health scientists, we share this information to keep you informed without causing undue worry. Many side effects diminish over time, and not all patients experience them. If prescribed medication, take it as directed and consult your healthcare professional if you have any concerning side effects.

  • Injection Site Reactions: Patients may experience pain, redness, or swelling at the injection site.
  • Anaphylactic Shock and Hypersensitivity Reactions: These severe allergic reactions can occur shortly after administration, emphasizing the need for readiness to manage such reactions effectively.
  • Tumor Cell Mobilization: In non-leukemia patients, there is a risk that tumor cells may be mobilized along with stem cells, potentially complicating the transplantation process.
  • Embryo-fetal Toxicity: The drug can harm the fetus if administered to pregnant women due to its mechanism of action.

What are the dosage recommendations and how is it prescribed?

The recommended dose of APHEXDA is 1.25 mg/kg of body weight, administered subcutaneously 10 to 14 hours before starting apheresis, after four days of receiving filgrastim. 

A second dose may be necessary before a third apheresis depending on the patient’s response.

Source:

  1. Highlights of Prescribing Information. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/217159s000lbl.pdf. Accessed 16 July 2024.

2. Drug Trials Snapshots: APHEXDA. FDA. Available from: https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-aphexda. Accessed 16 July 2024.

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