The first innovation in stem cell mobilization for multiple myeloma was approved on September 11, 2023. BioLineRx announced the FDA approval of APHEXDA (motixafortide) in combination with Filgrastim (G-CSF) to mobilize stem cells for autologous stem cell transplants of multiple myeloma patients. 

This is exciting news for the myeloma community, as stem cell transplant is still the standard of care, and currently, about 47% of patients have had challenges collecting target numbers of hematopoietic stem cells for an autologous stem cell transplant after one apheresis session. 

Increased age, as well as exposure to REVLIMID (lenalidomide)-containing combination regimens have been associated with impaired stem cell transplant mobilization.

The newly approved APHEXDA improves stem cell mobilization significantly, with patients in the GENESIS clinical trial arm of APHEDA plus filgrastim (versus placebo and filgrastim) collecting more than four times the amount of stem cells with a single dose over a twenty-four-hour period. 

Patients in this trial were reflective of the myeloma population, with the median age of 63 and ~70% of the patients having received a lenalidomide-containing regimen previous to their stem cell transplant. 

Transplant specialists expect around three to five million cells to be collected during apheresis. While that number sounds astronomical, that's the number of stem cells it takes to complete two successful stem cell transplants. 

APHEXDA is a subcutaneous injection, making it easy for myeloma patients to receive. That being said, some of the common side effects during the clinical trial were: 

• vomiting 
• injection site reaction (pain, erythema, and pruritus)
• flushing
• back pain 
• hypokalemia 
• hypoxia 

The list above is not comprehensive, please discuss with your healthcare team about potential side effects before receiving APHEXDA. 

Dr. John DiPersio, MD, PhD, the primary investigator for the GENESIS trial says,

"Innovation in this area of medicine has been needed...[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 [multiple myeloma]." 

For those who choose to have a stem cell transplant as part of their myeloma care, this newly approved injection will improve stem cell collection time and efficiency. It's an exciting time of improvement and progress in the myeloma treatment world! 

To learn more about Stem Cell Transplant, check out our HealthTree resources below: 

• HealthTree University Unit 9: Stem Cell Transplant
• Stem Cell Transplant Webinars and Recordings
• Stem Cell Transplant Comprehensive Guide for Myeloma Patients and Caregivers