Motixafortide (APHEXDA)_Dosage, benefits & legal status
Also known as APHEXDA, BL-8040, BKT140, 4F-benzoyl-TN14003, motixafortide acetate, TN14003
The same endpoint, counted twice: 92.5% in the journal, 67.5% on the label.
What is Motixafortide (APHEXDA)?
Plain language first. The technical label stays, but it never stands alone.Overview
Before a myeloma patient gets high-dose chemotherapy, doctors harvest the patient's own blood-forming stem cells and freeze them, then give them back afterward to rebuild the bone marrow. The harvest only works if enough stem cells can be coaxed out of the marrow and into the bloodstream, where a machine can filter them out. The standard drug for that, filgrastim, often does not shake loose enough of them, especially in older patients and in anyone who has had certain myeloma drugs first. This peptide blocks the molecular anchor that holds stem cells in the marrow. One injection, ten to fourteen hours before collection, and many more patients reach the target in a single session instead of coming back day after day. The catch is in the numbers. The journal paper that made this drug famous reported that 92.5% of patients hit the collection target. The FDA label for the same trial and the same endpoint says 67.5%. Both are accurate; they come from two different laboratories counting the same cells.
Motixafortide is a synthetic 14-residue cyclic peptide antagonist of CXCR4, approved by the FDA in September 2023 under NDA 217159 and given with filgrastim to mobilize hematopoietic stem cells into the blood for collection before autologous transplantation in multiple myeloma. One subcutaneous dose of 1.25 mg/kg is given 10 to 14 hours before the first apheresis. CXCR4 is the receptor that tethers stem cells to their marrow niche; blocking it releases them into circulation, and the receptor stays occupied for more than 72 hours, and that is why a single dose is enough. The drug requires antihistamine, H2 blocker and leukotriene inhibitor premedication 30 to 60 minutes before each dose and an hour of observation afterward, because of anaphylaxis and hypersensitivity risk.
What it's used for
Given with filgrastim to mobilize hematopoietic stem cells for collection and subsequent autologous transplantation in adults with multiple myeloma. Also under study in metastatic pancreatic adenocarcinoma and in stem cell collection for sickle cell gene therapy, neither of which is an approved use.
How does Motixafortide (APHEXDA) work?
What it does in the body, and where.Stem cells stay in the marrow because a chemokine called CXCL12 holds them there through CXCR4. Break that grip and they spill into the blood, where a machine can collect them.
- 01Filgrastim goes first, for four daysIt expands the stem cell pool and begins loosening it. On its own that approach often collects too little.
- 02One injection blocks CXCR4 for daysReceptor occupancy runs past 72 hours, far longer than the drug itself lasts in blood. That mismatch is the design, not a flaw.
- 03Stem cells let go of the nicheHematopoietic stem and progenitor cells detach from the marrow and circulate, where they can be filtered out.
- 04Apheresis 10 to 14 hours laterCD34+ cells are collected from the blood and frozen for reinfusion after high-dose chemotherapy. The label's goal is 6 million cells per kg.
A schematic of the compound's mechanism, not a diagnostic image.
What else is it used for?
Each group carries its own evidence level.Approved means regulators have cleared it for that purpose. Investigational means it is in trials. Early research means it is being studied and is not established.
GENESIS, and why it has two sets of numbers
FDA-approved- RegulatoryFDA label, central laboratory: 67.5% of 80 patients on motixafortide plus filgrastim collected at least 6 million CD34+ cells per kg within two apheresis procedures, against 9.5% of 42 on placebo plus filgrastim. Adjusted difference 56.8%, p below 0.0001.
- Human trialNature Medicine, local laboratory, intention-to-treat: 92.5% against 26.2% for that same endpoint, odds ratio 53.3, 95% CI 14.12 to 201.33, p below 0.0001. The secondary endpoint, the same collection goal in a single apheresis, was 88.8% against 9.5%, odds ratio 118.0.
- RegulatoryThe label resolves which is the regulatory finding: it states that the trial's cell counts were performed by central and local laboratories and that central laboratory assessments were used for the efficacy results. Neither 92.5% nor 26.2% appears in the label.
- RegulatoryLabel figures for the single-session endpoints, also central laboratory: 6 million CD34+ cells per kg in one apheresis in 63.8% against 2.4%, and 2 million per kg in one apheresis in 87.5% against 38.1%. Median fold increase in circulating CD34+ count 6.7 against 1.5.
- Human trialRegistry record: 180 participants enrolled across 18 sites in Germany, Hungary, Italy, Spain and the United States, with results first posted November 7, 2023. The 122 figure in the paper and the label is the randomized part of a two-part study.
Safety as the label and the paper describe it
FDA-approved- RegulatoryInjection site reactions in 73% against 5%, injection site pain in 53% against 5%, pruritus in 38% against 0%, flushing in 33% against 0%. These are the label's all-grade rates in the 92-patient safety population.
- Human trialGrade 3 or higher treatment-emergent events in 27.5% against 4.8%, with no grade 4 events and no deaths during the mobilization period, per the trial publication.
- RegulatoryThe premedication requirement is unusual for a mobilizing agent and is the clearest signal of how reactive this peptide is: an H1 antihistamine, an H2 blocker and a leukotriene inhibitor 30 to 60 minutes before each dose, an analgesic recommended, and one hour of observation after.
What the market actually shows
FDA-approved- RegulatoryReported APHEXDA sales fell through 2026: $6.7 million for full-year 2025, $2.7 million in the first quarter of 2026, and $1.6 million in the second quarter. The royalty to the developer tracked them at $1.2 million, $0.5 million and $0.3 million. A drug can be approved, marketed and clinically useful and still sell this little.
- RegulatoryThe $0.3 million royalty on $1.6 million of sales works out to 18.75%, sitting at the bottom of the 18% to 23% band disclosed in the license agreement.
Where else it is being tested
Investigational- Human trialMetastatic pancreatic adenocarcinoma: phase 2, 118 participants estimated, motixafortide with cemiplimab, gemcitabine and nab-paclitaxel. Sponsored by Columbia University with Regeneron and the developer as collaborators. Active and not recruiting, overall response rate at 16 weeks as the primary outcome, primary completion estimated June 2028. Efficacy in pancreatic cancer is not established.
- human-earlySickle cell gene therapy: a phase 1 study of motixafortide alone and with natalizumab for CD34+ cell collection, final results presented at a hematology meeting in December 2025. The rationale is that gene therapy needs far more cells than a transplant does.
The 92.5% figure. It is real, it is from the published phase 3 paper, and it is the number that traveled: it is the paper's headline result for the primary endpoint. But it is the local laboratory count, and the FDA's efficacy section for the same endpoint in the same trial says 67.5%, from the central laboratory. A second trap is the trial size. The registry records 180 participants enrolled, because GENESIS had an open-label lead-in part before the randomized part; the 122 figure everyone cites is the randomized part alone, and the label's safety table rests on 92 patients who received the drug. A third is the commercial picture, where sales are often attributed to the original developer. It licensed the drug away in November 2024 and now books royalties rather than product revenue.
The published record
Papers about Motixafortide (APHEXDA) · this compound only
Searched: motixafortide, APHEXDA, BL-8040, BKT140, 4F-benzoyl-TN14003, TN14003.
Left out: plerixafor (THE critical rejection. The OTHER CXCR4 antagonist used for exactly this job, a small molecule, not a peptide, and NOT on this site. Every clinical discussion of motixafortide compares the two, so a loose query returns plerixafor's far larger literature and risks importing its dosing, its half-life and its adverse event profile onto a peptide card); Mozobil (plerixafor's brand name, and the name a reader is more likely to have heard); CXCR4 (the receptor term. Returns the entire chemokine receptor literature including HIV entry and oncology, none of it about this drug); CXCL12 (the natural ligand, also called SDF-1, whose literature is larger than the drug's by orders of magnitude); filgrastim (the drug it is GIVEN WITH, always, and never without. A combination search returns filgrastim's own enormous literature and both arms of GENESIS used it); G-CSF (the class term for the above, with the same problem); GENESIS (the phase 3 trial name, which collides with unrelated trials and studies of that name across several fields); stem cell mobilization (the indication phrase, returning transplant literature spanning decades); multiple myeloma (the disease, one of the most published cancers there is); Chemo4METPANC (the pancreatic phase 2's name. Useful but narrow, and the trial is Columbia-sponsored with two collaborators, so sponsor-based searching misattributes it); Gamida Cell (the current US label holder, whose own literature is about OMISIRGE, a cell therapy, not a peptide); Ayrmid (the licensee's parent, a holding company spanning an unrelated portfolio); BioLineRx (the original developer, which also has GLIX1 in glioblastoma and formerly AGI-134, neither related); natalizumab (the antibody combined with it in the sickle cell gene therapy study. A large multiple sclerosis literature).
37 papers tagged human · 19 not reviews or lab · 10 clinical trial publications
How the compound was given: 8 injected · 4 by mouth or rectal · 35 route not stated in the abstract
Read from each paper's indexing terms and abstract, by rule. A paper that mentions a cream studied a cream, not necessarily the product being sold.
Furthest stage reached: Approved in the US
Counted from Europe PMC indexing, not read by a person; a human tag can land on reviews, commentary or lab papers; clinical trial publications is the narrower count. A preprint and its journal version count once. 47 papers and 0 trials held here.
At a glance
Four things to know about Motixafortide (APHEXDA)
Evidence
FDA-approved
Category
Immune support
Common vial
62 mg
Half-life
About 2 hours in plasma, against more than 72 hours of receptor occupancy
How much, and do you cycle it?
The range, and whether you take breaks.1.25 mg/kg, 10 to 14 hours before the first apheresis, once per mobilization, with a second dose allowed before a third apheresis. One dose per collection, not a course FDA label
The drug exists to make one apheresis session succeed. There is no ongoing schedule, no titration and nothing to cycle.
How long does it stay in the body?
The half-life, and where the figure comes from.About 2 hours in plasma, against more than 72 hours of receptor occupancyFDA label
The label gives an effective plasma half-life of about 2 hours, a Tmax of 0.25 to 1.17 hours, apparent total clearance of 46.5 L/h and a central compartment volume of 27 L, with more than 99% plasma protein binding. The gap between a two-hour half-life and a dose given 10 to 14 hours ahead of collection is the point of the molecule: what lasts is receptor occupancy, not drug in the blood. No human mass balance study was done; the drug is degraded to small peptides and amino acids.
Source: FDA prescribing information for APHEXDA, section 12.3
You can compare this against the whole library to see where it sits.
How is it given?
Dosing context and source notes.Given in hospital from a prepared product, so there is nothing to mix.
Label dose: 1.25 mg/kg per per-kg. FDA prescribing information, sections 2.1 and 2.2
A single 1.25 mg/kg subcutaneous dose by actual body weight, 10 to 14 hours before the first apheresis, after four days of filgrastim at 10 mcg/kg daily. A second dose may be given 10 to 14 hours before a third apheresis if one is needed. No injection may exceed 2 mL, so larger doses are split across syringes and sites.
How do you store it?
Before mixing, and after.Follow the label that came with it.
This is an approved product, and its own label sets how long it keeps once mixed. Those windows vary enormously between products, from use-immediately to about a month, so the label is the only thing worth following here. Vials of the same compound sold by peptide vendors arrive with no label and no established holding time.
What are the side effects and cautions?
Known cautions and warnings for this compound.Solubility, computed: 71% charged residues, 29% hydrophobic. Water usually sufficient.
A first-pass rule from peptide manufacturers' guidance. It does not account for disulfide bridges, salt form or how the powder was dried, and it predicts what should be easy rather than what will happen in your vial.
Who should avoid it
- Any use outside a transplant center, since the premedication regimen and the hour of post-dose observation exist because anaphylaxis has happened
- Self-administration of any kind: this is a weight-based hospital product given once in a planned collection sequence
- Pregnancy, on the embryo-fetal toxicity warning
- Reading the 92.5% figure as the regulator's finding when it is not
Stop and get help
- Hives, flushing with lightheadedness, throat tightness, wheezing or faintness during or after the injection. These are the hypersensitivity signs the premedication is meant to blunt
- Any decision here belongs to the transplant team, not the patient: the drug is given once, under observation, as part of a scheduled collection
Common effects
- Injection site pain, redness and itching in the majority of patients, transient and graded 1 or 2 in the trial
- Flushing and itching away from the injection site
- Back pain in 21% against 17% on placebo and paresthesia in 19% against 17%, both close to the placebo rate
- Hypokalemia in 15% against 12% and nausea in 14% against 12%
Where this page says nothing is established, that means nobody has studied it, not that a compound is safe.
Is Motixafortide (APHEXDA) approved?
And where the numbers on this page come from.Yes. Motixafortide (APHEXDA) is FDA approved and available on prescription in the United States.
- Approval statusFDA-approved
- RouteUnder the skin, one dose before apheresis, at a transplant center
- Checked11 November 2026
- DoseFrom the drug label. The manufacturer tested this range and a regulator reviewed it.
- Vial sizeFrom what vendors typically list; not a recommendation.
- SourcesFDA prescribing information for APHEXDA (motixafortide), NDA 217159, via DailyMedCrees ZD et al., Motixafortide and G-CSF to mobilize hematopoietic stem cells for autologous transplantation in multiple myeloma: a randomized phase 3 trial. Nature Medicine 2023;29(4):869-879. PMID 37069359, DOI 10.1038/s41591-023-02273-zClinicalTrials.gov, GENESIS phase 3 record NCT03246529ClinicalTrials.gov, Chemo4METPANC phase 2 record NCT04543071BioLineRx and Ayrmid exclusive license agreement, November 21, 2024BioLineRx second quarter 2026 results, Form 6-K exhibit filed with the SECBioLineRx second quarter 2026 results announcementFDA Global Substance Registration System record for motixafortide
What has happened recently
Approved by the FDA in September 2023 under NDA 217159. The current prescribing information is held by Gamida Cell Inc., not the original developer: on November 20, 2024 BioLineRx granted Ayrmid, Gamida Cell's parent, an exclusive license to develop and commercialize the drug in all indications except solid tumors and all territories except Asia, for $10 million upfront, up to $87 million in commercial milestones and royalties of 18% to 23% on net sales. The developer shut down its own US commercial operations in the fourth quarter of 2024 and retains solid tumors, including pancreatic cancer. Asia rights sit with a separate licensee. One widely repeated claim needs correcting: the developer's own filings report APHEXDA sales as the licensee's, not its own, and report its share as royalty revenue.
You can compare this against the whole library, or read how to check a seller.
What else is like Motixafortide (APHEXDA)?
Others in Immune support, side by side.If you're weighing Motixafortide (APHEXDA), it's worth reading Omiganan (MBI-226), P140 (forigerimod), and Pegcetacoplan (Empaveli, Syfovre) in the same class.
The same endpoint, counted twice: 92.5% in the journal, 67.5% on the label.
- Dose
- 1.25 mg/kg, 10 to 14 hours before the first apheresis
- How often
- Once per mobilization, with a second dose allowed before a third apheresis
A short, tryptophan- and arginine-rich peptide gel that disrupts microbial membranes. Two catheter-site phase 3 trials missed their primary endpoints, and its two rosacea phase 3 trials split, one positive and one negative.
- Dose
- 1% gel at catheter sites, 1.6% gel on rosacea (phase 3 trials)
- How often
- Each dressing change (catheters) or once daily (rosacea)
An experimental lupus peptide whose pivotal trial missed its endpoint in 2018.
- Dose
- 200 mcg every 4 weeks (every trial to date)
- How often
- Every 4 weeks
Blocks complement at its central protein, C3. Twice-weekly infusions for PNH and kidney disease; eye injections every one to two months for geographic atrophy.
- Dose
- 1,080 mg twice weekly under the skin; 15 mg into the eye every 25 to 60 days
- How often
- Twice weekly (Empaveli); every 25 to 60 days (Syfovre)
Guides for this compound
- How to Reconstitute a Peptide
Mix peptide powder with bacteriostatic water, step by step.
- How to Read an Insulin Syringe
Read the units, convert to millilitres, and draw the exact dose.
- mcg vs mg: The Peptide Dosing Mistake to Avoid
Your vial says mg and your protocol says mcg. Here is the conversion and how to check it.
Sources
- FDA prescribing information for APHEXDA (motixafortide), NDA 217159, via DailyMed
- Crees ZD et al., Motixafortide and G-CSF to mobilize hematopoietic stem cells for autologous transplantation in multiple myeloma: a randomized phase 3 trial. Nature Medicine 2023;29(4):869-879. PMID 37069359, DOI 10.1038/s41591-023-02273-z
- ClinicalTrials.gov, GENESIS phase 3 record NCT03246529
- ClinicalTrials.gov, Chemo4METPANC phase 2 record NCT04543071
- BioLineRx and Ayrmid exclusive license agreement, November 21, 2024
- BioLineRx second quarter 2026 results, Form 6-K exhibit filed with the SEC
- BioLineRx second quarter 2026 results announcement
- FDA Global Substance Registration System record for motixafortide
Beyond these, 47 records are indexed for this compound. Browse the records