Evidence review: Open Peptide Project (verified against primary PubMed, ClinicalTrials.gov & FDA records) · Last checked 2026-10-27. ← Back to Evidence Library
Motixafortide (Aphexda, BL-8040): what the evidence says
At a glance
What it is: a synthetic cyclic peptide that blocks the CXCR4 receptor, releasing blood-forming stem cells from the bone marrow into the blood so they can be collected. Approved as Aphexda (Sep 2023), together with filgrastim (G-CSF), for stem cell collection before autologous transplant in multiple myeloma. R1, R2
What the evidence says: Human in GENESIS (122 adults with myeloma), 92.5% collected the target number of stem cells within two collection sessions with motixafortide plus G-CSF vs 26.2% with placebo plus G-CSF. R3
Biggest risks: label warnings: anaphylactic shock and other allergic reactions (all patients need premedication), injection-site reactions, high white cell counts, and possible release of tumor cells; it should not be used in leukemia. R2
Rating
| Human Human evidence | Strong: phase 3 RCT (n = 122) R3 |
| Animal Animal / lab evidence | Not reviewed |
| Regulatory | FDA-approved R1 · not on FDA's 503A or 503B bulk-substance category lists and not on FDA's compounding safety-risk page R4, R5, R6 · not named by WADA R7 |
| Established human dose | Yes, label dose (see Study details) |
Evidence verdict: Stem cell mobilization before transplant in multiple myeloma: Strong (phase 3 RCT) · Other uses: No human data reviewed
This page reports what studies used. It is not dosing advice.
Legend: HUMAN data · ANIMAL / LAB data · UNKNOWN · UNVERIFIED: not yet confirmed against a primary source
About the evidence: one placebo-controlled sponsor RCT with a large effect on a collection endpoint, not on survival.
What is it?
Related pages: romiplostim, bortezomib, carfilzomib.
Does it work?
- Human GENESIS: target collected within two sessions in 92.5% vs 26.2%; within one session in 88.8% vs 9.5%. R3, R8
- Human Injection-site pain (50%), redness and itching were the most common side effects. R3
Is it safe?
- Warnings: anaphylactic shock and hypersensitivity, injection-site reactions, tumor cell mobilization (not for leukemia), leukocytosis. R2
- Most common: injection-site reactions, itching, flushing, back pain. R2
Gray-market vials: extra risks
A gray-market vial has no quality control and no guarantee of identity, strength or sterility. We test retail vials blind under the Open Peptide Standard; see /tests/. A passing test covers one batch on one date and does not mean the compound is safe.
Is it legal?
- US: FDA-approved, prescription (Aphexda), used only in transplant centers with G-CSF. R1, R2
- Compounding: not on FDA's 503A or 503B bulk-substance category lists and not on FDA's compounding safety-risk page. R4, R5, R6
- Sport: not named on WADA's 2026 Prohibited List. R7
Study details
Study details
These are amounts researchers used, not recommendations.
| Study | People / model | Route | Amount used | Follow-up | Reported | Citation |
|---|---|---|---|---|---|---|
| GENESIS (Crees 2023) | 122 adults with multiple myeloma | SC, with G-CSF | 1.25 mg/kg, per label | Mobilization period | Target collected: 92.5% vs 26.2% (placebo + G-CSF) | PMID 37069359 |
Full reference list (PubMed, CT.gov, FDA and WADA checked Oct 27, 2026)
- R1 Drugs@FDA, Aphexda (motixafortide), application NDA217159 (openFDA drugsfda record checked Oct 27, 2026). https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=NDA217159
- R2 Aphexda prescribing information, label published Oct 19, 2026 (DailyMed, checked Oct 27, 2026). https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f7cdc584-ebf0-a68e-e053-6294a90a9ea9
- R3 Crees ZD, et al. Nat Med. 2023. PMID 37069359
- R4 FDA, 503A bulk drug substances categories list (updated May 14, 2026). https://www.fda.gov/media/94155/download
- R5 FDA, 503B bulk drug substances categories list (updated Mar 21, 2025). https://www.fda.gov/media/94164/download
- R6 FDA, Certain bulk drug substances for use in compounding that may present significant safety risks (content current Apr 22, 2026). https://www.fda.gov/drugs/human-drug-compounding/certain-bulk-drug-substances-use-compounding-may-present-significant-safety-risks
- R7 WADA. The 2026 Prohibited List, full list checked; compound not named. https://www.wada-ama.org/sites/default/files/2025-09/2026list_en_final_clean_september_2025.pdf
- R8 ClinicalTrials.gov NCT03246529, GENESIS, phase 3, active not recruiting, 180 enrolled (CT.gov API v2, checked Oct 27, 2026). https://clinicaltrials.gov/study/NCT03246529
Disclaimer
This page is for education and harm reduction, not medical advice or a recommendation to use Motixafortide (Aphexda, BL-8040). This is a prescription medicine. Use it only under the care of a licensed clinician; do not buy it from research-chemical or gray-market sellers. Report problems to FDA MedWatch (https://www.fda.gov/safety/medwatch).