Is there any evidence of motixafortide use in pediatric patients?

Comment by InpharmD Researcher

At the present time, we were unable to identify any published clinical trials, case studies/series, or pharmacokinetic studies evaluating motixafortide in pediatric patients. However, limited unpublished data derived from company press releases describe real-world use of motixafortide, with the overall patient cohorts including individuals as young as 14 years; caution is warranted, as the data are not peer-reviewed, and specific outcomes for pediatric and adolescent patients remain unknown.

Pubmed: motixafortide and pediatric (0 relevant results)

Background

According to recent press releases from Ayrmid, a biopharmaceutical company that holds the licensed rights to motixafortide, the drug has been used in real‑world practice to mobilize hematopoietic stem cells (HSCs) in patients with sickle cell disease. The reported patient cohorts ranged from 14 to 50 years of age. These observations are from an unpublished, non-peer-reviewed context, and no detailed efficacy or safety outcomes were provided specifically for the adolescent or pediatric patients. Furthermore, the reports do not delineate the specific number of patients aged 14-18 years. [1], [2]

References: [1] Ayrmid Reports Additional New Real‑World Data on Motixafortide for Stem Cell Mobilization in Sickle Cell Disease and Beta‑Thalassemia. BioSpace. February 4, 2026. Accessed February 27, 2026. https://www.biospace.com/press-releases/ayrmid-reports-additional-new-real-world-data-on-motixafortide-for-stem-cell-mobilization-in-sickle-cell-disease-and-beta-thalassemia
[2] Ayrmid Ltd. Ayrmid reports promising new real‑world data on motixafortide for stem cell mobilization in sickle cell disease. Press release. Dec 6, 2025. Accessed February 27, 2026. http://gamida-cell.com/press_release/ayrmid-reports-promising-new-real-world-data-on-motixafortide-for-stem-cell-mobilization-in-sickle-cell-disease/