Israel-based clinical-stage biopharmaceutical firm BioLineRx has received notice from the US Food & Drug Administration (FDA) confirming an Orphan Drug Designation for BL-8040 as a treatment for stem cell mobilisation.
The FDA has previously granted orphan drug designation to BL-8040 for the treatment for acute myeloid leukemia (AML).
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BL-8040 is a clinical-stage drug candidate indicated for the treatment of AML, as well as other haematological indications.
The orphan drug status was granted for use of BL-8040, in combination with granulocyte colony-stimulating factor (G-CSF), to mobilise human stem cells from the bone marrow to the peripheral blood for collection for autologous or allogeneic transplantation.
BioLineRx chief executive officer Kinneret Savitsky said: "This is in line with our 2014 development plan for BL-8040, which includes clinical studies for both AML and stem cell mobilisation."
In the Phase I/II trial carried out in multiple myeloma patients, BL-8040 has already shown to be highly effective in mobilising stem cells in combination with G-CSF.
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By GlobalDataInitial results of BL-8040’s Phase II trial for AML patients show that, BL-8040 on a stand-alone basis triggers substantial mobilisation of cancer cells from the bone marrow to the peripheral blood.
The company expects to release the results of the clinical trials in both indications in the second half of 2014.
BL-8040 is a short peptide that functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival.
Image: Stem cells are mobilized from the bone marrow using granulocyte colony-stimulating factor (G-CSF). Photo: courtesy of freedigitalphotos.net.
