BioLineRx's BL-8040 gets FDA Orphan Drug Designation for stem cell mobilization treatment

BioLineRxJan 23 2014

BioLineRx (NASDAQ: BLRX; TASE: BLRX), a clinical-stage biopharmaceutical company dedicated to identifying, in-licensing and developing promising therapeutic candidates, announced today that it has received notice from the U.S. Food & Drug Administration (FDA) confirming an Orphan Drug Designation of BL-8040 as a treatment for stem cell mobilization, in addition to the Orphan Drug Designation previously granted to BL-8040 as a treatment for Acute Myeloid Leukemia (AML).

Orphan Drug Designation is granted to therapeutics intended to treat rare diseases that affect not more than 200,000 people in the United States. Orphan Drug Designation entitles the sponsor to a seven-year marketing exclusivity period, clinical protocol assistance with the FDA, as well as federal grants and tax credits.

The Orphan Drug Designation was granted for use of BL-8040, in combination with granulocyte colony-stimulating factor (G-CSF), to mobilize human stem cells from the bone marrow to the peripheral blood for collection for autologous or allogeneic (donor-based) transplantation.

"We are very pleased to have our second Orphan Drug Designation for BL-8040, in this case for stem cell mobilization treatment, after already having received the designation last year for the treatment of acute myeloid leukemia," stated Dr. Kinneret Savitsky, Chief Executive Officer of BioLineRx. "This is in line with our 2014 development plan for BL-8040, which includes clinical studies for both AML and stem cell mobilization. BL-8040 was already shown to be highly effective in mobilizing stem cells in combination with G-CSF in a Phase 1/2 study in multiple myeloma patients, and initial results of BL-8040's Phase 2 study for AML patients indicate that BL-8040 on a stand-alone basis triggers substantial mobilization of cancer cells from the bone marrow to the peripheral blood. We are eagerly looking forward to the results of the clinical trials in both indications expected in the second half of this year."