NOXXON Pharma announced that the FDA has granted Orphan Drug designation to olaptesed pegol (NOX-A12) for the treatment of glioblastoma in conjunction with radiotherapy.

Olaptesed pegol is a PEGylated mirror-image (L-) oligonucleotide that binds and neutralizes the chemokine CXCL12/SDF-1, preventing interaction with its receptors CXCR4 & CXCR7. The CXCL12/CXCR4/CXCR7 pathway directly affects tumor progression by controlling cancer cell survival, proliferation, and migration. It also has indirect effects through angiogenesis and recruitment of immune cells. Neutralization of CXCL12 with olaptesed pegol results in increased susceptibility of tumors to the effects of chemotherapy and radiotherapy.

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Currently, olaptesed pegol is under investigation in Phase 2a studies in two hematological cancers, multiple myeloma (MM) and chronic lymphocytic leukemia (CLL).

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