SRF231 is granted orphan designation status for the treatment of multiple myeloma

On 30 July 2018, the US Food and Drug Administration (FDA) included SRF231 (anti-CD47) among the list of orphan drugs to be used for treating multiple myeloma (MM) patients (pts).

SRF231 is a fully humanized monoclonal antibody, which targets the transmembrane protein CD47. Although CD47 is found at very low levels in most normal cell types, it is highly expressed by many different types of tumor cells, including myeloma cells. CD47 normally binds to the Signal Regulatory Protein Alpha (SIRPA), preventing cell phagocytosis. SRF231 binding to CD47 inhibits the link between CD47 and SIRPA, promoting phagocytosis and removal of myeloma cells.

Currently, the safety and maximum tolerated dose of SFR231 are being examined in a phase I clinical trial in patients with advanced solid and hematologic cancers.

SRF231’s orphan drug designation will support further investigations carried out by the drug research company Surface Oncology on the treating potential of this drug for MM pts.

References

1. FDA Orphan Drug Designation and Approvals
2. Rare DR (Rare Disease Report) online. Multiple Myeloma Treatment, SRF231, Receives Orphan Drug Designation by FDA. https://www.raredr.com/news/multiple-myeloma-treatment-srf231-receives-orphan-drug-designation-by-fda. [Assessed July 31 2018].