Orphan Status for Polycythemia Vera Drug

FDA has granted Disc Medicine an orphan drug designation for DISC-3405 for treating patients with polycythemia vera (PV). The investigational, anti-TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) monoclonal antibody is designed to increase hepcidin production and suppress serum iron, according to the company.

Disc Medicine says preclinical studies of DISC-3405 have demonstrated an increase in hepcidin production and suppression of serum iron levels in animal models of beta-thalassemia and polycythemia vera. “Disc initiated a Phase 1 study of DISC-3405 in healthy volunteers in 10/2023 and plans to develop DISC-3405 initially as a treatment for polycythemia vera as well as other hematologic conditions,” it says.

PV is described as a chronic and rare myeloproliferative neoplasm characterized by the abnormal proliferation of red blood cells. It affects about 150,000 patients in the U.S.