Disc Medicine, Inc. (NASDAQ: IRON), a company focused on developing treatments for serious hematologic diseases, announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to DISC-3405 for treating polycythemia vera (PV) patients.

Disc, stated, "Orphan drug designation is a significant milestone for DISC-3405 in addressing PV, a rare disease with limited treatment options." He added, "We anticipate sharing preliminary data from our Phase 1 trial of DISC-3405 in healthy volunteers in the first half of 2024."

Orphan Drug Designation by the FDA is given to investigational therapies for rare medical conditions affecting fewer than 200,000 individuals in the United States. This designation provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical expenses, exemptions from certain FDA fees, and seven years of marketing exclusivity following approval.

DISC-3405 is an investigational anti-TMPRSS6 monoclonal antibody designed to increase hepcidin production and suppress serum iron levels. Disc acquired DISC-3405 from Mabwell Therapeutics in January 2023. Preclinical studies have shown promising results in animal models of beta-thalassemia and polycythemia vera. Disc initiated a Phase 1 study of DISC-3405 in healthy volunteers in October 2023, with plans to develop it initially for polycythemia vera and other hematologic disorders.

DISC-3405 is an investigational compound and has not been approved for therapeutic use in any jurisdiction worldwide.

Polycythemia vera (PV) is a chronic and rare myeloproliferative neoplasm characterized by abnormal proliferation of red blood cells. It affects approximately 150,000 patients in the U.S. and has a similar prevalence in Europe. The overproduction of red blood cells leads to increased risks of cardiovascular and thromboembolic events, as well as complications such as enlarged spleen and symptoms including fatigue and difficulty concentrating. Current treatments involve managing red blood cell count and symptoms through phlebotomy or cytoreductive agents.

 

Source: globenewswire.com

Report Abuse