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Disc Medicine receives US FDA orphan drug designation for DISC-3405 to treat polycythemia vera

Watertown, Massachusetts
Monday, February 12, 2024, 17:00 Hrs  [IST]

Disc Medicine, Inc., a clinical-stage biopharmaceutical company, announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to DISC-3405 for the treatment of patients with polycythemia vera (PV).

“Orphan drug designation is an important milestone that highlights the potential of DISC-3405 in PV, a rare disease with few treatment options,” said John Quisel, J.D., Ph.D., president and chief executive officer of Disc. “We look forward to sharing initial data from our ongoing phase 1 trial of DISC-3405 in healthy volunteers in the first half of 2024.”

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.

DISC-3405 is an investigational, anti-TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) monoclonal antibody designed to increase hepcidin production and suppress serum iron. Disc in-licensed DISC-3405 from Mabwell Therapeutics in January 2023. 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 October 2023 and plans to develop DISC-3405 initially as a treatment for polycythemia vera as well as other hematologic conditions.

DISC-3405 is an investigational agent and is not approved for use as a therapy in any jurisdiction worldwide.

Polycythemia vera (PV) is a chronic and rare myeloproliferative neoplasm characterized by the abnormal proliferation of red blood cells. PV affects approximately 150,000 patients in the US and has a similar prevalence in Europe. The overproduction of red blood cells alters the viscosity of blood, causing it to thicken and placing patients at an elevated risk of cardiovascular and thromboembolic events, such as heart attack and stroke. Patients also experience complications such as enlarged spleen and symptoms of their disease such as fatigue, pruritis, difficulty concentrating and others. Current therapy involves phlebotomy to physically remove blood and iron to limit erythropoiesis or treatment with cytoreductive agents, with the goal of reducing red blood cell count and managing symptoms.

Disc Medicine is a clinical-stage biopharmaceutical company committed to discovering, developing, and commercializing novel treatments for patients who suffer from serious hematologic diseases.


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