FDA Grants Fast Track Designation for Disc Medicine’s Treatment for Polycythemia Vera

Rare Daily Staff

The U.S, Food and Drug Administration has granted Fast Track designation to Disc Medicine’s MWTX-003 for the treatment of patients with polycythemia vera.

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 U.S. 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.

MWTX-003, also known as DISC-3405, is an experimental, anti-transmembrane serine protease 6, also known as Matriptase-2, monoclonal antibody designed to increase hepcidin production and suppress serum iron, that Disc in-licensed from Mabwell Therapeutics in January 2023. Preclinical studies of MWTX-003 have demonstrated an increase in hepcidin production and suppression of serum iron levels in animal models of beta-thalassemia and polycythemia vera. The IND was accepted in November 2022 and Disc plans to initiate a phase 1 study of MWTX-003 in healthy volunteers during the second half of 2023. Disc plans to develop MWTX-003 initially as a treatment for polycythemia vera as well as other hematologic conditions.

Fast Track is a process designed by the FDA to facilitate the development and expedite the review of investigational drugs intended to treat serious conditions and for which nonclinical or clinical data demonstrate the potential to address unmet medical need. A therapeutic candidate that receives Fast Track designation may be eligible for more frequent interactions with the FDA to discuss the candidate’s development plan. Therapeutic candidates with Fast Track designation may also be eligible for priority review and accelerated approval if supported by clinical data.