The US Food and Drug Administration (FDA) has granted orphan drug designation for clinical-stage biopharmaceutical company Disc Medicine’s investigational therapy, DISC-3405, for the treatment of polycythemia vera (PV).
This designation is a significant milestone for the company as it aims to develop treatments for rare hematologic conditions.
DISC-3405, an anti-Transmembrane Serine Protease 6 (TMPRSS6) monoclonal antibody, is designed to modulate iron homeostasis by boosting hepcidin production and lowering serum iron levels.
According to preclinical studies, the drug has shown promising results in animal models of beta-thalassemia and PV.
In January last year, Disc in-licensed DISC-3405 from Mabwell Therapeutics.
Subsequently, the company initiated a Phase I clinical trial of DISC-3405 in healthy volunteers in October, with the goal of eventually treating not only PV but also other hematologic conditions.
The orphan drug designation is awarded to drugs that act on conditions affecting fewer than 200,000 people in the US.
The status offers various development incentives, such as trial tax credits, FDA fee exemptions, and seven years of marketing exclusivity following approval.
DISC-3405 is still an investigational agent and has not been approved for therapeutic use in any jurisdiction.
PV is a chronic, rare myeloproliferative neoplasm characterised by the overproduction of red blood cells.
Disc Medicine focuses on the discovery, development, and commercialisation of new treatments for patients with haematologic ailments.
The company is developing a portfolio of therapeutic candidates that target essential biological pathways in red blood cell biology, focusing on heme biosynthesis and iron homeostasis.
Disc Medicine president and CEO John Quisel said: “Orphan drug designation is an important milestone that highlights the potential of DISC-3405 in PV, a rare disease with few treatment options.
“We look forward to sharing initial data from our ongoing Phase I trial of DISC-3405 in healthy volunteers in the first half of 2024.”