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Rockwell Medical files IND to investigate Triferic for orphan disease

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Rockwell Medical Inc. has filed an investigational new drug application to investigate Triferic as a potential treatment for patients with a rare anemia condition called iron-refractory, iron-deficiency anemia (IRIDA).  Less than 100 patients from families of different ethnic origin have been described with IRIDA to date, according to Rockwell.

"IRIDA patients currently have no available treatment options, and we believe that Triferic can address this unmet need and offer patients a form of iron which can treat their anemia and improve their well-being," said Raymond D. Pratt, Chief Medical Officer of Rockwell. "We plan to begin conducting studies with Triferic for this orphan disease this year. Rockwell is committed to exploring the unique properties of Triferic to benefit patients not only with renal disease but also in conditions where elevated hepcidin leads to functional iron deficiency."

IRIDA is an orphan disease caused by a mutation in a gene controlling the expression of hepcidin, which leads to high levels of hepcidin. It is a life-long affliction that manifests in early childhood. Patients with IRIDA have high hepcidin levels and cannot absorb oral iron. Hepcidin blocks the iron transporter responsible for the release of iron from cells of the reticuloendothelial system (RES).

IRIDA patients also do not respond to IV iron preparations, which have to be processed in the RES.  Triferic has a unique mechanism of action which bypasses the RES processing step and donates iron directly to transferrin for use in erythropoiesis.