Drug intelligence / Profile preview

petadeferitrin

Development stage
Phase 2
Lead developer
Pharmacosmos
Modality
Small Molecules
Administration
Oral
01

Overview

Petadeferitrin (SP-420) is an orally bioavailable small molecule iron chelator of the desferrithiocin class. It is designed to bind and remove excess iron from the body by chelating free iron and promoting its excretion, thereby preventing iron accumulation and tissue damage associated with conditions of iron overload. The drug is primarily under development for transfusion-dependent thalassemia (including both β-thalassemia and α-thalassemia), where regular blood transfusions can lead to dangerous levels of systemic iron. Petadeferitrin is administered as oral capsules several times per week. Clinical trials are ongoing to establish its safety profile, dose-response relationship, and efficacy in reducing total body and organ-specific (especially hepatic) iron burden in affected patients. Pharmacosmos is the main developer.

Other names
911714-45-9WBX54NZ436WBX-54NZ436WBX 54NZ436Sp420Sp-420Sp 420
02

Targets

Fe3+ (Ferric ion (Fe3+) and polyvalent metal cations)

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