Drug intelligence / Profile preview

hemin

Development stage
Approved
Lead developer
Recordati
Modality
Small Molecules
Administration
Intravenous
01

Overview

Hemin (trade name Panhematin) is a sterile, lyophilized form of protoporphyrin IX iron (III) chloride, derived from processed red blood cells. It is indicated for the treatment of recurrent attacks of acute intermittent porphyria (AIP) related to the menstrual cycle, as well as other acute porphyrias like variegate porphyria and hereditary coproporphyria. The drug's primary mechanism of action involves the exogenous replacement of heme, which triggers negative feedback inhibition of delta-aminolevulinic acid (ALA) synthase, the rate-limiting enzyme in the heme biosynthetic pathway. By suppressing this enzyme, hemin reduces the overproduction and systemic accumulation of neurotoxic porphyrin precursors, specifically ALA and porphobilinogen (PBG). Approved by the FDA in 1983, it was the first orphan drug to reach the market in the United States.

Brand names
Panhematin
Other names
ferriheme chloridehematinprotoporphyrin IX iron(III) chloridechloro(protoporphyrinato)iron(III)
02

Targets

ALAS1 (Delta-aminolevulinate synthase)

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