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Heme arginate (Normosang)

University of Edinburgh · Phase 3 active Small molecule

Heme arginate replenishes depleted heme pools and inhibits delta-aminolevulinic acid synthase (ALAS1) to reduce the toxic accumulation of porphyrin precursors in acute porphyria attacks.

Heme arginate replenishes depleted heme pools and inhibits delta-aminolevulinic acid synthase (ALAS1) to reduce the toxic accumulation of porphyrin precursors in acute porphyria attacks. Used for Acute intermittent porphyria (acute attack), Variegate porphyria (acute attack), Hereditary coproporphyria (acute attack).

At a glance

Generic nameHeme arginate (Normosang)
Also known asHeme Arginate, Hemin
SponsorUniversity of Edinburgh
Drug classHeme supplement
TargetDelta-aminolevulinic acid synthase 1 (ALAS1)
ModalitySmall molecule
Therapeutic areaMetabolic/Genetic Disorders
PhasePhase 3

Mechanism of action

In acute intermittent porphyria and other acute porphyrias, overproduction of toxic porphyrin precursors (ALA and PBG) causes severe neurovisceral symptoms. Heme arginate provides exogenous heme, which suppresses ALAS1 activity through negative feedback, thereby reducing precursor synthesis and alleviating acute attack symptoms. This mechanism addresses the underlying biochemical defect rather than treating symptoms alone.

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

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