Acute intermittent porphyria (AIP) – one of the hepatic porphyrias – is a rare, inherited genetic condition caused by a partial deficiency of the enzyme porphobilinogen (PBG) deaminase,1 which can disrupt normal heme production in the body.2
Exposure to one or more precipitating factors, such as endogenous steroid hormones or certain prescription drugs, may increase the body’s need for heme.3 The partial deficiency of PBG deaminase, the third enzyme in the multi-step heme biosynthetic pathway, can become rate-limiting in the production of heme.4 This can cause neurotoxic porphyrin precursors to accumulate as the body continues to signal for additional heme.4
If unrecognized and untreated, an AIP attack can progress to neurologic damage or death due to cardiac arrhythmia or paralysis.3
The most common symptom is severe abdominal pain, which is neuropathic in origin.3 Other symptoms that can increase suspicion of an AIP attack are urine that turns dark or reddish when exposed to light or air and hyponatremia.1 Untreated attacks can cause serious consequences both during and after attacks.3
A PBG urine test should be done at or near the time of AIP symptoms. Urinary PBG levels are substantially increased (20–200 mg/L) in patients with acute attacks of AIP.3
If PBG levels are elevated, second-line testing should be done to determine the precise type of acute porphyria. These tests may include δ-aminolevulinic acid (ALA) and porphyrin testing on the same urine sample, as well as testing for plasma and fecal porphyrins and erythrocyte PBG deaminase. Treatment should not be delayed pending these results.3
can be done to determine the disease-causing mutation(s) in the defective gene. Family members can then be tested to determine if they may be at risk of AIP attacks.3
Urine porphyrin levels (mostly uroporphyrin)
markedly increased
Fecal porphyrin levels
normal or slightly increased
Plasma porphyrin levels
normal or slightly increased
Erythrocyte PBG deaminase levels
decreased by ~50%
What is PANHEMATIN?
PANHEMATIN (hemin for injection) is a prescription medicine used to relieve repeated attacks of acute intermittent porphyria related to the menstrual cycle in affected women, after initial carbohydrate therapy is known or suspected to be inadequate.
Limitations of Use
Who should not use PANHEMATIN?
Do not take PANHEMATIN if you are allergic to this drug.
Before starting PANHEMATIN, tell your healthcare provider (HCP) about all your medical conditions, including if you are pregnant or plan to become pregnant, breastfeeding or plan to breastfeed. Tell your HCP about all the medicines you take, including any prescription and over-the-counter medicines, vitamins, or herbal supplements.
What are the possible side effects of PANHEMATIN?
PANHEMATIN may cause side effects including:
The most common side effects of PANHEMATIN include headache, fever, infusion site reactions, and vein inflammation.
These are not all the possible side effects of PANHEMATIN. Call your HCP for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Call 1-800-FDA-1088 or visit www.fda.gov/medwatch.
When taking PANHEMATIN, do not take drugs such as estrogens (e.g., oral contraceptives), barbiturates (drugs that help with sleep and used to treat epilepsy) or steroids (body hormone-like drugs), because such drugs can trigger an attack or make an attack worse.
PANHEMATIN® (hemin for injection), for intravenous infusion only, is available as powder for reconstitution in 350 mg vials.
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