Panhematin (hemin for injection)

The hospital treatment for an acute porphyria attack: a black powder made from human red blood cells, mixed and infused into a large vein once a day for 3 to 14 days. It was the first drug approved under the 1983 Orphan Drug Act and remains the only heme product sold in the United States.

During a porphyria attack the liver senses a shortage of heme and drives ALAS1, the first enzyme in the heme pathway, to work harder, which floods the body with ALA and PBG^[1]. Hemin is heme itself, purified from donated red blood cells. Infused into a vein, it tops up the liver's heme supply, and the label says it likely works by inhibiting ALA synthase, which shuts the pathway down and lets ALA and PBG fall back toward normal^[1]. The label is candid that the exact way hematin relieves symptoms has not been worked out, that treatment is not curative, and that symptoms generally return after a course ends, though some people stay in remission for a long time^[1]. Some nerve symptoms improve weeks to months after treatment even when little changes during the infusion days^[1].

The label has no boxed warning. Its main caution is phlebitis, inflammation of the vein, so Panhematin is given through a large arm vein or a central venous catheter, never a small hand vein, and filtered through a 0.45 micron or smaller filter because the dark solution hides undissolved particles^[1]. Because each vial carries iron, people who receive repeated courses can build up iron and ferritin; the label asks doctors to monitor both and consider chelation if they rise^[1]. Hemin has a mild, short-lived blood-thinning effect, so anticoagulants are avoided during treatment^[1]. One patient given an excessive 12.2 mg/kg dose had reversible kidney shutdown, which is why the 6 mg/kg daily ceiling matters; no kidney harm has been seen at recommended doses^[1]. Because it is made from human blood, there is a theoretical risk of passing on viruses or prion disease despite donor screening and viral inactivation^[1]. In the compassionate use study of 130 people, the reactions reported in more than 1% were headache (9.2% of adverse events), fever (4.6%), phlebitis (3.6%), catheter complications (3.6%), cellulitis (1.5%) and rash (1.5%)^[1]. Since marketing there have been reports of low platelets, clotting abnormalities, hemolysis, hypersensitivity including 1 anaphylactoid reaction with circulatory collapse, vein thrombosis including in large central veins, infusion site bleeding or leakage, and iron overload^[1]. The label also tells patients not to take estrogens such as birth control pills, barbiturates or steroid-type hormones, which can trigger or worsen an attack^[1].

Panhematin is for IV infusion only and only by or with doctors experienced in porphyria^[1]. The label requires a confirmed diagnosis first: symptoms of an attack plus a quantitative urine PBG, on a sample kept cold and out of the light^[1]. For a mild attack (mild pain, no vomiting, no paralysis, normal sodium, no seizures) it suggests a trial of glucose while hemin is on its way; for a moderate or severe attack (severe or lasting pain, persistent vomiting, low sodium, seizure, psychosis or neuropathy) it calls for immediate hemin^[1]. The dose is 1 to 4 mg/kg a day for 3 to 14 days, with 3 to 4 mg/kg a day the standard in practice; in severe cases a dose can be repeated no sooner than every 12 hours, and the total must never exceed 6 mg/kg in 24 hours^[1]. Each vial holds the equivalent of 350 mg of hemin as a black powder and is mixed with 48 mL of sterile water right before use, because the solution breaks down quickly; after mixing, 1 mg of hematin is 0.14 mL, so a 70 kg adult at 4 mg/kg receives about 40 mL^[1]. It runs over at least 30 minutes through its own line into a large arm vein or a central catheter, with a 100 mL saline flush afterward; nothing else is mixed into it and leftover solution is discarded^[1]. Recordati says infusions can be done in the hospital or at an infusion center or doctor's office^[2]. Unopened vials are stored at room temperature (20 to 25 C), and the stopper contains natural rubber latex^[1]. Most hospitals do not stock it; the APF advises that the pharmacy request air freight of enough for 5 days as soon as a patient is admitted, since shipping takes at least 24 hours, and Recordati's Panhematin Locator lists facilities that keep it on hand or can get it quickly^[4][2]. The label notes that porphyria experts sometimes reconstitute it with human albumin to reduce phlebitis; directions come from specialists and the APF emergency room kit^[4].

Yes, Hemin for injection (Panhematin) is FDA approved (1983) for the treatment of Acute Hepatic Porphyria.

Recordati says the faster treatment starts, the better the outcome is likely to be, and the label says clinical benefit depends on prompt administration because an attack can progress to nerve damage that Panhematin cannot repair. In the earliest open-label study on the label (Watson, 15 patients) all but 1 had a clear clinical response, most of them rapid, and urine ALA and PBG fell in every patient. Some nerve symptoms improve only weeks to months later.

Hemin irritates veins. The label warns of phlebitis and asks for a large arm vein or a central venous catheter, infusion over at least 30 minutes, a 0.45 micron filter and a 100 mL saline flush. Specialists sometimes mix it with human albumin to reduce vein irritation.

Most hospitals do not stock it. The American Porphyria Foundation advises asking the pharmacy to order enough for 5 days as soon as you are admitted, because shipping takes at least 24 hours; Cencora is the exclusive distributor and ships priority overnight. Recordati's Panhematin Locator on Panhematin.com lists hospitals that keep it or can get it quickly. Carry an emergency letter from your porphyria doctor.

The approved indication is worded for recurrent attacks tied to the menstrual cycle in women, after carbohydrate therapy is inadequate, because that is the group in which the original evidence was gathered. NORD and the APF describe it as the treatment for confirmed attacks in all 4 acute porphyrias in both sexes, and the label's own compassionate use study included men (28% of 130 patients).

Panhematin is heme infused into a vein for 3 to 14 days to stop an attack that has started. Givlaari is an RNA medicine injected under the skin once a month to prevent attacks in adults who keep having them. They are not substitutes: trial patients on Givlaari still received hemin for breakthrough attacks, just far less often.

Recordati Rare Diseases Patient Solutions (1-866-209-7604) offers benefit checks, prior authorization help, a copay program for eligible commercially insured patients and a Patient Assistance Program for eligible patients with insurance or financial barriers. Government plan members are not eligible for the copay program.