Important Safety Information
Prescribing Information
Indications and Usage
For Healthcare Professionals For Patients

About PANHEMATIN for
the Treatment of AIP

Panhematin® is a hemin for injection indicated for the amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporally related to the menstrual cycle in susceptible women, after initial carbohydrate therapy is known or suspected to be inadequate.

Limitations of Use

Before administering PANHEMATIN, consider an appropriate period of carbohydrate loading (i.e., 400 g glucose/day for 1 to 2 days). Attacks of porphyria may progress to a point where irreversible neuronal damage has occurred. PANHEMATIN therapy is intended to prevent an attack from reaching the critical stage of neuronal degeneration. PANHEMATIN is not effective in repairing neuronal damage.

Clinical benefit with PANHEMATIN depends on prompt administration.

Recommended dosing guidelines should be strictly followed.

How PANHEMATIN Works

AIP is caused by a deficiency of the enzyme porphobilinogen deaminase (PBGD) in the heme biosynthetic pathway.1

  • Heme is synthesized primarily in the liver and the bone marrow by a complex pathway of eight enzymatic reactions.7,8 The first step is the formation of d-aminolevulinic acid (ALA), catalyzed by the enzyme ALA synthase.7 ALA synthase is repressed via a negative feedback loop.7,9
  • In AIP, precipitating factors markedly increase the demand for hepatic heme.10 The resulting decreased heme pool induces the synthesis of ALA synthase to produce more heme.10 The partial deficiency of PBGD becomes rate-limiting, causing an accumulation of the neurotoxic porphyrin precursors ALA and porphobilinogen (PBG).1,10
  • Panhematin replenishes the depleted heme pool. This limits the production of the porphyrin precursors, most likely due to the inhibition of ALA synthase. The exact mechanism by which hematin produces symptomatic improvement in individuals with acute episodes of the hepatic porphyrias has not been elucidated.6

PANHEMATIN is the 1st
FDA-approved therapy for recurrent AIP attacks.

PANHEMATIN Treatment for AIP Attacks

Biochemical and clinical response to the treatment of AIP attacks in 72 patients

Summary of open-label studies which supported the approval of PANHEMATIN

Clinical and chemical response after hemin therapy

Data from open-label studies which supported approval of PANHEMATIN3

Observational study with patient-reported outcomes

  • Study included 90 AIP patients
  • 55% reported receiving hemin during acute attacks
  • 74% reported PANHEMATIN as being very successful in treatment of abdominal pain and other symptoms
  • 50% reported having received treatment with opiates during an acute attack, and 44% of these patients reported that opiates were effective3

PANHEMATIN Safety Summary

The most common adverse reactions (occurring in >1% of patients) are: headache, pyrexia, infusion site reactions, and phlebitis.

Clinical Trials Experience: Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

The safety of PANHEMATIN use was evaluated in a compassionate use study. A total of 130 patients were treated with hemin for acute attacks, prophylaxis or both. Of those, 111 patients were administered hemin for treatment of 305 acute porphyria attacks and to 40 patients for prophylaxis. The majority (92%) of patients were Caucasian. Most (72%) were female; all adult patients had a mean age ± SD of 40.3 ± 12.3 years. Proportionally more females (15 out of 19) received prophylaxis or a combination of acute treatment and prophylaxis (19 out of 21). For the treatment of acute attacks, patients received 2 to 4 mg/kg/day PANHEMATIN IV for 1 to 9 doses. For prophylaxis patients, the most common doses were weekly or biweekly infusions. Table 1 summarizes adverse reactions occurring in >1% of patients treated with PANHEMATIN, categorized by body system and order of decreasing frequency.

Adverse Reactions in >1% of Patients Treated with PANHEMATIN

Post-Marketing Experience

The following adverse reactions associated with the use of PANHEMATIN were identified in open-label clinical trials or postmarketing reports. Because these reactions were reported voluntarily from a population of uncertain size, it is not always possible to estimate their frequency reliably or to establish a causal relationship to drug exposure.

Blood and Lymphatic System Disorders: thrombocytopenia, coagulopathy (including prolonged prothrombin time and prolonged partial thromboplastin time), and hemolysis

Immune System Disorders: hypersensitivity reactions including a report of infusion-related anaphylactoid reaction presenting as circulatory collapse

Vascular Disorders: injection site venous thrombosis including some that occurred in large veins such as venae cavae

General Disorders and Administration Site Conditions: infusion site reactions (such as erythema, pain, bleeding and extravasation)

Metabolism and Nutrition Disorders: iron overload and serum ferritin increased [See Important Safety Information]

Ordering PANHEMATIN

Contact:

Your primary wholesaler, or

ASD Healthcare:

Ordering Tips:

Orders placed Monday – Thursday by 6:30 PM central time:

  • Sent UPS Next Day Air Saver for mid-afternoon delivery

Orders placed Friday by 6:00 PM central time:

  • Sent UPS Next Day Air Saver for Monday mid-afternoon delivery
  • For next day delivery, Saturday delivery must be requested

Earlier, same day, and weekend delivery are available with an additional shipping cost

Include any specific delivery instructions when ordering

How supplied

PANHEMATIN is supplied as a sterile, lyophilized black powder in single dose dispensing vials (NDC 55292-702-54) in a carton (NDC 55292-702-55).

The vial stopper contains natural rubber latex.

Store lyophilized powder at 20-25°C (68-77°F).

Dosing & Administration Get step-by-step instructions
Resources for Your Patients Help your patients access support

Indications And Usage

PANHEMATIN® is a hemin for injection indicated for the amelioration of recurrent attacks of acute intermittent porphyria temporally related to the menstrual cycle in susceptible women, after initial carbohydrate therapy is known or suspected to be inadequate.

Limitations of Use

  • Before administering PANHEMATIN, consider an appropriate period of carbohydrate loading (i.e., 400 g glucose/day for 1 to 2 days).
  • Attacks of porphyria may progress to a point where irreversible neuronal damage has occurred. PANHEMATIN therapy is intended to prevent an attack from reaching the critical stage of neuronal degeneration. PANHEMATIN is not effective in repairing neuronal damage.

Indications and Important
Safety Information

+ See More

Indications And Usage

PANHEMATIN® is a hemin for injection indicated for the amelioration of recurrent attacks of acute intermittent porphyria temporally related to the menstrual cycle in susceptible women, after initial carbohydrate therapy is known or suspected to be inadequate.

Important Safety Information

  • Do not use in patients with known hypersensitivity to PANHEMATIN.
  • Phlebitis is possible. Utilize a large arm vein or a central venous catheter for administration to minimize the risk of phlebitis.
  • Elevated iron and serum ferritin may occur. Monitor iron and serum ferritin in patients receiving multiple administrations of PANHEMATIN.
  • PANHEMATIN has transient and mild anticoagulant effect. Avoid concurrent anticoagulant therapy.
  • Reversible renal shutdown has been observed with an excessive hematin dose (12.2 mg/kg in a single infusion). Strictly follow recommended dosage guidelines.
  • PANHEMATIN may carry a risk of transmitting infectious agents, e.g., viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent.
  • Most common adverse reactions in >1% of patients are headache, pyrexia, infusion site reactions, and phlebitis.
  • To report SUSPECTED ADVERSE EFFECTS, contact Recordati Rare Diseases Inc. at 1-888-575-8344, or FDA at 1800-FDA-1088 or www.fda.gov/medwatch.
  • Avoid CYP inducing drugs such as estrogens, barbituric acid derivatives and steroid metabolites which induce δ‑aminolevulinic acid synthetase 1 (ALAS1) through a feedback mechanism.

For more information, please see Full Prescribing Information at www.recordatirarediseases.com.


References

  1. Watson CJ, Pierach CA, Bossenmaier I, Cardinal R. Use of hematin in the acute attack of the “inducible” hepatic prophyrias. Adv Intern Med. 1978;23:265-286.
  2. Pierach CA, Bossenmaier I, Cardinal R, Weimer M, Watson CJ. Hematin therapy in porphyric attacks. Klin Wochenschr. 1980;58:829-832.
  3. Lamon JM, Frykholm BC, Hess RA, Tschudy DP. Hematin therapy for acute porphyria. Medicine (Baltimore). 1979;58:252-269.
  4. McColl KE, Moore MR, Thompson GG, Goldberg A. Treatment with haematin in acute hepatic porphyria. Q J Med. 1981;50:161-174.
  5. Anderson KE, Collins S. Open-label study of hemin for acute porphyria: clinical practice implications. Am J Med. September 2006;119:801.e19-801.e24.
  6. PANHEMATIN® package insert. Recordati Rare Diseases Inc., Lebanon, NJ 08833 USA; 2017
  7. Khanderia U, Bhattacharya A. Acute intermittent porphyria: Pathophysiology and treatment. Pharmacother. 1984;4(3):144-150.
  8. Ramanujam V-MS, Anderson KE. Porphyria diagnostics – part 1: A brief overview of the porphyrias. Curr Protoc Hum Genet. 2016;86:17.20.1–17.20.26.
  9. Anderson KE, Sassa S, Bishop DF, Desnick RJ. Disorders of heme biosynthesis: X-linked sideroblastic anemia and the porphyrias. In: Valle D, Beaudet AL, Vogelstein B, Kinzler KW, Antonarakis SE, Ballabio A, Scriver CR, Childs B, Sly WS, eds. The Online Metabolic & Molecular Bases of Inherited Disease. New York: Mc-Graw Hill, 2001:1-212.
  10. Balwani M, Wang B, Anderson KE, Bloomer JR, Bissell DM, Bonkovsky HL, Phillips JD, Desnick RJ; for the Porphyrias Consortium of the Rare Diseases Clinical Research Network. Acute hepatic porphyrias: Recommendations for evaluation and long-term management. Hepatol. 2017;66(4):1314-1322.

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