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About AHP

Acute hepatic porphyria (AHP) is a rare genetic disease characterized by debilitating, potentially life-threatening attacks1,2

There are 4 types of AHP1,3

Most common
Extremely rare
Chart showing the 4 types of AHP ranging from most to least common
  • Acute Intermittent Porphyria (AIP)
    • ~80% of all AHP cases are AIP
  • Variegate Porphyria (VP)
  • Hereditary Coproporphyria (HCP)
  • ALAD-Deficiency Porphyria (ADP)

ALAD, delta-aminolevulinic acid dehydratase.

AHP attacks can be severe, unpredictable, and progressive

  • AHP can affect anyone, but is most commonly seen in women of childbearing age4
  • Attacks generally last 3 to 7 days, but recovery can take longer1
  • Severe attacks can progress to respiratory failure or paralysis, leading to temporary or permanent disability1,2
  • Some patients with recurrent attacks report chronic symptoms and long-term complications and describe how chronic symptoms have contributed to their burden of disease1,5

Common signs and symptoms of an AHP attack1,2

CARDINAL SYMPTOM

SEVERE, DIFFUSE ABDOMINAL PAIN1,3

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1 OR MORE ADDITIONAL FINDINGS3

AUTONOMIC
Nervous System3,5

  • Nausea/Vomiting
  • Constipation
  • Tachycardia

CENTRAL
Nervous System3,5

  • Seizures
  • Anxiety
  • Mental status changes

PERIPHERAL
Nervous System3,5

  • Limb weakness
    or pain

CUTANEOUS3✱

  • Skin lesions on
    sun-exposed areas

*Cutaneous symptoms only occur in HCP and VP.3

AHP=acute hepatic porphyria;

HCP=hereditary coproporphyria; VP=variegate porphyria.

>90% of patients report abdominal pain during AHP attacks.3

AHP can go undiagnosed for many years4,6

Because of overlapping symptoms, patients with AHP are often misdiagnosed with other diseases such as:3,7-9

Icon of intestines

  • Acute gastroenteritis with vomiting
  • Appendicitis
  • Cholecystitis
  • Intestinal occlusion
  • Irritable bowel syndrome (IBS)
  • Peritonitis

Icon of a person

  • Chronic hepatitis
  • Endometriosis
  • Pancreatitis

Icon of a brain

  • Anxiety/depression
  • Fibromyalgia
  • Guillain-Barré syndrome
  • Polyneuropathy
  • Psychosis
  • Seizure disorders

Explore the journeys of real patients with AHP

Every AHP patient has a unique story to tell. Discover the journeys to diagnosis and beyond for two real patients with AHP.

See Their Stories
NEXT: How GIVLAARI® (givosiran) works

References: 1. Simon A, Pompilus F, Querbes W, et al. Patient perspective on acute intermittent porphyria with frequent attacks: a disease with intermittent and chronic manifestations. Patient. 2018;11(5):527-537. 2. Neeleman RA, Wagenmakers MAEM, Koole-Lesuis RH, et al. Medical and financial burden of acute intermittent porphyria. J Inherit Metab Dis. 2018;41(5):809-817. 3. Ventura P, Cappellini MD, Biolcati G, Guida CC, Rocchi E; Gruppo Italiano Porfiria (GrlP). A challenging diagnosis for potential fatal diseases: recommendations for diagnosis acute porphyrias. Eur J Intern Med. 2014;25(6):497-505. 4. Bissell DM, Anderson KE, Bonkovsky HL. Porphyria. N Engl J Med. 2017;377(9):862-872. 5. Puy H, Gouya L, Deybach JC. Porphyrias. Lancet. 2010;375(9718):924-937. 6. Anderson KE. Acute hepatic porphyrias: current diagnosis & management. Mol Genet Metab. 2019;128(3):219-227. 7. Alfadhel M et al. Acute intermittent porphyria caused by novel mutation in HBMS gene, misdiagnosed as cholecystitis. Neuropsychiatr Dis Treat. 2014;10:2135-2137. 8. Ko JJ et al. Real-world analysis of symptoms, diagnostic patterns, and provider perspective on acute hepatic porphyrias. Poster presented at: American College of Gastroenterology Annual Scientific Meeting; October 5-10, 2018; Philadelphia, PA. 9. Kondo M, Yano Y, Shirataka M, Urata G, Sassa S. Porphyrias in Japan: compilation of all cases reported through 2002. Int J Hematol. 2004;79:448-456.
 

IMPORTANT SAFETY INFORMATION 

Contraindications

GIVLAARI® (givosiran) is contraindicated in patients with known severe hypersensitivity to givosiran. Reactions have included anaphylaxis.

Anaphylactic Reaction

Anaphylaxis has occurred with GIVLAARI treatment (<1% of patients in clinical trials). Ensure that medical support is available to appropriately manage anaphylactic reactions when administering GIVLAARI. Monitor for signs and symptoms of anaphylaxis. If anaphylaxis occurs, immediately discontinue administration of GIVLAARI and institute appropriate medical treatment.

Hepatic Toxicity

Transaminase elevations (ALT) of at least 3 times the upper limit of normal (ULN) were observed in 15% of patients receiving GIVLAARI in the placebo-controlled trial. Transaminase elevations primarily occurred between 3 to 5 months following initiation of treatment.

Measure liver function tests prior to initiating treatment with GIVLAARI, repeat every month during the first 6 months of treatment, and as clinically indicated thereafter. Interrupt or discontinue treatment with GIVLAARI for severe or clinically significant transaminase elevations. In patients who have dose interruption and subsequent improvement, reduce the dose to 1.25 mg/kg once monthly. The dose may be increased to the recommended dose of 2.5 mg/kg once monthly if there is no recurrence of severe or clinically significant transaminase elevations at the 1.25 mg/kg dose.

Renal Toxicity

Increases in serum creatinine levels and decreases in estimated glomerular filtration rate (eGFR) have been reported during treatment with GIVLAARI. In the placebo-controlled study, 15% of patients receiving GIVLAARI experienced a renally-related adverse reaction. The median increase in creatinine at Month 3 was 0.07 mg/dL. Monitor renal function during treatment with GIVLAARI as clinically indicated.

Injection Site Reactions

Injection site reactions were reported in 25% of patients receiving GIVLAARI in the placebo-controlled trial. Symptoms included erythema, pain, pruritus, rash, discoloration, or swelling around the injection site. One (2%) patient experienced a single, transient, recall reaction of erythema at a prior injection site with a subsequent dose administration.

Drug Interactions

Concomitant use of GIVLAARI increases the concentration of CYP1A2 or CYP2D6 substrates, which may increase adverse reactions of these substrates. Avoid concomitant use of GIVLAARI with CYP1A2 or CYP2D6 substrates for which minimal concentration changes may lead to serious or life-threatening toxicities. If concomitant use is unavoidable, decrease the CYP1A2 or CYP2D6 substrate dosage in accordance with approved product labeling.

Adverse Reactions

The most common adverse reactions that occurred in patients receiving GIVLAARI were nausea (27%) and injection site reactions (25%).

INDICATION

GIVLAARI is indicated for the treatment of adults with acute hepatic porphyria (AHP).

For additional information about GIVLAARI, please see full Prescribing Information.