Understanding AIP | PANHEMATIN® (hemin for injection)

UNDERSTANDING AIP

Acute intermittent porphyria (AIP) is a rare inherited disease that can cause serious symptoms that affect your body, brain, and nervous system.

What is AIP? Signs & Symptoms Diagnosis & Testing
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What is AIP?

AIP is caused by a partial lack of activity of an enzyme known as porphobilinogen deaminase (PBGD). The PBGD enzyme is used in the production of heme, a molecule that carries oxygen throughout your body. If you have AIP, you have about half of the normal amount of PBGD enzyme activity in your body. This is usually enough for your body to do what it is supposed to do. But certain situations can upset your body’s chemical balance and disrupt the heme production process, resulting in an AIP attack.

There is no cure for AIP, but it can be managed.

AIP attacks disrupt the heme cycle

Graphic image of the AIP Attack Cycle

1

Exposure to triggers increases the need for heme in the body.
Many situations may trigger an AIP attack.

2

The body sends signals to produce more heme. Triggers may upset your body's chemical balance enough to prompt your body to produce more heme.

3

Heme cannot be produced fast enough due to the enzyme deficiency.
In certain situations, there's not enough PBGD enzyme activity in your body to keep up with the demand.

4

The process gets backed up.
Heme production becomes stalled partway through the process due to the lack of sufficient PBGD enzyme activity.

5

Harmful molecules are produced.
Two harmful molecules—aminolevulinic acid (ALA) and porphobilinogen (PBG)—build up and cause AIP attack symptoms.

6

The heme pool is depleted.
Low PBGD activity depletes the heme pool, triggering continued signals to make more heme and perpetuating the cycle.

Putting off treatment can lead to serious health problems

If an AIP attack is not treated promptly, it can progress to a point where lasting damage to your nervous system may occur.

Learn Why
Image of a woman appearing in pain, a patient portrayal

Signs & Symptoms

Experiencing symptoms is known as having an “AIP attack.” Symptoms may occur for a set period of time, then go away—only to come back later.

Recognizing the signs of an AIP attack early is important because its symptoms can often look a lot like other, more common conditions. This can make it tricky for first-time attack patients, as well as doctors who are not very familiar with AIP, to identify and diagnose it.

The most common symptom of AIP is severe abdominal pain. During an acute attack, the pain can become intense enough to cause nausea, vomiting, hallucinations, and typically require an ER visit.

More than 85% of people who develop AIP symptoms have abdominal pain.

Other common AIP attack symptoms

Symptoms can also occur in many different areas of your body during an AIP attack. These symptoms may also be common in other conditions, so having one or many of these symptoms does not necessarily mean that you have AIP.

Gastrointestinal symptoms icon

Gastrointestinal (GI) symptoms

  • Abdominal pain
  • Vomiting
  • Constipation
  • Diarrhea
Urinary system symptoms icon

Urinary system symptoms

  • Dark-colored urine
  • Red urine
Brain or nervous system symptoms icon

Brain or nervous system symptoms

  • Pain in your arms, hands, legs, feet, back, chest, neck, or head
  • Weak muscles
  • Trouble breathing
  • Psychological symptoms (such as minor behavioral changes, anxiety, confusion or depression)
  • Convulsions
  • Hallucinations
Heart or blood vessel symptoms icon

Heart or blood vessel symptoms

  • Fast heart rate
  • High blood pressure

What can trigger an AIP attack?

Naturally produced hormones—especially estrogen and progesterone—that fluctuate most in the two weeks before a woman’s period starts.

Behaviors such as drinking alcohol, smoking cigarettes, using marijuana or certain illicit drugs.

Stress on the body caused by infections, surgery, or physical exhaustion.

Attacks can also be triggered by starting a new prescription medication — including certain commonly prescribed drugs or even over-the-counter (OTC) products.

Low-carb dieting, including fasting or keto diet.

Mental stress or emotional exhaustion.

Proactive care can make all the difference

If you have attacks of severe abdominal pain or other symptoms that seem to be triggered by any of the factors above, talk to your doctor right away, even if you’re not sure if you are experiencing an AIP attack. Use the symptom checklist to track occurrences and document potential triggers.

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Diagnosing & Testing

Because AIP attacks can be very serious, early diagnosis and treatment of AIP are critical. Symptoms may get worse over time, and untreated attacks can cause serious damage to your nervous system—including paralysis and even death. Unfortunately, it’s common for people with AIP to be misdiagnosed, and to have symptoms for more than 10 years before getting an accurate diagnosis.

If there is a suspicion you may have AIP, your doctor will order lab work, starting with a specific type of urine test that measures your levels of the molecule porphobilinogen (PBG).

An increased PBG level in your urine is typically present in patients at or near the time of an AIP attack—and since it’s not a feature of any other disease—doctors use this test to help confirm an AIP diagnosis.

Image of a doctor talking to a woman in the hospital bed, a patient portrayal

Find a doctor who understands AIP

The American Porphyria Foundation (APF) maintains a list of porphyria specialists who are experts in treating AIP. You can contact the APF to find a doctor located in your area.

Visit APF Site

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

  • 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 nerve damage has occurred. PANHEMATIN therapy is intended to prevent an attack from reaching the critical stage of nerve breakdown. PANHEMATIN is not effective in repairing nerve damage that has already occurred.

IMPORTANT SAFETY INFORMATION

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...

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INDICATION(S) AND IMPORTANT SAFETY INFORMATION

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

  • Before starting PANHEMATIN, your doctor may consider an appropriate trial of carbohydrates or sugar per day for 1 to 2 days.
  • Attacks of porphyria may progress to a point where irreversible nerve damage has occurred. PANHEMATIN therapy is intended to prevent an attack from reaching the critical stage of nerve breakdown. PANHEMATIN is not effective in repairing nerve damage that has already occurred.

IMPORTANT SAFETY INFORMATION

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:

  • Vein inflammation is possible. Your doctor will use a large arm vein or a central line to administer PANHEMATIN to minimize the possibility of vein inflammation.
  • Elevated iron levels may occur. Your doctor must monitor your iron levels if you receive multiple courses of PANHEMATIN.
  • PANHEMATIN has a short-term and mild blood thinning effect. Avoid blood thinners during PANHEMATIN therapy.
  • Reversible kidney shutdown has occurred when too high a dose of PANHEMATIN was given in a single dose.
  • PANHEMATIN may carry a risk of transmitting agents that can cause infections, such as viruses, and theoretically, the Creutzfeldt-Jakob disease (CJD) agent or other unknown agents.

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.

Please see the accompanying full Prescribing Information.