Understanding PROMACTA | PROMACTA® (eltrombopag)

How PROMACTA works for patients with persistent or chronic ITP

When you have low platelets due to persistent or chronic ITP, the treatment you start on, such as steroids, may stop working. That's where PROMACTA can help. PROMACTA is a platelet booster that functions differently than steroids or intravenous immunoglobulin (IVIg). It works with your body to create more platelets so it can make up for the ones your body is missing. This in turn helps boost platelet counts and may reduce bruising and bleeding caused by persistent or chronic ITP.

How PROMACTA treats persistent or chronic ITP

PROMACTA binds to TPO receptors like regular TPO to boost platelet production

Illustration is for representative purposes only.

A hormone called thrombopoietin (TPO) naturally creates platelets in your body and, unlike some other drugs used to treat persistent or chronic ITP, PROMACTA acts the same as TPO—working alongside your body to create more platelets.

PROMACTA is a once-daily oral platelet booster that comes in both a tablet and an oral suspension, so even if you can’t swallow a pill, you can still count on a once-daily oral therapy. Some other ITP treatments require you to visit your doctor for an injection, while PROMACTA can be taken at home, at work, or on the go!

Take PROMACTA:

Without a meal or with a meal low in calcium (≤50 mg)
2 hours before or 4 hours after taking medications like antacids, mineral supplements, or foods high in calcium such as dairy products, calcium-fortified juices, and certain fruits and vegetables

PROMACTA may boost your platelet count and keep the levels stable. See below to learn more about how PROMACTA performed in clinical studies in people with persistent or chronic ITP.

How does PROMACTA work?

PROMACTA is the #1 prescribed platelet booster, taken by more than 500,000 people worldwide across all approved uses*^,†

*PROMACTA has been proven to work for children over the age of 1 year with persistent or chronic ITP.
^†Source: IQVIA prescription claims data for March 2021 through August 2022. Includes people with chronic immune thrombocytopenia, severe aplastic anemia, and chronic hepatitis C virus.

Have other treatments, such as steroids or IVIg, not worked to keep your platelet levels stable or have they caused too many side effects?

PROMACTA may be an option to treat your persistent or chronic immune thrombocytopenia (ITP) that is due to low platelets. Once-daily PROMACTA may quickly boost your platelet counts and keep them stable over time. It is not used to make your platelet counts normal.

PROMACTA has been studied extensively in more people with persistent or chronic ITP than any other drug of its kind, including 3 clinical trials for adults and 1 long-term study that lasted 7 years. More than 700 adults were enrolled in the 3 separate studies and 302 of them went on to participate in the long-term study, which was the longest and largest ever run in persistent or chronic ITP.

Did you know PROMACTA is also proven to work for children with persistent or chronic ITP as young as 1 year old? Go here to learn more.

PROMACTA is proven to boost platelet counts

PROMACTA worked fast
In 2 separate trials, the majority of patients achieved their target platelet goal (≥50,000/mcL) in as early as 2 weeks.

A graph showing how PROMACTA worked in two clinical studies

^‡Results for patients receiving 50 mg PROMACTA once daily.

The primary end point of both studies was response rate at Day 43. In Study 1, 59% (43/73) of patients receiving PROMACTA had achieved a response at Day 43 (vs 16% [6/37] for placebo). In Study 2, 70% (19/27) of patients receiving PROMACTA^‡ had achieved a response at Day 43 (vs 11% [3/27] for placebo).

In a clinical trial of 302 adults with persistent or chronic ITP, PROMACTA demonstrated long-term results.

PROMACTA may also help with fatigue, bleeding, and bruising due to reduced platelet counts

By year 1 of a long-term study, 80% of people on PROMACTA self-reported less fatigue (231 of 291) and less bleeding or bruising (228 of 281). PROMACTA showed a 76% reduction in serious bleeding vs placebo at 1 year, from 17% to 4% vs placebo.

A wide range of patients were included in these clinical studies

Data from 3 clinical studies and 1 long-term study of PROMACTA
% of Males	31%-39%
% of Females	61%-69%
Median Age	47-52 years old
Average Starting Platelet Counts in 4 Clinical Studies	Less than 30,000/mcL

Did you know? ITP affects more women than men, at a rate of almost 2:1.

^‡Consistently proven across studies TRA100773B and TRA100773A (platelet count response ≥50 x 10⁹/L), RAISE study (mean number of weeks with platelet counts ≥50 x 10⁹/L and requirement for rescue therapy), and PETIT2/PETIT studies (platelet count response ≥50 x 10⁹/L without rescue).

PROMACTA has demonstrated consistent safety, study after study

No other treatment for persistent or chronic ITP has been studied more than PROMACTA:

The safety of PROMACTA was established in ~900 people, ranging in age from 1 to 85
No single side effect occurred in more than 10% of people across 4 clinical studies
- PROMACTA may cause serious side effects, including liver problems, blood clots, and cataracts
- The most common side effects of PROMACTA in adults and children include:
  - low red blood cell count (anemia)
  - nausea
  - fever
  - abnormal liver function tests
  - cough
  - tiredness
  - headache
  - diarrhea
- See additional safety information in the full Prescribing Information
At 6 years of treatment, no new side effects were seen in comparison with the short-term trials

Tap to see IMPORTANT SAFETY INFORMATION AND APPROVED USES

Important Safety Information for PROMACTA^® (eltrombopag)

What is the most important information I should know about PROMACTA?

PROMACTA can cause serious side effects, including:

Liver problems.

What is the most important information I should know about PROMACTA?

PROMACTA can cause serious side effects, including:

Liver problems.
If you have chronic hepatitis C virus (HCV) and take PROMACTA with interferon and ribavirin treatment, PROMACTA may increase your risk of liver problems. If your health care provider tells you to stop treatment with interferon and ribavirin, you will also need to stop taking PROMACTA.

PROMACTA may increase your risk of liver problems that may be severe and possibly life-threatening. Your health care provider will do blood tests to check your liver function before you start taking PROMACTA and during treatment. Your health care provider may stop your treatment with PROMACTA if you have changes in your liver function blood tests.

Tell your health care provider right away if you have any of these signs and symptoms of liver problems:

yellowing of the skin or the whites of the eyes (jaundice)
unusual darkening of the urine
unusual tiredness
right upper stomach area (abdomen) pain
confusion
swelling of the stomach area (abdomen)

What are the possible side effects of PROMACTA?

PROMACTA may cause serious side effects, including:

Worsening of a precancerous blood condition to a blood cancer called acute myelogenous leukemia (AML). PROMACTA is not for treatment of people with a precancerous condition called myelodysplastic syndromes (MDS). If you have MDS and receive PROMACTA, your MDS condition may worsen and become AML. If MDS worsens to become AML, you may die sooner from AML
High platelet counts and higher risk for blood clots. Your risk of getting a blood clot is increased if your platelet count is too high during treatment with PROMACTA. Your risk of getting a blood clot may also be increased during treatment with PROMACTA if you have normal or low platelet counts. You may have severe problems or die from some forms of blood clots, such as clots that travel to the lungs or that cause heart attacks or strokes. Your health care provider will check your blood platelet counts and change your dose or stop PROMACTA if your platelet counts get too high. Tell your health care provider right away if you have signs and symptoms of a blood clot in the leg such as swelling, pain, or tenderness.

People with chronic liver disease may be at risk for a type of blood clot in the stomach area. Tell your health care provider right away if you have stomach area pain that may be a symptom of this type of blood clot
New or worsened cataracts (a clouding of the lens in the eye). New or worsened cataracts have happened in people taking PROMACTA. Your health care provider will check your eyes before and during your treatment with PROMACTA. Tell your health care provider about any changes in your eyesight while taking PROMACTA

What should I tell my health care provider before taking PROMACTA?

Before you take PROMACTA, tell your health care provider about all of your medical conditions, including if you:

have liver problems
have a precancerous condition called MDS or a blood cancer
have or have had a blood clot
have a history of cataracts
have had surgery to remove your spleen (splenectomy)
have bleeding problems
are of Asian ancestry (such as Chinese, Japanese, Taiwanese, or Korean). You may need a lower dose of PROMACTA
are pregnant or plan to become pregnant. It is not known if PROMACTA will harm an unborn baby. Tell your health care provider if you become pregnant or think you may be pregnant during treatment with PROMACTA. If you are a woman who is able to become pregnant, you must use reliable birth control (contraception) while taking PROMACTA and for at least 7 days after you stop taking PROMACTA. Talk to your health care provider about options of effective birth control methods that may be right for you during this time
are breastfeeding or plan to breastfeed. You should not breastfeed during treatment with PROMACTA. Talk to your health care provider about the best way to feed your baby during this time

Tell your health care provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. PROMACTA may affect the way certain medicines work. Certain other medicines may affect the way PROMACTA works.

Especially tell your health care provider if you take:

certain medicines used to treat high cholesterol, called “statins”
a blood thinner medicine

Certain medicines may keep PROMACTA from working correctly. Take PROMACTA at least 2 hours before or 4 hours after taking these products:

antacids used to treat stomach ulcers or heartburn
multivitamins, mineral supplements, or products that contain iron, calcium, aluminum, magnesium, selenium, and zinc

Ask your health care provider if you are not sure if your medicine is one that is listed above.

Know the medicines you take. Keep a list of them and show it to your health care provider and pharmacist when you get a new medicine.

What should I avoid while taking PROMACTA?

Avoid situations and medicines that may increase your risk of bleeding.

The most common side effects of PROMACTA in adults when used to treat persistent or chronic immune thrombocytopenia (ITP) are:

nausea
diarrhea
upper respiratory tract infection (symptoms may include runny nose, stuffy nose, and sneezing)
vomiting
urinary tract infection
pain or swelling (inflammation) in your throat or mouth (oropharyngeal pain and pharyngitis)
abnormal liver function tests
muscle aches

The most common side effects of PROMACTA in children 1 year and older when used to treat persistent or chronic ITP are:

upper respiratory tract infection (symptoms may include runny nose, stuffy nose, and sneezing)
pain or swelling (inflammation) in your nose or throat (nasopharyngitis)

The most common side effects when PROMACTA is used in combination with other medicines to treat chronic HCV are:

low red blood cell count (anemia)
fever
tiredness
headache
nausea
diarrhea
decreased appetite
flu-like symptoms including fever, headache, tiredness, cough, sore throat, and body aches
trouble sleeping
feeling weak
cough
itching
chills
muscle aches
hair loss
swelling in your ankles, feet, and legs

The most common side effects associated with PROMACTA when used in combination with standard immunosuppressive therapy to treat severe aplastic anemia (SAA) reported more frequently than in patients with SAA when other medicines to treat SAA have not worked well enough are:

abnormal liver function tests
rash
skin discoloration including darkening of skin patches (hyperpigmentation)

The most common side effects when PROMACTA is used to treat SAA when other medicines to treat SAA have not worked well enough are:

nausea
feeling tired
cough
diarrhea
headache

Laboratory tests may show abnormal changes to the cells in your bone marrow.

Tell your health care provider about any bruising or bleeding that happens while you take or after you stop taking PROMACTA.

Tell your health care provider if you have any side effect that bothers you or does not go away.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.