Clinical Trial Results

AIM FOR ZERO: Most people on HEMLIBRA didn’t have any bleeds that needed treatment with additional therapy, regardless of their dosing option

Percentage of people with zero bleeds in scientific studies*

Adults and young adults WITHOUT factor VIII inhibitors: HAVEN 3 study

HEMLIBRA® (emicizumab-kxwh) Clinical Trial Results Haven 3 graphic 1

56%

of people taking HEMLIBRA once a week had 0 bleeds (20/36)

HEMLIBRA® (emicizumab-kxwh) Clinical Trial Results Haven 3 infographic 2

60%

of people taking HEMLIBRA once every 2 weeks had 0 bleeds (21/35)

VS

HEMLIBRA® (emicizumab-kxwh) Clinical Trial Results Haven 3 infographic 4

0%

of people taking factor VIII to treat bleeds on-demand (no prophylaxis) had 0 bleeds (0/18)

*The need to treat was determined by the individual or the study physician.

Adults and young adults WITH factor VIII inhibitors: HAVEN 1 study

HEMLIBRA® (emicizumab-kxwh) Clinical Trial Results Haven 1 graphic 1

63%

of people taking HEMLIBRA once a week had 0 bleeds (22/35)

VS

HEMLIBRA® (emicizumab-kxwh) Clinical Trial Results Haven 1 graphic 2

6%

of people taking bypassing agents to treat bleeds (no prophylaxis) had 0 bleeds (1/18)

97% of people without inhibitors (HAVEN 3) chose to stay on for the long-term portion of this study. Data from long-term treatment and additional studies are shown below

Explore more data below

Children WITH factor VIII inhibitors: HAVEN 2 study

HEMLIBRA® (emicizumab-kxwh) Clinical Trial Results Haven 2 infographic 1

77%

of children taking HEMLIBRA once a week had 0 bleeds (50/65)

HEMLIBRA was also studied in children without FVIII inhibitors

A study in Japan called HOHOEMI measured the annualized bleed rate (ABR) in children without factor VIII inhibitors using HEMLIBRA. This was a descriptive study with a small patient population (13 children). In a descriptive analysis, researchers are simply looking at what happened in the clinical trial, without determining a definitive benefit.

1.3 ABR

Children using HEMLIBRA every 2 weeks (6 people)

0.7 ABR

Children using HEMLIBRA every 4 weeks (7 people)

Children who were included in HOHOEMI were in the study for at least 24 weeks.

The median time on HEMLIBRA every 2 weeks and HEMLIBRA every 4 weeks was 39 weeks and 32 weeks, respectively.

FVIII=factor VIII.

HEMLIBRA® (emicizumab-kxwh) Clinical Trial Results Haven 4 infographic

56%

of people taking HEMLIBRA once every 4 weeks had 0 bleeds (23/41)

Adults: 18 years or older.
Young adults (adolescents): 12 to less than 18 years.
Children: less than 12 years.

People who were included in HAVEN 1 and HAVEN 3 were in the study for at least 24 weeks. People in HAVEN 2 were in the study for at least 52 weeks. People in HAVEN 4 were in the study for at least 24 weeks.

The median time on HEMLIBRA was 31 weeks (HAVEN 3), 29 weeks (HAVEN 1), 58 weeks (HAVEN 2), and 26 weeks (HAVEN 4). A median is a numeric middle. That means that in a list of numbers, the median is the number in the middle.

A long-term descriptive study of people who took HEMLIBRA

Adults and young adults WITHOUT factor VIII inhibitors: HAVEN 3 study

Percentage of people who had zero bleeds that needed treatment in each 24-week period
WEEKS 1-24
63% 03/148 people
WEEKS 25-48
73% 105/144 people
WEEKS 49-72
74% 104/140 people
WEEKS 73-96
79% 104/131 people
WEEKS 97-120
80% 83/104 people
WEEKS 121-144
82% 73/89 people
Percentage of people who had zero bleeds that needed treatment in each 24-week period
WEEKS
1-24
WEEKS
25-48
WEEKS
49-72
WEEKS
73-96
WEEKS
97-120
WEEKS
121-144
63% 73% 74% 79% 80% 82%
93/148 people 105/144 people 104/140 people 104/131 people 83/104 people 73/89 people

Data for each period should be looked at individually—not over time.

What's a descriptive study?

Researchers are simply looking at what happened in a clinical trial, without determining a definitive benefit. Therefore, data for each period should be looked at individually and not over time.

Why do groups differ in size?
When HEMLIBRA was approved by the FDA for people without FVIII inhibitors in October 2018, participants could choose to leave the long-term study, but still continue on HEMLIBRA. People who left the study are not included in the later intervals of the analysis.

Making a change can make a difference

Decrease in bleed rate in a study of adults and young adults without factor VIII inhibitors (HAVEN 3)

Median observational period of 34 weeks.

Decrease in bleed rate in a study of adults and young adults with factor VIII inhibitors (HAVEN 1)

Median observational period of 30 weeks.

Make a choice for fewer target joint bleeds

People taking HEMLIBRA for at least 24 weeks (HAVEN 3) saw a 95% reduction in target joint bleeds that needed treatment.

HEMLIBRA once a week

95%

REDUCTION IN TARGET JOINT BLEEDS THAT NEEDED TREATMENT
vs no prophylaxis
| 36 people

HEMLIBRA once every 2 weeks

95%

REDUCTION IN TARGET JOINT BLEEDS THAT NEEDED TREATMENT
vs no prophylaxis
| 35 people

A Post Hoc, Descriptive Study of Target Joint Health in People Taking HEMLIBRA Long-Term

In a Study of Adults and Young Adults Without Factor VIII Inhibitors (HAVEN 3)

99%

OF TARGET JOINTS WERE RESOLVED
in patients taking HEMLIBRA

These data are from 96 people who reported a combined total of 237 target joints before starting HEMLIBRA and were then on HEMLIBRA for a median of 163 weeks

What is a post hoc, descriptive study?

  • Researchers are simply looking at what happened in a clinical trial after it was completed, without determining a definitive benefit. Therefore, conclusions should not be drawn based on this information

What is a resolved target joint?

  • A target joint is resolved if it had 2 or fewer spontaneous bleeds during 52 weeks of HEMLIBRA treatment

More than 15,000 people have been treated with HEMLIBRA worldwide

Ready to talk to your doctor? Download the doctor discussion guide to start the conversation.

Individual results may vary. Not all patients in the clinical trials had zero bleeds. See results for clinical trial patients with or without factor VIII inhibitors at the top of this page.

The HEMLIBRA community members shown have been compensated for their participation.

When starting HEMLIBRA, a “loading dose” is taken once weekly for 4 weeks. After that, HEMLIBRA offers 3 regular dosing options: once weekly, once every 2 weeks, or once every 4 weeks. Talk to a doctor about dosing.

As of February 2022.

Downloadable Resources

Make sure you have all the information you need at your fingertips.

More Than 15,000 People Have Been Treated With HEMLIBRA Worldwide

Ready to talk to your doctor? Download the doctor discussion guide to start the conversation.

What is HEMLIBRA?

HEMLIBRA is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A with or without factor VIII inhibitors.

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

HEMLIBRA increases the potential for your blood to clot. People who use activated prothrombin complex concentrate (aPCC; Feiba®) to treat breakthrough bleeds while taking HEMLIBRA may be at risk of serious side effects related to blood clots.

These serious side effects include: 

  • Thrombotic microangiopathy (TMA), a condition involving blood clots and injury to small blood  vessels that may cause harm to your kidneys, brain, and other organs 
  • Blood clots (thrombotic events), which may form in blood vessels in your arm, leg, lung, or head  

Talk to your doctor about the signs and symptoms of these serious side effects, which can include: 

  • Confusion
  • Stomach, chest, or back pain
  • Weakness
  • Nausea or vomiting
  • Swelling, pain, or redness
  • Feeling sick or faint
  • Decreased urination
  • Swelling of arms and legs
  • Yellowing of skin and eyes
  • Eye pain, swelling, or trouble seeing
  • Fast heart rate
  • Numbness in your face
  • Headache
  • Shortness of breath
  • Coughing up blood

If you experience any of these symptoms during or after treatment with HEMLIBRA, get medical help right away. 

Carefully follow your healthcare provider’s instructions regarding when to use an on-demand bypassing agent or factor VIII, and the dose and schedule to use for breakthrough bleed treatment. If aPCC (Feiba®) is needed, talk to your healthcare provider in case you feel you need more than 100 U/kg of aPCC (Feiba®) total. 

Your body may make antibodies against HEMLIBRA, which may stop HEMLIBRA from working properly. Contact your healthcare provider immediately if you notice that HEMLIBRA has stopped working for you (eg, increase in bleeds).

The most common side effects of HEMLIBRA include: injection site reactions (redness, tenderness, warmth, or itching at the site of injection), headache, and joint pain. These are not all of the possible side effects of HEMLIBRA. You can speak with your healthcare provider for more information.

What else should I know about HEMLIBRA?

See the detailed “Instructions for Use” that comes with your HEMLIBRA for information on how to prepare and inject a dose of HEMLIBRA, and how to properly throw away (dispose of) used needles and syringes.

  • Stop taking your prophylactic bypassing therapy the day before you start HEMLIBRA
  • You may continue taking your prophylactic factor VIII for the first week of HEMLIBRA

HEMLIBRA may interfere with laboratory tests that measure how well your blood is clotting and create an inaccurate result. Speak with your healthcare provider about how this may affect your care. 

Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Only use HEMLIBRA for the condition it was prescribed. Do not give HEMLIBRA to other people, even if they have the same symptoms that you have. It may harm them.

Tell your healthcare provider about all the medicines you take, including prescription medicines, over-the-counter medicines, vitamins, or herbal supplements. Keep a list of them to show your healthcare provider and pharmacist.

Before using HEMLIBRA, tell your healthcare provider about all of your medical conditions, including if you are pregnant, plan to become pregnant, are breastfeeding, or plan to breastfeed.

Since HEMLIBRA was tested in males, there is no information on whether HEMLIBRA may impact your unborn baby or breast milk. Females who are able to become pregnant should use birth control during treatment.

Side effects may be reported to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at (888) 835-2555.

Please see Important Safety Information, including Serious Side Effects, as well as the HEMLIBRA full Prescribing Information and Medication Guide.