For Patients & Caregivers

In Infants and Children

HEMLIBRA WAS STUDIED IN PEDIATRIC HEMOPHILIA A PATIENTS WITH AND WITHOUT FACTOR VIII INHIBITORS1,4,7,9

HEMLIBRA® (emicizumab-kxwh) Clinical Trials Haven 2 Icon

Children with inhibitors4
n=88
1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W4

See the Results

HEMLIBRA® (emicizumab-kxwh) Clinical Trials Haven 7 Icon

Infants without inhibitors
n=55
1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W*9

See the Results

HEMLIBRA® (emicizumab-kxwh) Clinical Trials HOHOEMI Icon

Children without inhibitors
n=13
3 mg/kg Q2W or 6 mg/kg Q4W7

See the Results

*Patients in the HAVEN 7 primary analysis received HEMLIBRA at a maintenance dose of 3 mg/kg Q2W for 52 weeks. After 52 weeks, patients could continue HEMLIBRA at the same dosing regimen or switch to other dosing regimens (1.5 mg/kg QW or 6 mg/kg Q4W) for the 7 year follow-up.

Starting early Children with inhibitors Children without inhibitors Infants and previously untreated patients
Starting early

Starting prophylaxis at a young age helps prevent breakthrough bleeds24

HEMLIBRA® (emicizumab-kxwh) Pediatric Patients Joint

Breakthrough bleeds impact patients in several ways.24,25

  • Clinical bleeds and subclinical bleeds can both result in increased joint damage over time24
  • Repeated bleeds can cause irreversible damage, leading to chronic pain and arthropathy, even in early childhood25
  • A single serious bleed can cause new bleeds to occur in a damaging cycle25

Prophylactic FVIII in young boys (≤6 years of age) with severe hemophilia A reduced the risk of joint damage by 83% vs on-demand treatment†26

Percentage of patients observed at 6 years of age with normal ankle, knee, and elbow joints 

 

Based on a randomized trial (N=65) with a mean observation period of 49 months.

†Results from a multicenter, randomized, open-label trial of young boys (<30 months old) with severe hemophilia A without inhibitors randomized to regular prophylactic recombinant FVIII infusions or to an enhanced episodic infusion schedule. Clinical assessments of index joints were performed with plain-film x-rays or magnetic resonance imaging at age 6.26

The MASAC recommendations discuss using HEMLIBRA early on in hemophilia A patients experiencing bleeds15,27

  • MASAC recommends that prophylaxis be considered optimal therapy for individuals with severe hemophilia A and should be instituted early (prior to the onset of frequent bleeding)
  • MASAC further recommends that infants should be considered for prophylaxis with HEMLIBRA or FVIII at any time after birth, given the increased risk of intracranial hemorrhage prior to initiation of FVIII prophylaxis
Children with inhibitors

Additional HEMLIBRA data for pediatric patients with FVIII inhibitors5:

  • 70% of patients receiving HEMLIBRA Q2W (3 mg/kg) had zero treated bleeds
  • 60% of patients receiving HEMLIBRA Q4W (6 mg/kg) had zero treated bleeds
  • The ABRs† were 0.2 (95% CI: 0.06; 0.54) for the Q2W arm and 1.8 (95% CI: 0.32; 10.59) for the Q4W arm
  • The median efficacy periods were 67.1 weeks (54.7-129.4) for the Q2W arm (n=10) and 66.6 weeks (8.1-140.4) for the Q4W arm (n=10)
  • HAVEN 2 was designed to be a descriptive study

HEMLIBRA prophylaxis showed improvement at 25 weeks from baseline in physical health score.1

The HAVEN 2 study evaluated patient-reported hemophilia-related symptoms (painful swellings and presence of joint pain) and physical functioning (pain with movement) using the Physical Health Score of the Hemophilia-specific Quality of Life Short Form (Haemo-QoL-SF) questionnaire for patients ≥8 to <12 years of age.

ABRs shown are for treated bleeds.
†ABR was calculated with a negative binomial regression model, accounting for the different follow-up times.

Intra-patient data for pediatric patients with FVIII inhibitors taking HEMLIBRA

67% of pediatric patients receiving HEMLIBRA had zero treated bleeds vs 7% on prior BPAs.4

HAVEN 2‡: INDIVIDUAL PATIENT ABR AT A MEDIAN STUDY DURATION OF 89 WEEKS IN QW COHORT (N=15)§4

INTRA-INDIVIDUAL COMPARISON

  • HAVEN 2 intra-patient comparison was not prespecified to show superiority in ABR vs prior BPAs

ABRs shown are for treated bleeds.
‡Based on HAVEN 2: Non-randomized, multicenter, open-label clinical trial in pediatric patients (age <12 years, or 12–17 years who weigh <40 kg) with hemophilia A with FVIII inhibitors. All patients received HEMLIBRA prophylaxis at 3 mg/kg QW for the first 4 weeks. Sixty-eight patients received 1.5 mg/kg QW, 10 patients received 3 mg/kg Q2W, and 10 patients received 6 mg/kg Q4W thereafter.4
§A comparison of data from HAVEN 2 vs data in the NIS prior to enrollment.

Intraindividual comparison of 15 participants who previously took BPA prophylaxis showed that emicizumab prophylaxis reduced the ABR by 99% (95% CI: 97.4; 99.4).4

Children without inhibitors

A majority of patients on HEMLIBRA had zero bleeds requiring treatment, regardless of dosing option

  • HAVEN 2: 77% of pediatric patients with FVIII inhibitors taking HEMLIBRA QW (n=65) had zero treated bleeds4
  • HAVEN 4†: 56% of adults and adolescents with or without FVIII inhibitors taking HEMLIBRA Q4W (n=41) had zero treated bleeds1

As observed over at least 52 weeks (HAVEN 2) and at least 24 weeks (HAVEN 4).1,4
†Based on HAVEN 4: A single-arm, multicenter, open-label trial in 41 adult and adolescent males with hemophilia A with or without FVIII inhibitors who previously received either on-demand or prophylactic treatment with FVIII or BPAs. All patients received HEMLIBRA prophylaxis at 3 mg/kg QW for the first 4 weeks followed by 6 mg/kg Q4W thereafter.1

HOHOEMI‡: A multicenter, open-label study of pediatric patients without FVIII inhibitors7

  • Patients were <12 years old
  • One patient in the Q4W cohort who was 4 months old had never been treated with FVIII
  • This trial had a limited sample size (n=13) and was conducted in Japan
  • HOHOEMI was a descriptive study with no formal hypothesis testing

HEMLIBRA Q2W prophylaxis

1.3 ABR§

(95% Cl: 0.6; 2.9)
(n=6)

HEMLIBRA Q4W prophylaxis

0.7 ABR§

(95% Cl: 0.2; 2.6)
(n=7)

In a safety analysis of the HOHOEMI study7:

  • The most common adverse reactions were contusion (n=10), nasopharyngitis (n=5), excoriation (n=4), and falls (n=4)
  • Two serious adverse events were reported, which consisted of bleeding from soft tissue surrounding a right knee and pain from bruised lower leg, both of which were unrelated to HEMLIBRA

‡HOHOEMI was a non-randomized study in pediatric patients (Japanese children <12 years) with hemophilia A without FVIII inhibitors. All patients received HEMLIBRA prophylaxis at 3 mg/kg once weekly for the first 4 weeks. Six patients received 3 mg/kg Q2W an 7 patients received 6 mg/kg Q4W thereafter.7
§ABR was calculated with a negative binomial regression model, accounting for the different follow-up times.1
ABRs shown are for treated bleeds.1

Infants and previously untreated patients

A majority of patients on HEMLIBRA had zero treated bleeds, regardless of their age, dosing option, or inhibitor status in the pivotal HAVEN 1-4 trials1,4,5

  • HAVEN 2: 77% of pediatric patients (children <12 years*) with FVIII inhibitors taking HEMLIBRA QW (n=65) had zero treated bleeds4
  • HAVEN 3: 60% of adults and adolescents (ages 12 and older) without FVIII inhibitors taking HEMLIBRA Q2W (n=35) had zero treated bleeds1

*With allowance for patients 12–17 years old who weigh <40 kg.

HAVEN 7: A clinical trial for infants and PUPs

  • HAVEN 7 was a descriptive single-arm study with no comparator group9
  • Patients with ICH were excluded from the study. This study was not powered to establish evidence on whether HEMLIBRA prophylaxis protects against ICH9
  • A total of 55 PUPs or MTPs from birth to ≤12 months of age with severe hemophilia A without factor VIII inhibitors were enrolled9

PATIENT BASELINE CHARACTERISTICS IN HAVEN 7 (n=55)9,28

NBDF’s MASAC recommends that infants should be considered for prophylaxis with HEMLIBRA or factor VIII at any time after birth.15

  • However, many infants with severe hemophilia A do not receive prophylaxis until >1 year of age, despite diagnosis at birth29

†Defined as a patient with ≤5 exposure days to FVIII.
‡The reporting period was variable across the 36 patients who had ≥1 bleed prior to receiving HEMLIBRA, with the median (min, max) age at time of first historical treated or untreated bleed being 1 (0, 49) week(s).9

Pharmacokinetic profile of HEMLIBRA in HAVEN 7

MEAN HEMLIBRA TROUGH CONCENTRATIONS BY STUDY WEEK9

For the patient, whose dose was up-titrated, only data before up-titration are included.

Results for our youngest patients

In HAVEN 7, 55% of infants had zero treated bleeds while on HEMLIBRA prophylaxis9

HAVEN 7: INFANTS WITHOUT FVIII INHIBITORS9

  • 55 participants in the primary analysis received HEMLIBRA at a maintenance dose of 3 mg/kg Q2W for 52 weeks
  • Median (range) age at primary analysis: 29.0 (12-39) months (n=55)
  • Median (range) treatment duration was 100.3 (52-118) weeks
  • The ABRs (95% CI) for all bleeds, treated spontaneous bleeds, and treated joint bleeds were 2.0 (1.5; 2.7), 0,§ and 0 (0.0; 0.1), respectively
  • There were 42 treated bleeds in 25 patients all of which were traumaticII
  • 140 untreated traumatic bleeds were reported

These data are a descriptive analysis and therefore should be interpreted with caution.

§ABR could not be estimated via the negative binomial regression model as no treated spontaneous bleeds were observed in the study; as a result, a value of 0.0 is reported instead.
IIBleeds were categorized as traumatic if parents/caregivers recorded a bleed with a known or believed reason for the bleed.
Model-based ABRs were estimated using negative binomial regression.

HAVEN 7 Safety Profile

In HAVEN 7, no new safety signals were observed

ADVERSE EVENTS (n=55)9

  • Of the 55 patients evaluated, none tested positive for ADAs to HEMLIBRA9
  • Of the 24 patients tested for FVIII inhibitors following FVIII exposure, 2 PUPs tested positive, both of whom were less than 3 months of age at enrollment9
    • One PUP was confirmed positive on Day 603 and Day 681 following 3 non-consecutive exposure days with standard half-life FVIII for traumatic bleed management9
    • The second PUP tested positive on Day 428 and was confirmed for inhibitors on Day 532 following 10 non-consecutive exposure days with extended half-life FVIII related to bleed treatment and surgical procedures9

These data are a descriptive analysis and therefore should be interpreted with caution.

#All treatment-related AEs were Grade 1 ISRs.
**Sixteen patients reported 30 SAEs; none were considered related to HEMLIBRA, and all considered serious due to hospitalization. SAEs included: fall (n=4); head injury (n=4); bronchiolitis, bronchitis, pneumonia, tonsillitis, mouth hemorrhage, tongue hemorrhage (n=2 for each); ear infection, laryngitis, upper respiratory tract infection, urinary tract infection, viral infection, eyelid contusion, post-procedural fever (liver biopsy), post-procedural hemorrhage (tonsillectomy), skin laceration, tongue injury (n=1 for each).
††One anaphylactic reaction due to an egg allergy was reported in one patient; this event resolved and was considered not related to HEMLIBRA.

Frequently Asked Questions

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Get the Facts

ADA=anti-drug antibody; ABR=annualized bleed rate. ABR calculated with a negative binomial regression model, which accounts for different follow-up times; AE=adverse event; BPA=bypassing agent; CI=confidence interval; FVIII=factor VIII; ICH=intracranial hemorrhage; IQR=interquartile range; ISR=injection-site reaction; MASAC=Medical and Scientific Advisory Council; MTP=minimally treated patient; NBDF=National Bleeding Disorders Foundation; NIS=non-interventional study; PUP=previously untreated patient; QW=once weekly; Q2W=once every 2 weeks; Q4W=once every 4 weeks; SAE=serious adverse event; SD=standard deviation; TMA=thrombotic microangiopathy.

Doctor Discussion Guide

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Indication & Important Safety Information

Indication
HEMLIBRA is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A with or without factor VIII inhibitors.

Boxed WARNING: THROMBOTIC MICROANGIOPATHY and THROMBOEMBOLISM
Cases of thrombotic microangiopathy and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC) was administered for 24 hours or more to patients receiving HEMLIBRA prophylaxis. Monitor for the development of thrombotic microangiopathy and thrombotic events if aPCC is administered. Discontinue aPCC and suspend dosing of HEMLIBRA if symptoms occur. 

Warnings and Precautions
Thrombotic Microangiopathy (TMA) and Thromboembolism Associated With HEMLIBRA and aPCC
In clinical trials, TMA was reported in 0.8% of patients (3/391) and thrombotic events were reported in 0.5% of patients (2/391). In patients who received at least one dose of aPCC, TMA was reported in 8.1% of patients (3/37) and thrombotic events were reported in 5.4% of patients (2/37). Patients with TMA presented with thrombocytopenia, microangiopathic hemolytic anemia, and acute kidney injury, without severe deficiencies in ADAMTS13.

Consider the benefits and risks if aPCC must be used in a patient receiving HEMLIBRA prophylaxis. Due to the long half-life of HEMLIBRA, the potential for an interaction with aPCC may persist for up to 6 months after the last dose. Monitor for the development of TMA and/or thromboembolism when administering aPCC. Immediately discontinue aPCC and interrupt HEMLIBRA prophylaxis if clinical symptoms, imaging, or laboratory findings consistent with TMA and/or thromboembolism occur, and manage as clinically indicated. Consider the benefits and risks of resuming HEMLIBRA prophylaxis following complete resolution of TMA and/or thrombotic events on a case-by-case basis.

Immunogenicity
Treatment with HEMLIBRA may induce anti-drug antibodies. Anti-emicizumab-kxwh antibodies were reported in 5.1% of patients (34/668) treated with HEMLIBRA in clinical trials. Most patients with anti-emicizumab-kxwh antibodies did not experience a change in HEMLIBRA plasma concentrations or an increase in bleeding events; however, in uncommon cases (incidence <1%), the presence of neutralizing antibodies with decreasing plasma concentration may be associated with loss of efficacy.

Monitor for clinical signs of loss of efficacy (eg, increase in breakthrough bleeding events) and if observed, promptly assess the etiology and consider a change in treatment if neutralizing anti-emicizumab-kxwh antibodies are suspected.

Laboratory Coagulation Test Interference
HEMLIBRA affects intrinsic pathway clotting-based laboratory tests, including activated clotting time (ACT); activated partial thromboplastin time (aPTT); and all assays based on aPTT, such as one-stage, factor VIII (FVIII) activity. Therefore, intrinsic pathway clotting-based coagulation laboratory test results in patients who have been treated with HEMLIBRA prophylaxis should not be used to monitor HEMLIBRA activity, determine dosing for factor replacement or anti-coagulation, or measure FVIII inhibitor titers.

Results affected by HEMLIBRA: aPTT; Bethesda assays (clotting-based) for FVIII inhibitor titers; one-stage, aPTT-based single-factor assays; aPTT-based Activated Protein C Resistance (APC-R); ACT.

Results unaffected by HEMLIBRA: Bethesda assays (bovine chromogenic) for FVIII inhibitor titers; thrombin time (TT); one-stage, prothrombin time (PT)-based single-factor assays; chromogenic-based single-factor assays other than FVIII (see Drug Interactions for FVIII chromogenic activity assay considerations); immuno-based assays (ie, ELISA, turbidimetric methods); genetic tests of coagulation factors (eg, Factor V Leiden, Prothrombin 20210).

Most Common Adverse Reactions
The most common adverse reactions (incidence ≥10%) are injection site reactions, headache, and arthralgia.

Adverse Reactions
Characterization of aPCC Treatment in Pooled Clinical Trials
There were 130 instances of aPCC treatment in 37 patients, of which 13 instances (10%) consisted of on average a cumulative amount of >100 U/kg/24 hours of aPCC for 24 hours or more; 2 of the 13 were associated with thrombotic events and 3 of the 13 were associated with TMA. No TMA or thrombotic events were associated with the remaining instances of aPCC treatment.

Injection Site Reactions
In total, 85 patients (22%) reported injection site reactions (ISRs). All ISRs observed in HEMLIBRA clinical trials were reported as mild to moderate intensity and 93% resolved without treatment. The commonly reported ISR symptoms were injection site erythema (11%), injection site pruritus (4%), and injection site pain (4%).

Other Less Common (<1%) Reactions
Rhabdomyolysis was reported in 2 adult patients with asymptomatic elevations in serum creatine kinase without associated renal or musculoskeletal symptoms. In both instances, the event occurred following an increase in physical activity.

Drug Interactions
Clinical experience suggests that a drug interaction exists with HEMLIBRA and aPCC.

Pregnancy, Lactation, Females and Males of Reproductive Potential
Women of childbearing potential should use contraception while receiving HEMLIBRA. It is not known whether HEMLIBRA can cause fetal harm when administered to a pregnant woman or can affect reproduction capacity. HEMLIBRA should be used during pregnancy only if the potential benefit for the mother outweighs the risk to the fetus. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for HEMLIBRA and any potential adverse effects on the breastfed child from HEMLIBRA or from the underlying maternal condition.

You may report side effects 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 the HEMLIBRA full Prescribing Information for additional Important Safety Information, including Boxed WARNING.

    • HEMLIBRA package insert. South San Francisco, CA: Genentech, Inc.; 2024.

      HEMLIBRA package insert. South San Francisco, CA: Genentech, Inc.; 2024.

    • FDA Approves Genentech’s HEMLIBRA (emicizumab-kxwh) for Hemophilia A Without Factor VIII Inhibitors. Genentech Press Release. South San Francisco, CA: Genentech; October 4, 2018.

      FDA Approves Genentech’s HEMLIBRA (emicizumab-kxwh) for Hemophilia A Without Factor VIII Inhibitors. Genentech Press Release. South San Francisco, CA: Genentech; October 4, 2018.

    • Data on File. Genentech, Inc.

      Data on File. Genentech, Inc.

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