{
 "query": "emicizumab",
 "ingredient": "emicizumab",
 "slug": "emicizumab",
 "rxcui": "1989794",
 "brands": [],
 "names": [
  "emicizumab"
 ],
 "classes": [],
 "alternatives": [],
 "label": {
  "route_mode": "SUBCUTANEOUS",
  "route_counts": {
   "SUBCUTANEOUS": 1
  },
  "boxed_warning": "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 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. WARNING: THROMBOTIC MICROANGIOPATHY and THROMBOEMBOLISM See full prescribing information for complete boxed warning. 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.",
  "indications_and_usage": "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 (congenital factor VIII deficiency) with or without factor VIII inhibitors. HEMLIBRA is a bispecific factor IXa- and factor X-directed antibody 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 (congenital factor VIII deficiency) with or without factor VIII inhibitors. ( 1 )",
  "contraindications": "None. None ( 4 )",
  "warnings_and_cautions": "Immunogenicity: Anti-emicizumab antibodies (including neutralizing antibodies) have developed in HEMLIBRA-treated patients. In case of clinical signs of loss of efficacy, promptly assess the etiology and consider a change in treatment if neutralizing antibodies are suspected. ( 5.3 , 12.6 , 14.3 ) Laboratory Coagulation Test Interference: HEMLIBRA interferes with activated clotting time (ACT), activated partial thromboplastin time (aPTT), and coagulation laboratory tests based on aPTT, including one-stage aPTT-based single-factor assays, aPTT-based Activated Protein C Resistance (APC-R), and Bethesda assays (clotting-based) for factor VIII (FVIII) inhibitor titers. Intrinsic pathway clotting-based laboratory tests should not be used. ( 5.4 , 7.2 ) 5.1 Thrombotic Microangiopathy Associated with HEMLIBRA and aPCC Cases of thrombotic microangiopathy (TMA) were reported from clinical trials 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. In clinical trials, thrombotic microangiopathy was reported in 0.8% of patients (3/391) and in 8.1% of patie...",
  "warnings": "",
  "adverse_reactions": "The following serious adverse reactions are described elsewhere in the labeling: Thrombotic Microangiopathy Associated with HEMLIBRA and aPCC [see Warnings and Precautions (5.1) ] Thromboembolism Associated with HEMLIBRA and aPCC [see Warnings and Precautions (5.2) ] Most common adverse reactions (incidence \u2265 10%) are injection site reactions, headache, and arthralgia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genentech at 1-888-835-2555 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The following adverse reactions are based on pooled data from two randomized trials in adult and adolescent patients (HAVEN 1 and HAVEN 3), one single-arm trial in adult and adolescent patients (HAVEN 4), one single-arm trial in pediatric patients (HAVEN 2), and one dose-finding trial, in which a total of 391 male patients with hemophilia A received at least one dose of HEMLIBRA as routine prophyl...",
  "drug_abuse_and_dependence": "",
  "controlled_substance": "",
  "recent_major_changes": "",
  "information_for_patients": "Advise the patient to read the FDA-approved patient labeling ( Medication Guide and Instructions for Use ). Use of Bypassing Agents or FVIII Inform the patient and/or caregiver that HEMLIBRA increases coagulation potential. Advise the patient and/or caregiver to discontinue prophylactic use of bypassing agents the day before starting HEMLIBRA prophylaxis. Advise the patient and/or caregiver that prophylactic use of FVIII may be continued for the first week of HEMLIBRA prophylaxis. Discuss the appropriate dosing of concomitant agents such as bypassing agents or FVIII with the patient and/or caregiver prior to starting HEMLIBRA prophylaxis [see Warnings and Precautions (5.1 , 5.2) and Drug Interactions (7.1) ] . Thrombotic Microangiopathy Associated with HEMLIBRA and aPCC Inform the patient and/or caregiver of the potential risk of thrombotic microangiopathy if aPCC is administered while receiving HEMLIBRA prophylaxis. Instruct the patient and/or caregiver to consult their healthcare provider if aPCC is required in cumulative doses exceeding 100 U/kg. Advise the patient and/or caregiver to seek immediate medical attention if any signs or symptoms of thrombotic microangiopathy occur [...",
  "spl_medguide": "This Medication Guide has been approved by the U.S. Food and Drug Administration Revised: 07/2025 Medication Guide HEMLIBRA \u00ae (hem-lee-bruh) (emicizumab-kxwh) injection, for subcutaneous use What is the most important information I should know about HEMLIBRA? HEMLIBRA increases the potential for your blood to clot. Carefully follow your healthcare provider's instructions regarding when to use an on-demand bypassing agent or factor VIII (FVIII) and the recommended dose and schedule to use for breakthrough bleed treatment. HEMLIBRA may cause the following serious side effects when used with activated prothrombin complex concentrate (aPCC; FEIBA \u00ae ), including: Thrombotic microangiopathy (TMA). This is a condition involving blood clots and injury to small blood vessels that may cause harm to your kidneys, brain, and other organs. Get medical help right away if you have any of the following signs or symptoms during or after treatment with HEMLIBRA: confusion weakness swelling of arms and legs yellowing of skin and eyes stomach (abdomen) or back pain nausea or vomiting feeling sick decreased urination Blood clots (thrombotic events). Blood clots may form in blood vessels in your arm, le...",
  "clinical_studies": "14.1 Hemophilia A without FVIII Inhibitors The efficacy of HEMLIBRA for routine prophylaxis in patients with hemophilia A without FVIII inhibitors was evaluated in two clinical trials [adult and adolescent studies (HAVEN 3 and HAVEN 4)]. HAVEN 3 (Adult and Adolescent Patients) The HAVEN 3 study (NCT02847637) was a randomized, multicenter, open-label, clinical trial in 152 adult and adolescent males (aged \u2265 12 years and \u2265 40 kg) with hemophilia A without FVIII inhibitors who previously received either episodic (on demand) or prophylactic treatment with FVIII. Patients received HEMLIBRA prophylaxis, 3 mg/kg once weekly for the first 4 weeks followed by either 1.5 mg/kg once every week [Arms A and D] or 3 mg/kg once every two weeks [Arm B] thereafter, or no prophylaxis (Arm C). Patients in Arm C could switch to HEMLIBRA prophylaxis (3 mg/kg once every two weeks) after completing at least 24 weeks without prophylaxis. For Arms A and B, dose up-titration to 3 mg/kg once every week was allowed after 24 weeks on HEMLIBRA prophylaxis for patients who experienced two or more qualified bleeds (i.e., spontaneous and clinically significant bleeds occurring at steady state). For Arm D patients,...",
  "brand_name": "Hemlibra",
  "manufacturer": "Genentech, Inc.",
  "application_number": "BLA761083",
  "pharm_class_epc": [],
  "route": [
   "SUBCUTANEOUS"
  ],
  "effective_time": "20250711",
  "set_id": "2483adba-fab6-4d1b-96c5-c195577ed071",
  "has_boxed_warning": true,
  "controlled": false,
  "mentions_rems": false
 },
 "faers": {
  "total": 1018,
  "serious": 734,
  "death": 57,
  "hospitalization": 218,
  "primary_suspect": 1016,
  "top_reactions": [
   [
    "Off Label Use",
    158
   ],
   [
    "Haemarthrosis",
    132
   ],
   [
    "Haemorrhage",
    110
   ],
   [
    "No Adverse Event",
    84
   ],
   [
    "Drug Ineffective",
    67
   ],
   [
    "Haematoma",
    42
   ],
   [
    "Fall",
    32
   ],
   [
    "Muscle Haemorrhage",
    30
   ],
   [
    "Arthralgia",
    28
   ],
   [
    "Headache",
    24
   ],
   [
    "Anti Factor Viii Antibody Positive",
    23
   ],
   [
    "Head Injury",
    22
   ],
   [
    "Pyrexia",
    21
   ],
   [
    "Drug Specific Antibody Present",
    20
   ],
   [
    "Haematuria",
    20
   ]
  ],
  "by_year": {
   "2019": 32,
   "2020": 62,
   "2021": 95,
   "2022": 87,
   "2023": 113,
   "2024": 325,
   "2025": 178,
   "2026": 99
  },
  "by_year_primary": {
   "2019": 32,
   "2020": 62,
   "2021": 94,
   "2022": 87,
   "2023": 113,
   "2024": 324,
   "2025": 178,
   "2026": 99
  }
 },
 "faers_base": {
  "total": 20692304,
  "serious_share": 0.5742697381596559,
  "death_share": 0.09102355155810585
 },
 "recalls": [],
 "recall_total": 0,
 "drugsfda": {
  "applications": 1,
  "nda": 0,
  "anda": 0,
  "first_approval": "20171116",
  "rems": false,
  "rems_last": "",
  "labeling_supplements": 0,
  "labeling_recent": 0,
  "sponsors": [
   "GENENTECH INC"
  ],
  "unapproved": false,
  "first_approval_fmt": "11/16/2017"
 },
 "trials": {
  "total": 57,
  "completed": 16,
  "terminated": 8,
  "with_results": 14,
  "industry": 40,
  "phase3": 20,
  "results_share": 0.875,
  "terminated_share": 0.14035087719298245
 },
 "pubmed": {
  "rct": 7,
  "meta": 11,
  "recent_rct": 3,
  "cochrane": []
 },
 "payments": {
  "year": "2024",
  "usd": 0.0,
  "records": 0,
  "by_name": [],
  "names_checked": [
   "emicizumab"
  ],
  "source": "bulk-index"
 },
 "partd": null,
 "mdl": [],
 "dsc": [],
 "settlements": [],
 "prescrire": null,
 "eml": {
  "listed": true,
  "status": "AddedToEml",
  "name": "emicizumab",
  "matched": "emicizumab",
  "stewardship": []
 },
 "exposure": null,
 "scores": {
  "safety": {
   "score": 65,
   "letter": "C",
   "why": [
    "Carries an FDA boxed warning, the strongest warning the FDA issues (-30)",
    "Death share in reports is near or below the FAERS average (0.6x)",
    "Off-label use is among the five most-reported items: widely used beyond its approved evidence (-5)"
   ],
   "weight": 0.35
  },
  "evidence": {
   "score": 36,
   "letter": "F",
   "why": [
    "7 randomized trials and 11 systematic reviews/meta-analyses indexed in PubMed",
    "On the WHO Model List of Essential Medicines (shown for context; no score effect)",
    "No Cochrane review found (-5)"
   ],
   "weight": 0.25
  },
  "regulatory": {
   "score": 100,
   "letter": "A",
   "why": [
    "No recalls on record since 2016"
   ],
   "weight": 0.15
  },
  "conflict": {
   "score": 100,
   "letter": "A",
   "why": [
    "Pharma paid doctors $0 tied to this drug last year: negligible",
    "Not named in any pending federal MDL"
   ],
   "weight": 0.15
  },
  "options": {
   "score": 40,
   "letter": "F",
   "why": [
    "No generic version approved: brand-only pricing (-30)",
    "No same-class alternatives found: little leverage to switch (-30)"
   ],
   "weight": 0.1
  }
 },
 "total": 66,
 "grade": "C",
 "questions": [
  "This drug carries a boxed warning. Which specific risk does it cover, and does it apply to me?",
  "The most-reported problems in FDA's adverse event data are haemarthrosis, haemorrhage, no adverse event. How would we recognize and handle those?",
  "Off-label use is among the most-reported items for this drug. Is my use on-label, and if not, what evidence supports it?",
  "There is no generic. Is there an older generic in the same class that works as well for my situation?",
  "What result are we expecting, by when, and what is the plan if it does not happen?",
  "If 100 people like me took this drug, how many would actually get better because of it, and how many would be harmed by it? (Doctors call these the number needed to treat and the number needed to harm.)"
 ]
}