Hemophilia A

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ORPHA:98878OMIM:306700D66
Who is this for?
Show terms as
11FDA treatments19Active trials53Specialists8Treatment centers4Financial resources

Where are you in your journey?

UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Hemophilia A is treated with 9 medications in our database, including tranexamic acid, ALTUVIIIO, Tranexamic Acid in Sodium Chloride, ROCTAVIAN, Hemlibra, and 4 more. 4 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by "Avenacy, Inc.", Sanofi, Exela, BioMarin, Genentech and others. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Hemophilia A treatment below.

Also known as:

Clinical phenotype terms— hover any for plain English:

Reduced factor VIII activityHP:0003125Bleeding with minor or no traumaHP:0011889ThromboembolismHP:0001907Spontaneous hematomasHP:0007420Oral cavity bleedingHP:0030140Abnormality of the elbowHP:0009811Intramuscular hematomaHP:0012233Intraventricular hemorrhageHP:0030746Splenic ruptureHP:0012223
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Apr 2026Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery

Octapharma — PHASE4

TrialRECRUITING
Mar 2026A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

Hoffmann-La Roche — PHASE1, PHASE2

TrialNOT YET RECRUITING
Mar 2026Liver Biopsy Following Gene Therapy For Hemophilia

St. Jude Children's Research Hospital

TrialNOT YET RECRUITING
Nov 2025A Research Study Looking at How Food Intake Affects Inno8 in the Body of Healthy People

Novo Nordisk A/S — PHASE1

TrialRECRUITING
Nov 2025Factor VIII Inhibitor Bypass Activity (FEIBA) Versus Fresh Frozen Plasma As First Line Therapy For Bleeding After Cardiac Surgery

Northwell Health — PHASE2

TrialRECRUITING
Oct 2025A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.

Pfizer

TrialACTIVE NOT RECRUITING
Oct 2025A Research Study Looking at How Different Doses of Study Medicine (Inno8) Work in the Body of People With Haemophilia A

Novo Nordisk A/S — PHASE1

TrialRECRUITING
May 2025Post-marketing Surveillance (Special Use-results Surveillance) on Treatment With Alhemo

Novo Nordisk A/S

TrialENROLLING BY INVITATION
Mar 2025Assessing Different FVIII Doses and Frequencies in Immune Tolerance Induction (ITI) with ADVATE Among Hemophilia a Boys with Inhibitor (INITIATE Study)

Runhui WU — PHASE4

TrialRECRUITING
Feb 2025Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A

Swedish Orphan Biovitrum — PHASE1

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

11 available

ALTUVIIIO

ANTIHEMOPHILIC FACTOR (RECOMBINANT), FC-VWF-XTEN FUSION PROTEIN-EHTL· Bioverativ Therapeutics Inc.Orphan Drug
1 INDICATIONS AND USAGE ALTUVIIIO is indicated for use in adults and pediatric patients with hemophilia A (congenital factor VIII deficiency) for: Routine prophylaxis to reduce the frequency of bleedi

1 INDICATIONS AND USAGE ALTUVIIIO is indicated for use in adults and pediatric patients with hemophilia A (congenital factor VIII deficiency) for: Routine prophylaxis to reduce the frequency of bleeding episodes On-demand treatment and control of bleeding episodes Perioperative management of bleeding ALTUVIIIO [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl] is a recombinant DNA-derived, Factor VIII concentrate indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for: Routine prophylaxis to reduce the frequency of bleeding episodes On-demand treatment & control of bleeding episodes Perioperative management of bleeding ( 1 ) Limitation of Use: ALTUVIIIO is not indicated for the treatment of von Willebrand disease. ( 1 ) Limitation of Use ALTUVIIIO is not indicated for the treatment of von Willebrand disease.

ROCTAVIAN

VALOCTOCOGENE ROXAPARVOVEC-RVOX· BioMarin Pharmaceutical Inc.Orphan Drug
1 INDICATIONS AND USAGE ROCTAVIAN is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor V

1 INDICATIONS AND USAGE ROCTAVIAN is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. ROCTAVIAN is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. ( 1 )

Hemlibra

EMICIZUMAB· Genentech, Inc.■ Boxed WarningOrphan Drug
1 INDICATIONS AND USAGE 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 (

1 INDICATIONS AND USAGE 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 (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 )

Alhemo

CONCIZUMAB· Novo Nordisk
routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A (congenital factor VIII deficiency) with or with

routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A (congenital factor VIII deficiency) with or without FVIII inhibitors

HYMPAVZI

MARSTACIMAB-HNCQ· Pfizer Laboratories Div Pfizer IncOrphan Drug
1 INDICATIONS AND USAGE HYMPAVZI is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with: • hemophil

1 INDICATIONS AND USAGE HYMPAVZI is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with: • hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors, or • hemophilia B (congenital factor IX deficiency) without factor IX inhibitors. HYMPAVZI is a tissue factor pathway inhibitor (TFPI) antagonist indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with: • hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors, or • hemophilia B (congenital factor IX deficiency) without factor IX inhibitors. ( 1 )

Humate-P

Antihemophilic factor/von Willebrand factor complex (human), dried, pasteurized· CSL Behring

treatment and prevention of bleeding in hemophilia A (classic hemophilia)

Qfitlia

FITUSIRAN· Genzyme Corporation■ Boxed Warning

routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or B with or without factor VIII or IX inhibitors

Tranexamic Acid, TRANEXAMIC ACID

· Pfizer

• Patient Copay Amount: Not Publicly Available • Maximum Annual Benefit Limit: Not Publicly Available • Core Eligibility Restrictions: Not Publicly Available • RxBIN, PCN, and Group numbers: Not Pu

TRANEXAMIC ACID

TRANEXAMIC ACID· Pfizer

• Patient Copay Amount: Not Publicly Available • Maximum Annual Benefit Limit: Not Publicly Available • Core Eligibility Restrictions: Not Publicly Available • RxBIN, PCN, and Group numbers: Not Pu

TRANEXAMIC ACID IN SODIUM CHLORIDE

TRANEXAMIC ACID· Exela

Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo

Cyklokapron

tranexamic acid· Pfizer

• Patient Copay Amount: Not Publicly Available • Maximum Annual Benefit Limit: Not Publicly Available • Core Eligibility Restrictions: Not Publicly Available • RxBIN, PCN, and Group numbers: Not Publi

Clinical Trials

19 recruitingView all trials with filters →
Phase 34 trials
A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa
Phase 3
Active
PI: Study Physician, MD, PhD (Swedish Orphan Biovitrum AB) · Sites: Vienna; Zagreb +18 more · Age: 1299 yrs
Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Participants With Hemophilia A Previously Treated With FVIII Products
Phase 3
Actively Recruiting
PI: Study Director (CSL Behring) · Sites: Hefei, Anhui; Beijing, Beijing Municipality +18 more · Age: 065 yrs
Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults
Phase 3
Active
PI: Pfizer CT.gov Call Center (Pfizer) · Sites: Mountain View, California; Palo Alto, California +34 more · Age: 1864 yrs
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Phase 3
Actively Recruiting
PI: Pfizer CT.gov Call Center (Pfizer) · Sites: Salt Lake City, Utah; Mendoza +64 more · Age: 117 yrs
Phase 44 trials
Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery
Phase 4
Actively Recruiting
PI: Johannes Oldenburg (Experimental Haematology and Transfusion Medicine,) · Sites: San Antonio, Texas; Helsinki +13 more · Age: 1299 yrs
Hemlibra in Mild Hemophilia A
Phase 4
Actively Recruiting
PI: Amy D Shapiro, MD (Indiana Hemophilia &Thrombosis Center, Inc.) · Sites: Indianapolis, Indiana · Age: 545 yrs
Pharmacokinetic-guided Dosing of Emicizumab
Phase 4
Actively Recruiting
PI: Study Officials Fischer, Dr, MD (UMC Utrecht) · Sites: Nijmegen, Gelderland; Maastricht, Limburg +6 more · Age: 199 yrs
Assessing Different FVIII Doses and Frequencies in Immune Tolerance Induction (ITI) with ADVATE Among Hemophilia a Boys with Inhibitor (INITIATE Study)
Phase 4
Actively Recruiting
· Sites: Beijing, Beijing Municipality · Age: 018 yrs
Phase 11 trial
Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A
Phase 1
Actively Recruiting
PI: Elena Santagostino, MD (Sobi AB) · Sites: Sofia; Frankfurt +6 more · Age: 1865 yrs
N/A1 trial
Embolization in Hereditary Coagulopathies
N/A
Actively Recruiting
PI: Fabiane E FArias, PT (IOT-HC-FMUSP (Orthopedics Institute - General Hosp) · Sites: São Paulo
Other6 trials
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Active
PI: Pfizer CT.gov Call Center (Pfizer) · Sites: Tokyo
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan
Actively Recruiting
· Sites: Changhua; Kaohsiung City +8 more
Treatment of Hemophilia A Patients With FVIII Inhibitors
Actively Recruiting
PI: Robert Sidonio, MD, MSc (Emory University) · Sites: Atlanta, Georgia; Mörfelden-Walldorf
Hemophilia A Research Program
Actively Recruiting
PI: Jill M Johnsen, MD (University of Washington) · Sites: Seattle, Washington
Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
Actively Recruiting
PI: Rebecca Kruse-Jarres, MD, MPH (Washington Institute for Coagulation) · Sites: Los Angeles, California; San Diego, California +2 more · Age: 1699 yrs
Prevalence of Synovitis in Patients With Haemophilia A
Actively Recruiting
PI: Andreas C Strauss, PD Dr. med. (University Hospital, Bonn) · Sites: Bonn, North Rhine-Westphalia · Age: 1899 yrs

Specialists

Showing 25 of 53View all specialists →
RM
Robert Sidonio, Jr., MD
ATLANTA, GA
Specialist
PI on 1 active trial
EM
Elena Santagostino, MD
Sofia
Specialist

Rare Disease Specialist

PI on 3 active trials
MD
Marc TROSSAERT, Dr
Specialist
PI on 1 active trial
BM
Brigitte Tardy, MD
Specialist
PI on 4 active trials
PM
Peter A Kouides, MD
ROCHESTER, NY
Specialist
PI on 1 active trial
PD
Program Director
Specialist
PI on 9 active trials
SP
Sigurd Knaub, PhD
Specialist
PI on 4 active trials
CM
Caifeng Yang, Master
Specialist
PI on 1 active trial
RM
Rebecca Kruse-Jarres, MD, MPH
SEATTLE, WA
Specialist
PI on 3 active trials
XM
Xiao-Hui Zhang, MD
Specialist
PI on 16 active trials
PM
Paul Batty, MBBS MRCP
ROCKFORD, IL
Specialist
PI on 1 active trial
TM
Tiffany Chang, MD
Specialist
PI on 1 active trial
RY
Renchi Yang
Specialist
PI on 6 active trials165 Hemophilia A publications
WB
Wan Jung Kao, bachelor
Specialist
PI on 1 active trial
BM
Bita Behnava, M.D.
Specialist
PI on 1 active trial
FM
Fariborz Mansour-Ghanaei, M.D.
Specialist
PI on 1 active trial
MM
Maryam Keshvari, M.D.
Specialist
PI on 1 active trial
MM
Mohammad Hossein Somi, M.D.
Specialist
PI on 1 active trial
SM
Seyyed Mohammad Miri, M.D.
Specialist
PI on 1 active trial
PM
Pegah Karimi, M.D.
Specialist
PI on 1 active trial
BM
Bashir HajiBeigi, M.D.
Specialist
PI on 1 active trial
SF
Savita Rangarajan, FRCP FRCPath
Specialist
PI on 1 active trial
EW
Eric Wolford
CHARLESTON, SC
Specialist
PI on 3 active trials
ND
Nevin Atalay Güzel, Prof Dr
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Financial Resources

4 resources
ALTUVIIIO(ANTIHEMOPHILIC FACTOR (RECOMBINANT), FC-VWF-XTEN FUSION PROTEIN-EHTL)Bioverativ Therapeutics Inc.
ROCTAVIAN(VALOCTOCOGENE ROXAPARVOVEC-RVOX)BioMarin Pharmaceutical Inc.
Hemlibra(EMICIZUMAB)Genentech, Inc.
Alhemo(CONCIZUMAB)Novo Nordisk

Travel Grants

No travel grants are currently matched to Hemophilia A.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Hemophilia A

5 articles
Clinical trialRSSApr 24, 2026
Single infusion of Roctavian linked to fewer bleeds in hemophilia A
A new treatment called Roctavian was given as a single injection to men with severe hemophilia A (a bleeding disorder). Seven years later, most patients had few
ResearchRSSApr 22, 2026
Regular exercise safely eases chronic pain in hemophilia: Study
A new study shows that regular exercise — including activities like walking, strength training, and balance work — is safe and helpful for people with hemophili
ResearchRSSApr 22, 2026
AI may help hemophilia patients detect joint bleeding at home
Researchers developed an artificial intelligence tool that can accurately detect bleeding inside joints in adults with hemophilia by analyzing ultrasound images
ResearchPUBMEDApr 22, 2026
Can German Health Insurance Claims Data Fill Information Gaps in Rare Chronic Diseases: Use Case of Haemophilia A.
Researchers in Germany studied health insurance records from 2017-2019 to understand how hemophilia A (a bleeding disorder) affects patients and how much it cos
Clinical trialUNITERAREApr 3, 2026
New Recruiting Trial: Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery
This is a clinical trial testing a blood clotting medicine called Nuwiq in women and girls with hemophilia A who need surgery. Hemophilia A is a rare bleeding d
See all news about Hemophilia A

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Hemophilia A

What is Hemophilia A?

Hemophilia A is treated with 9 medications in our database, including tranexamic acid, ALTUVIIIO, Tranexamic Acid in Sodium Chloride, ROCTAVIAN, Hemlibra, and 4 more. 4 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by "Avenacy, Inc.", Sanofi, Exela, BioMarin, Genentech and others. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Hemophilia A treatment below.

Are there clinical trials for Hemophilia A?

Yes — 19 recruiting clinical trials are currently listed for Hemophilia A on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Hemophilia A?

25 specialists and care centers treating Hemophilia A are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Hemophilia A?

7 FDA-approved treatments and 3 patient support programs are currently tracked on UniteRare for Hemophilia A. See the treatments and support programs sections for copay assistance, eligibility, and contact details.