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.
Clinical phenotype terms— hover any for plain English:
- Reduced factor VIII activityHP:0003125
- Bleeding with minor or no traumaHP:0011889
- ThromboembolismHP:0001907
- Spontaneous hematomasHP:0007420
- Oral cavity bleedingHP:0030140
- Abnormality of the elbowHP:0009811
- Intramuscular hematomaHP:0012233
- Intraventricular hemorrhageHP:0030746
- Splenic ruptureHP:0012223
FDA & Trial Timeline
10 eventsOctapharma — PHASE4
Hoffmann-La Roche — PHASE1, PHASE2
St. Jude Children's Research Hospital
Novo Nordisk A/S — PHASE1
Northwell Health — PHASE2
Pfizer
Novo Nordisk A/S — PHASE1
Novo Nordisk A/S
Runhui WU — PHASE4
Swedish Orphan Biovitrum — PHASE1
Data is compiled from FDA regulatory filings and ClinicalTrials.gov, then processed through automated extraction; event classifications and dates may occasionally be misclassified. Verify against the linked FDA filing or trial record before clinical decisions. Updated periodically.
Treatments
7 FDA-approved · 4 other trackedSource: openFDA + DailyMed · NDA / BLA labels with structured indications · refreshed weekly
FDA-approved (7)
ALTUVIIIO
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
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
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
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
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
treatment and prevention of bleeding in hemophilia A (classic hemophilia)
Qfitlia
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
Other tracked therapies (4)
Medications tracked in our therapeutics database that are not FDA-approved for Hemophilia A via a labelled indication. May include investigational, off-label, or supportive therapies. Always verify with a clinician before use.
Tranexamic Acid, TRANEXAMIC ACID
• 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
• 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
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
• 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
Source: ClinicalTrials.gov · synced daily · phases, status, and PI names normalized at ingest
Source: NPI Registry + PubMed · trial PI roles cross-referenced with ClinicalTrials.gov · NORD partners highlighted · ordered by verified-leader / active-researcher / listed-specialist tier
Specialty unconfirmed
Emory University
Specialty unconfirmed
Universidad de Oviedo
Specialty unconfirmed
Peking University Institute of Hematology, Peking University People's Hospital
Specialty unconfirmed
CSL Behring
Specialty unconfirmed
Hematology Hospital, Chinese Academy of Medical Sciences
Specialty unconfirmed
Centre Hospitalier Universitaire de Saint Etienne
Specialty unconfirmed
University of Washington
Specialty unconfirmed
Octapharma
Rare Disease Specialist
Sobi Investigational Site
Specialty unconfirmed
Iranian Blood Transfusion Research Center
Specialty unconfirmed
Baqiyatallah Research Center for Gastroenterology and Liver Diseases
Specialty unconfirmed
Shanghai Xinzhi BioMed Co., Ltd.
Specialty unconfirmed
Gastroenterology and Liver Diseases, Gastrointestinal and Liver Diseases Research Center (GLDRC), Guilan University of Medical Sciences, Rasht, Iran
Specialty unconfirmed
Nantes University Hospital
Specialty unconfirmed
Iranian blood Transfusion Research Center
Specialty unconfirmed
Research Center for Gastroenterology and Hepatology, Tabriz University of Medical Sciences, Tabriz
Specialty unconfirmed
Royal Free Hospital NHS Foundation Trust
Specialty unconfirmed
Baqiyatallah Research Center for Gastroenterology and Liver Diseases
Specialty unconfirmed
Mary M. Gooley Hemophilia Center
Specialty unconfirmed
Baqiyatallah Research Center for Gastroenterology and Liver Diseases
Specialty unconfirmed
Spark Therapeutics, Inc.
Specialty unconfirmed
staff
Specialty unconfirmed
Khyber Medical University Peshawar
Specialty unconfirmed
The National Hemophilia Center Ministry of Health Sheba Medical Center Ramat Gan, Israel
Specialty unconfirmed
Gazi University
Treatment Centers
8 centersSource: NORD Rare Disease Centers + NIH Undiagnosed Diseases Network (UDN) · centers verified active within last 12 months
Children's Hospital Colorado Rare Disease Program ↗
Children's Hospital Colorado
📍 Aurora, CO
👤 Boston Children's Hospital Rare Disease Program
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDBoston Children's Hospital Rare Disease Program ↗
Boston Children's Hospital
📍 Boston, MA
👤 Boston Children's Hospital Rare Disease Program
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
🏨 Children'sAnn & Robert H. Lurie Children's Hospital Genetics ↗
Lurie Children's Hospital
📍 Chicago, IL
👤 Boston Children's Hospital Rare Disease Program
🏥 NORDCincinnati Children's Hospital Medical Center ↗
Cincinnati Children's
📍 Cincinnati, OH
👤 Boston Children's Hospital Rare Disease Program
🏨 Children'sNationwide Children's Hospital Rare Disease Center ↗
Nationwide Children's Hospital
📍 Columbus, OH
👤 Boston Children's Hospital Rare Disease Program
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
Financial Resources
4 resourcesSource: manufacturer patient-assistance programs (PAP) + copay-card programs · NORD Patient Assistance · HealthWell Foundation + disease-specific foundation grants · links verified by automated cron
Travel Grants
No travel grants are currently matched to Hemophilia A.
Community
No community posts yet. Be the first to share your experience with Hemophilia A.
Start the conversation →Latest news about Hemophilia A
5 articlesSource: PubMed + NIH RePORTER + openFDA + clinical-journal RSS · last 30 days · disease-tagged at ingest by AI extraction with human QC
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 — 20 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.
Conditions related to Hemophilia A
Other rare diseases that share clinical features, genetic basis, or diagnostic-code family with Hemophilia A. These are starting points for further reading, not a substitute for a clinician's assessment.
Frequently asked questions about Hemophilia A
Auto-generated from canonical disease facts (Orphanet, OMIM, ClinicalTrials.gov, openFDA, NPPES). Not a substitute for clinical guidance.
What is Hemophilia A?
Hemophilia A is a rare disease catalogued in international rare-disease ontologies (Orphanet ORPHA:98878, OMIM 306700). Inheritance pattern depends on the specific subtype. For verified primary sources, see the UniteRare Hemophilia A page.
Are there FDA-approved treatments for Hemophilia A?
Yes — UniteRare tracks 11 FDA-approved treatments with indications relevant to Hemophilia A. Each entry includes prescribing information, orphan-drug-designation status where applicable, and the FDA application number for verification.
Are there clinical trials recruiting for Hemophilia A?
UniteRare currently lists 20 clinical trials relevant to Hemophilia A sourced from ClinicalTrials.gov. Each trial entry includes recruitment status, eligibility criteria summary, principal-investigator information, and study locations. Patients should discuss eligibility with their healthcare provider before enrolling.
How do I find a specialist for Hemophilia A?
UniteRare lists 25 verified clinicians with documented expertise in Hemophilia A, sourced from ClinicalTrials.gov principal-investigator records, PubMed publication histories, and the NPPES NPI registry. Filter by state or browse our state-specific specialist pages for nearby options.
See full Hemophilia A page for complete clinical details, sources, and verified-specialist listings.
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