F8 coagulation factor VIII
ENSG00000185010 · protein coding · Open Targets profile
Factor VIII, along with calcium and phospholipid, acts as a cofactor for F9/factor IXa when it converts F10/factor X to the activated form, factor Xa.
Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.
Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.
Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.
Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.
Tractability
- Small molecule
- Structure with Ligand, Med-Quality Pocket
- Antibody
- Advanced Clinical, GO CC high conf, UniProt loc med conf, UniProt SigP or TMHMM
- PROTAC
- Small Molecule Binder
- Other modalities
- Approved Drug
gnomAD loss of function constraint: o/e 0.08532600104808807 (upper bound 0.13824999332427979). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.
Who runs trials of these drugs
Principal investigators and study chairs on 103 registry trials, updated in the last three years, of the most advanced drugs against F8 (efanesoctocog alfa, susoctocog alfa, turoctocog alfa pegol, damoctocog alfa pegol, simoctocog alfa, turoctocog alfa, antihemophilic factor, pegylated (mw 20000) human sequence recombinant, lonoctocog alfa, moroctocog alfa, efmoroctocog alfa, giroctocogene fitelparvovec, omfiloctocog alfa), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Renchi YangInstitute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College. · 5 of 5 trials Phase 2+
- Lei ZhangInstitute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College · 2 of 5 trials Phase 2+
- Robert SidonioEmory University · 2 of 2 trials Phase 2+
- Akhil R. BiswasDhaka Medical College · 1 of 1 trials Phase 2+
- Amy D ShapiroIndiana Hemophilia &Thrombosis Center, Inc. · 1 of 1 trials Phase 2+
- Aziz EghbaliIran University of Medical Sciences · 1 of 1 trials Phase 2+
- Jessica GarciaUniversity of Texas Southwestern Medical Center · 1 of 1 trials Phase 2+
- Johannes OldenburgExperimental Haematology and Transfusion Medicine, University Clinic Bonn · 1 of 1 trials Phase 2+
- Martin SiegemundUniversity Hospital, Basel, Switzerland · 1 of 1 trials Phase 2+
- Michael Ken WoodwardInstituto Grifols SA · 1 of 1 trials Phase 2+
- Miguel ESCOBARThe University of Texas Health Science Center, Houston · 1 of 1 trials Phase 2+
- NUCHANUN KESSAKORNKing Chulalongkorn Memorial Hospital · 1 of 1 trials Phase 2+
- Pey-Jen YuNorthwell Health · 1 of 1 trials Phase 2+
- Pratima ChowdaryRoyal Free London NHS Foundation Trust · 1 of 1 trials Phase 2+
- Sandra CamprubiInstituto Grifols, S.A. · 1 of 1 trials Phase 2+
Registry trials of efanesoctocog alfa, susoctocog alfa, turoctocog alfa pegol, damoctocog alfa pegol, simoctocog alfa, turoctocog alfa, antihemophilic factor, pegylated (mw 20000) human sequence recombinant, lonoctocog alfa, moroctocog alfa, efmoroctocog alfa, giroctocogene fitelparvovec, omfiloctocog alfa updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 52 of 99 have all sites in one country.
Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. 4 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
38 trials of drugs against F8 were stopped early; 6 give no reason
Terminated, withdrawn and suspended interventional trials of efanesoctocog alfa, susoctocog alfa, turoctocog alfa pegol, damoctocog alfa pegol, simoctocog alfa, turoctocog alfa, antihemophilic factor, pegylated (mw 20000) human sequence recombinant, lonoctocog alfa, moroctocog alfa, efmoroctocog alfa, giroctocogene fitelparvovec, omfiloctocog alfa, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 25 most recent of 38.
- withdrawn · 2026-11-06 · Emory University · matched by the registry's drug synonyms“The study was withdrawn because the pharmaceutical sponsor withdrew support for the study. No participants were enrolled before termination.”
- terminated · 2026-10-15 · Octapharma · matched by the registry's drug synonyms“Study was terminated due to SADRs”
- terminated · 2026-09-30 · Grifols Biologicals, LLC · matched by the registry's drug synonyms“Due to recruitment challenges during the trial, this study was terminated early by the Sponsor.”
- terminated · 2026-07-31 · Laboratoire français de Fractionnement et de Biotechnologies · matched by the registry's drug synonyms“Feasibility considerations - extended timelines to complete recruitment”
- withdrawn · 2026-05-07 · Ezeldeen Abuelkasem · matched by the registry's drug synonyms“No supply of the test substance was provided by the sponsor.”
- withdrawn · 2026-04-07 · University of Texas Southwestern Medical Center · matched by the registry's drug synonyms“Due to COVID-19 pandemic, recruitment was temporarily put on hold. We are withdrawing this registration due to a recent amendment to the protocol that includes major changes to data elements. A new registration will be submitted for the new protocol.”
- terminated · 2025-08-22 · BioMarin Pharmaceutical · matched by the registry's drug synonyms“Early rollover into long-term extension study”
- terminated · 2025-01-16 · Swedish Orphan Biovitrum · matched by the registry's drug synonyms“The Sponsor has decided to stop the phase 4 study Sobi.HAEM89-007 (JOIN-us) due to a realignment of its development activities. The clinical trial hasn't been terminated due to a change in the benefit-risk balance.”
- withdrawn · 2024-12-13 · Spark Therapeutics, Inc. · matched by the registry's drug synonyms“Strategic Decision”
- terminated · 2024-09-04 · Eli Lilly and Company · matched by the registry's drug synonyms“SIG-001 programme terminated”
- withdrawn · 2024-01-05 · Emory University · matched by the registry's drug synonyms“The Emi PUPs and Nuwiq ITI study has been closed due to slow enrollment and study site startup.”
- terminated · 2023-04-11 · Margaret Ragni · matched by the registry's drug synonyms“The INHIBIT Trials IDSMB, in a letter dated 05-18-22, recommended, given the slow enrollment, that the INHIBIT Trials be discontinued due to futility.”
- terminated · 2023-02-21 · Margaret Ragni · matched by the registry's drug synonyms“The INHIBIT Trials IDSMB, in a letter dated 05-18-22, recommended, given the slow enrollment, that the INHIBIT Trials be discontinued due to futility.”
- terminated · 2022-06-15 · Wyeth is now a wholly owned subsidiary of Pfizer · Moroctocog alfa“See termination reason in detailed description.”
- terminated · 2021-11-23 · Grifols Therapeutics LLC · matched by the registry's drug synonyms“Terminated due to limited enrollment (non-safety related decision)”
- terminated · 2021-10-20 · Baxalta now part of Shire · matched by the registry's drug synonyms“Takeda has determined that the benefit/risk profile does not support continuation of the surgery Study for this specific Congenital Hemophilia A with Inhibitors patient population.”
- terminated · 2021-05-24 · Baxalta now part of Shire · matched by the registry's drug synonyms“It became unlikely to achieve the study objective of 50% reduction over published inhibitor rates. The Data Monitoring Committee supported this decision.”
- terminated · 2021-05-14 · Baxalta now part of Shire · matched by the registry's drug synonymsNo reason given
- withdrawn · 2020-12-17 · City of Hope Medical Center · matched by the registry's drug synonyms“complete per PI”
- terminated · 2020-09-25 · Craig Seaman · matched by the registry's drug synonyms“Insufficient enrollment”
- terminated · 2020-07-07 · Novo Nordisk A/S · Turoctocog alfa“The trial was terminated as the participant withdrew from the trial.”
- terminated · 2020-06-16 · PTC Therapeutics · matched by the registry's drug synonyms“Study was terminated early due to Sponsor decision and not reflective of adverse safety findings.”
- terminated · 2020-05-29 · University of California, Davis · matched by the registry's drug synonyms“Termination of funding from sponsor.”
- withdrawn · 2019-10-22 · Bayer · matched by the registry's drug synonyms“Company decision”
- withdrawn · 2019-08-20 · Margaret Ragni · matched by the registry's drug synonyms“The trial was revised to be two protocols, one Prevention, one Eradication Trials.”
Paediatric incentives and obligations for F8's drugs
For efanesoctocog alfa, susoctocog alfa, turoctocog alfa pegol, damoctocog alfa pegol, simoctocog alfa, turoctocog alfa, antihemophilic factor, pegylated (mw 20000) human sequence recombinant, lonoctocog alfa, moroctocog alfa, efmoroctocog alfa, giroctocogene fitelparvovec, omfiloctocog alfa: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.
- FDA pediatric target list: no · F8 is not on the listRule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
- EU paediatric investigation plan: yes · EMEA-002724-PIP01-19-M03 (Giroctocogene fitelparvovec, Treatment of Haemophilia A): PM: decision on the application for modification of an agreed PIP, 6 May 2024; EMEA-002501-PIP01-18-M03 (Efanesoctocog alfa, Treatment of Haemophilia A): PM: decision on the application for modification of an agreed PIP, 10 Oct 2022; compliance positive 26 Apr 2023; EMEA-001215-PIP01-11-M07 (lonoctocog alfa, Haemophilia A (congenital Factor VIII deficiency)): PM: decision on the application for modification of an agreed PIP, 10 Sept 2019; compliance positive 10 Sept 2021; EMEA-001174-PIP02-12-M02 (turoctocog alfa pegol, Treatment of hereditary factor VIII deficiency): PM: decision on the application for modification of an agreed PIP, 7 Jun 2017; compliance positive 23 Jul 2021; +4 moreRule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
- EU orphan designation: yes · EU/3/19/2176 positive 28 Jun 2019: Treatment of haemophilia A; EU/3/12/995 withdrawn 26 Apr 2012: Treatment of haemophilia A; EU/3/10/847 withdrawn 23 Feb 2011: Treatment of haemophilia A; EU/3/10/783 withdrawn 20 Sept 2010: Treatment of haemophilia ARule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
- Authorised in the EU: yes · Jivi: authorised since 22 Nov 2018; Afstyla: authorised since 4 Jan 2017; Altuvoct: authorised since 17 Jun 2024, orphan medicine; Elocta: authorised since 18 Nov 2015; +6 moreRule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
- FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the linkRule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
- FDA pediatric oncology approval: no · FDA's table lists no pediatric approval of efanesoctocog alfa, susoctocog alfa, turoctocog alfa pegol, damoctocog alfa pegol, simoctocog alfa, turoctocog alfa, antihemophilic factor, pegylated (mw 20000) human sequence recombinant, lonoctocog alfa, moroctocog alfa, efmoroctocog alfa, giroctocogene fitelparvovec, omfiloctocog alfaRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-12
- Registered studies open to children: yes · 210 interventional studies of efanesoctocog alfa, susoctocog alfa, turoctocog alfa pegol, damoctocog alfa pegol, simoctocog alfa, turoctocog alfa, antihemophilic factor, pegylated (mw 20000) human sequence recombinant, lonoctocog alfa, moroctocog alfa, efmoroctocog alfa, giroctocogene fitelparvovec, omfiloctocog alfa include patients under 18; 27 are recruiting, not yet recruiting or activeRule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-12
Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs
All 16 drugs and clinical candidates as a table
Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.