F9 coagulation factor IX
ENSG00000101981 · protein coding · Open Targets profile
Factor IX is a vitamin K-dependent plasma protein that participates in the intrinsic pathway of blood coagulation by converting factor X to its active form in the presence of Ca(2+) ions, phospholipids, and factor VIIIa (PubMed:8295821, PubMed:2592373, PubMed:20121197, PubMed:20121198, PubMed:1730085, PubMed:19846852, PubMed:39880037). {ECO:0000269|PubMed:1730085, ECO:0000269|PubMed:19846852, ECO:0000269|PubMed:20121197, ECO:0000269|PubMed:20121198, ECO:0000269|PubMed:2592373, ECO:0000269|PubMed:39880037, ECO:0000269|PubMed:8295821}.
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 Withdrawn means the drug was pulled from the market or from development.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, High-Quality Ligand, Druggable Family
- Antibody
- UniProt loc high conf, GO CC high conf, UniProt SigP or TMHMM
- PROTAC
- Half-life Data, Small Molecule Binder
- Other modalities
- Approved Drug
gnomAD loss of function constraint: o/e 0.058598000556230545 (upper bound 0.18445999920368195). 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 91 registry trials, updated in the last three years, of the most advanced drugs against F9 (valoctocogene roxaparvovec, etranacogene dezaparvovec, nonacog beta pegol, coagulation factor ix recombinant human, albutrepenonacog alfa, emicizumab, eftrenonacog alfa, prothrombin complex concentrate, trenonacog alfa, pegnivacogin), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Lei ZhangInstitute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College · 2 of 6 trials Phase 2+
- Amy D ShapiroIndiana Hemophilia &Thrombosis Center, Inc. · 2 of 2 trials Phase 2+
- Robert SidonioEmory University · 2 of 2 trials Phase 2+
- Steven PipeUniversity of Michigan · 2 of 2 trials Phase 2+
- Akhil R. BiswasDhaka Medical College · 1 of 1 trials Phase 2+
- Caifeng YangShanghai Xinzhi BioMed Co., Ltd. · 1 of 1 trials Phase 2+
- Ginger NicolWashington University School of Medicine · 1 of 1 trials Phase 2+
- Jake RachieleUniversity of Miami · 1 of 1 trials Phase 2+
- Jessica GarciaUniversity of Texas Southwestern Medical Center · 1 of 1 trials Phase 2+
- Jia ShiChinese Academy of Medical Sciences, Fuwai Hospital · 1 of 1 trials Phase 2+
- Kathirvel SurbramaniamUniversity of Pittsburgh Medical Center · 1 of 1 trials Phase 2+
- Kenichi TanakaUniversity of Maryland, Baltimore · 1 of 1 trials Phase 2+
- Khaled MohamedMedexus Pharma, Inc. · 1 of 1 trials Phase 2+
- Lijian PeiPeking Union Medical College Hospital · 1 of 1 trials Phase 2+
- Luis T Da LuzSunnybrook Health Sciences Centre · 1 of 1 trials Phase 2+
Registry trials of valoctocogene roxaparvovec, etranacogene dezaparvovec, nonacog beta pegol, coagulation factor ix recombinant human, albutrepenonacog alfa, emicizumab, eftrenonacog alfa, prothrombin complex concentrate, trenonacog alfa, pegnivacogin 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; 50 of 89 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. 2 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
38 trials of drugs against F9 were stopped early; 4 give no reason
Terminated, withdrawn and suspended interventional trials of valoctocogene roxaparvovec, etranacogene dezaparvovec, nonacog beta pegol, coagulation factor ix recombinant human, albutrepenonacog alfa, emicizumab, eftrenonacog alfa, prothrombin complex concentrate, trenonacog alfa, pegnivacogin, 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.
- terminated · 2026-11-10 · University of Maryland, Baltimore · matched by the registry's drug synonyms“Standard practice change prohibited enrollment”
- withdrawn · 2026-11-06 · Emory University · Emicizumab“The study was withdrawn because the pharmaceutical sponsor withdrew support for the study. No participants were enrolled before termination.”
- terminated · 2026-10-21 · University Health Network, Toronto · matched by the registry's drug synonyms“Upon IDSMC's review of interim data, they recommended the trial be stopped based on futility.”
- terminated · 2026-10-05 · Pfizer · matched by the registry's drug synonyms“The study was terminated early due to Sponsor and participants may transition into a long-term follow-up study. The decision to terminate the trial was not based on any safety and/or efficacy concerns.”
- terminated · 2026-07-31 · Spur Therapeutics · matched by the registry's drug synonyms“Freeline has decided to pause development of FLT180a for business priorities”
- terminated · 2026-07-24 · AstraZeneca · matched by the registry's drug synonyms“The study terminated because the clinical development programme for Ceralasertib has been discontinued.”
- terminated · 2026-06-25 · Octapharma · matched by the registry's drug synonyms“Following Interim Analysis IDMEAC concluded:1-No safety concerns 2-Suspend enrolment: Futility based on treatment effect size at interim analysis. Enrollment was suspended on 22DEC25. Study was terminated FEB2026. Final analysis is planned in Q2 2026”
- terminated · 2026-06-24 · SHI Jia · Prothrombin Complex Concentrate, Human“At a meeting on February 17, 2025, the Data and Safety Monitoring Board recommended early termination because the predefined efficacy boundary was crossed.”
- withdrawn · 2026-04-07 · University of Texas Southwestern Medical Center · Emicizumab“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 · 2025-12-10 · CSL Behring · matched by the registry's drug synonyms“Study was terminated early because mortality was lower than expected and a significant increase in sample size would have been required to meet statistical goals of the study. No safety reasons were involved in the decision to terminate the study.”
- terminated · 2025-08-22 · BioMarin Pharmaceutical · Valoctocogene Roxaparvovec“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 · 2024-07-19 · Sangamo Therapeutics · matched by the registry's drug synonymsNo reason given
- terminated · 2024-04-18 · Hospices Civils de Lyon · matched by the registry's drug synonyms“More Hardware”
- terminated · 2024-04-11 · Indiana Hemophilia &Thrombosis Center, Inc. · Emicizumab“Global COVID-19 pandemic prevented further study visits or enrollment.”
- 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 · 2023-07-20 · Spur Therapeutics · matched by the registry's drug synonyms“Freeline has decided to pause development of FLT180a for business priorities”
- terminated · 2023-04-11 · Margaret Ragni · Emicizumab Injection [Hemlibra]“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 · Emicizumab“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 · 2022-12-08 · Aspen Global Incorporated · matched by the registry's drug synonyms“Prolonged delay as a result of impact of Covid19 pandemic”
- withdrawn · 2022-12-05 · Mayo Clinic · matched by the registry's drug synonyms“Patient population changed and no subjects eligible for the study.”
- terminated · 2022-12-02 · University College, London · matched by the registry's drug synonyms“Terminated early due to challenges during the COVID-19 pandemic and a change in requirements of data to be submitted for marketing authorisation.”
- terminated · 2021-11-03 · IRCCS Policlinico S. Donato · Prothrombin Complex Concentrate“The study encountered unexpected difficulties in enrolling patients due to requirements for the Informed Consent. 60% of our patients comes from abroad with only one accompanying parent. The rules of our EC require both parents to sign the consent.”
- terminated · 2021-03-15 · Medexus Pharma, Inc. · matched by the registry's drug synonymsNo reason given
Paediatric incentives and obligations for F9's drugs
For valoctocogene roxaparvovec, etranacogene dezaparvovec, nonacog beta pegol, coagulation factor ix recombinant human, albutrepenonacog alfa, emicizumab, eftrenonacog alfa, prothrombin complex concentrate, trenonacog alfa, pegnivacogin: 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 · F9 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-002722-PIP01-19-M03 (Etranacogene dezaparvovec, Treatment of Haemophilia B): PM: decision on the application for modification of an agreed PIP, 8 Sept 2023; EMEA-002427-PIP01-18-M02 (valoctocogene roxaparvovec, Treatment of Haemophilia A): PM: decision on the application for modification of an agreed PIP, 12 May 2023; EMEA-001107-PIP01-10-M04 (albutrepenonacog alfa, Treatment of hereditary factor IX deficiency): PM: decision on the application for modification of an agreed PIP, 29 Jan 2020; EMEA-001839-PIP01-15 (emicizumab, Treatment of hereditary factor VIII deficiency): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 15 Jul 2016; compliance positive 26 Apr 2019; +3 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/3/18/1999 positive 21 Mar 2018: Treatment of haemophilia B; EU/3/09/723 positive 4 Feb 2010: Treatment of haemophilia B; EU/3/09/640 expired 15 May 2009: Treatment of haemophilia B; EU/3/13/1221 withdrawn 16 Jan 2014: Treatment of haemophilia A; +1 moreRule: 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 · Hemlibra: authorised since 23 Feb 2018; Idelvion: authorised since 11 May 2016, orphan medicine; Hemgenix: authorised since 20 Feb 2023, orphan medicine, conditional; Alprolix: authorised since 12 May 2016, orphan medicine; +4 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 valoctocogene roxaparvovec, etranacogene dezaparvovec, nonacog beta pegol, coagulation factor ix recombinant human, albutrepenonacog alfa, emicizumab, eftrenonacog alfa, prothrombin complex concentrate, trenonacog alfa, pegnivacoginRule: 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 · 111 interventional studies of valoctocogene roxaparvovec, etranacogene dezaparvovec, nonacog beta pegol, coagulation factor ix recombinant human, albutrepenonacog alfa, emicizumab, eftrenonacog alfa, prothrombin complex concentrate, trenonacog alfa, pegnivacogin include patients under 18; 22 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 12 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.