COMPANY
Swedish Orphan Biovitrum
FDA-approved therapeutic company Clinical Phase 3
Trial and FDA signals
- FDA complete response letter Received an FDA complete response letter (BLA 761438, letter dated Jun 26, 2026). FDA BLA 761438 (Jun 26, 2026)
Evidence (1 event)
- Jun 26, 2026 · FDA BLA 761438
CRL text (OCR, redacted by FDA; first 200 chars from "COMPLETE RESPONSE"): COMPLETE RESPONSE Swedish Orphan Biovitrum AB (publ) Attention: Bao Le US Regulatory Lead, Immunology Waltham, MA (street omitted) Dear Bao Le: Please refer to your biologics license appli
- Jun 26, 2026 · FDA BLA 761438
Timeline
- Trial start —: Pacritinib Effectiveness in Real-world Settings source ↗
- Trial start Phase 4: Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study source ↗
- Trial start Phase 2/Phase 3: A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment source ↗
- Trial start Phase 4: A Study to Evaluate Efficacy and Safety of Anakinra in Chinese Patients With Colchicine-resistent FMF source ↗
- Trial start Phase 4: An Efficacy and Safety Study of Subcutaneous Anakinra in Chinese Patients With Systemic Juvenile Idiopathic Arthritis (SJIA) and Adult-Onset Still's Disease (AOSD) source ↗
- Trial start —: A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients source ↗
- FDA FDA New indication / efficacy supplement: GAMIFANT (emapalumab-lzsg) — BLA761107/18, Jun 27, 2025. Applicant at approval: Swedish Orphan Biovitrum AB (publ). source ↗
- Trial start —: Assessment of Macrophage Activation syndromE in STill's Disease in Italy source ↗
- Trial start Phase 2: A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) source ↗
- Trial start Phase 4: Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa source ↗
- Trial start Phase 3: Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa source ↗
- Trial start Phase 1: Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A source ↗
- Trial start Phase 4: A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis source ↗
- Trial start —: Assessment of Macrophage Activation syndromE in STill's Disease source ↗
- Trial start Phase 4: An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa source ↗
- Trial start Phase 3: A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa source ↗
- Trial start —: A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) source ↗
- Trial start Phase 4: A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients source ↗
- Trial start Phase 3: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD) source ↗
- Trial start Phase 3: A Study of Anakinra in Japanese Patients With Still's Disease (SJIA and AOSD) source ↗
- Trial start —: An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP source ↗
- Trial start Phase 2: A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation source ↗
- Trial start Phase 3: Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE source ↗
- Trial start Phase 2: Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk Af source ↗
- Trial start —: Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST) source ↗
Upcoming milestones
- Oct 5, 2026 (ESTIMATED) · 3 · A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis · NCT03165734
- Nov 17, 2026 (ESTIMATED) · Phase 4 · A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis · NCT06752850
- Dec 30, 2026 (ESTIMATED) · Phase 2 · A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) · NCT06782373
- Jan 27, 2027 (ESTIMATED) · · Pacritinib Effectiveness in Real-world Settings · NCT07774455
- 2027-03 (ESTIMATED) · Phase 3 · Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa · NCT06716814
- Jun 30, 2028 (ESTIMATED) · Phase 2/Phase 3 · A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment · NCT07786922
- 2028-12 (ESTIMATED) · Phase 4 · Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa · NCT06940830
- Aug 15, 2029 (ESTIMATED) · · A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) · NCT05776472
Clinical trials (lead sponsor) Showing 50 of 80
| Record | Title | Phase | Status | Start | Primary completion |
|---|---|---|---|---|---|
| NCT06405152 | Assessment of Macrophage Activation syndromE in STill's Disease | Not listed | Completed | Sep 27, 2023 | Sep 26, 2024 (actual) |
| NCT06940830 | Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa | Phase 4 | Active, not recruiting | May 21, 2025 | 2028-12 (estimated) |
| NCT04293523 | A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health | Not listed | Completed | Mar 30, 2020 | May 4, 2026 (actual) |
| NCT06782373 | A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) | Phase 2 | Active, not recruiting | May 28, 2025 | Dec 30, 2026 (estimated) |
| NCT07774455 | Pacritinib Effectiveness in Real-world Settings | Not listed | Recruiting | Aug 13, 2026 | Jan 27, 2027 (estimated) |
| NCT07746895 | Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study | Phase 4 | Recruiting | Aug 5, 2026 | Dec 31, 2031 (estimated) |
| NCT05776472 | A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Not listed | Active, not recruiting | Jun 26, 2023 | Aug 15, 2029 (estimated) |
| NCT05744063 | A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients | Phase 4 | Completed | Feb 3, 2023 | Feb 21, 2025 (actual) |
| NCT07786922 | A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment | Phase 2/Phase 3 | Not yet recruiting | Jul 31, 2026 | Jun 30, 2028 (estimated) |
| NCT05001737 | Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE | Phase 3 | Completed | Dec 15, 2021 | Jun 30, 2024 (actual) |
| NCT07706868 | An Efficacy and Safety Study of Subcutaneous Anakinra in Chinese Patients With Systemic Juvenile Idiopathic Arthritis (SJIA) and Adult-Onset Still's Disease (AOSD) | Phase 4 | Withdrawn | Sep 1, 2025 | Sep 30, 2028 (estimated) |
| NCT05817812 | A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa | Phase 3 | Completed | Jul 31, 2023 | Jun 2, 2026 (actual) |
| NCT06666335 | A Study to Evaluate Efficacy and Safety of Anakinra in Chinese Patients With Colchicine-resistent FMF | Phase 4 | Withdrawn | Mar 1, 2026 | Aug 30, 2027 (estimated) |
| NCT06716814 | Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa | Phase 3 | Recruiting | Mar 6, 2025 | 2027-03 (estimated) |
| NCT06579144 | Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A | Phase 1 | Completed | Feb 24, 2025 | May 21, 2026 (actual) |
| NCT06752850 | A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis | Phase 4 | Active, not recruiting | Dec 19, 2024 | Nov 17, 2026 (estimated) |
| NCT03165734 | A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis | 3 | Active, not recruiting | Jun 26, 2017 | Oct 5, 2026 (estimated) |
| NCT06227429 | A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients | Not listed | Withdrawn | 2025-09 | Jun 30, 2027 (estimated) |
| NCT04596540 | A Study of SEL-212 in Patients With Gout Refractory to Conventional Therapy II | Phase 3 | Completed | Nov 30, 2020 | Jan 10, 2023 (actual) |
| NCT06992505 | Assessment of Macrophage Activation syndromE in STill's Disease in Italy | Not listed | Completed | Jun 6, 2025 | Nov 27, 2025 (actual) |
| NCT05148299 | A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation | Phase 2 | Completed | Feb 1, 2022 | Dec 8, 2024 (actual) |
| NCT05096403 | A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD) | Phase 3 | Completed | Oct 20, 2022 | May 27, 2024 (actual) |
| NCT03901755 | An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B | Not listed | Completed | Sep 12, 2019 | Mar 12, 2024 (actual) |
| NCT04513366 | A Study of SEL-212 in Patients With Gout Refractory to Conventional Therapy | Phase 3 | Completed | Aug 18, 2020 | Jul 21, 2022 (actual) |
| NCT04943042 | An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP | Not listed | Completed | Feb 22, 2022 | Jun 10, 2025 (actual) |
| NCT05814159 | A Study of Anakinra in Japanese Patients With Still's Disease (SJIA and AOSD) | Phase 3 | Active, not recruiting | Aug 24, 2022 | Dec 10, 2025 (estimated) |
| NCT05856266 | An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa | Phase 4 | Terminated | Aug 24, 2023 | Dec 6, 2023 (actual) |
| NCT02976753 | Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products | Not listed | Completed | 2016-12 | Nov 3, 2021 (actual) |
| NCT03055611 | A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B | Not listed | Completed | May 9, 2017 | Apr 19, 2022 (actual) |
| NCT06047210 | Evaluation of IFNγ and Inflammatory Mediators in Patients With Hemophagocytic Lymphohistiocytosis | Not listed | Completed | Dec 14, 2016 | Oct 30, 2019 (actual) |
| NCT03951103 | rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A | Not listed | Completed | Nov 7, 2018 | Sep 30, 2022 (actual) |
| NCT04133883 | Impact on French Physician's Haemophilia Treatment Management Decision Based on Systematic Joint Examination | Not phased (device or behavioral study) | Completed | Jan 13, 2020 | Jul 13, 2022 (actual) |
| NCT03655340 | A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France | Not listed | Completed | Sep 12, 2018 | Mar 16, 2022 (actual) |
| NCT03103542 | Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies | Phase 4 | Completed | Aug 29, 2017 | Sep 4, 2019 (actual) |
| NCT02320084 | Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care | Not listed | Completed | 2013-09 | Sep 30, 2019 (actual) |
| NCT03932344 | Long-term Safety Study of Kineret® in Patients With Systemic Juvenile Idiopathic Arthritis (SJIA) | Not listed | Completed | Apr 10, 2019 | Aug 31, 2019 (actual) |
| NCT04731701 | Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST) | Not listed | Completed | May 5, 2021 | Jul 19, 2022 (actual) |
| NCT03259230 | Evaluation of IFNγ and Inflammatory Mediators in Patients With Malignancy-Associated Hemophagocytic Lymphohistiocytosis | Not listed | Completed | Oct 13, 2016 | Jan 29, 2020 (actual) |
| NCT03312751 | Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis | Phase 3 | Completed | Feb 6, 2019 | Aug 18, 2021 (actual) |
| NCT03276130 | Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and B | Not listed | Completed | Oct 30, 2018 | Jan 30, 2020 (actual) |
| NCT04731298 | Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After HSCT. | Phase 2 | Terminated | May 25, 2021 | Apr 21, 2022 (actual) |
| NCT04765553 | A Study to Evaluate Emapalumab in Japanese Healthy Volunteers. | Phase 1 | Completed | Jan 14, 2021 | Jun 25, 2021 (actual) |
| NCT03985423 | A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Emapalumab in Adult Patients With HLH | Phase 2/Phase 3 | Terminated | Jun 2, 2020 | Jun 29, 2021 (actual) |
| NCT01459562 | First in Human Study of an Anti-IFN Gamma Monoclonal Antibody in Healthy Volunteers | Phase 1 | Completed | 2011-09 | 2013-01 (actual) |
| NCT01818492 | Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis | Phase 2/Phase 3 | Completed | 2013-07 | 2019-01 (actual) |
| NCT04494061 | A Clinical Study to Investigate Interferon Gamma (IFNɣ) Signature in Patients Post HSCT and in Patients With Impaired HSC Proliferation Pre-transplant | Not listed | Terminated | Nov 16, 2020 | Aug 31, 2022 (actual) |
| NCT02069899 | A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody | Phase 2/Phase 3 | Completed | Aug 4, 2014 | May 18, 2021 (actual) |
| NCT03311854 | A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH) | Phase 2 | Completed | Feb 20, 2018 | May 19, 2020 (actual) |
| NCT04324021 | Efficacy and Safety of Emapalumab and Anakinra in Reducing Hyperinflammation and Respiratory Distress in Patients With COVID-19 Infection. | Phase 2/Phase 3 | Terminated | Apr 2, 2020 | Nov 13, 2020 (actual) |
| NCT03811028 | A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients | Phase 1/Phase 2 | Completed | Jan 19, 2019 | Apr 30, 2021 (actual) |
FDA records (last 2 years)
- FDA New indication / efficacy supplement: GAMIFANT (emapalumab-lzsg) — BLA761107/18, Jun 27, 2025. Applicant at approval: Swedish Orphan Biovitrum AB (publ). source ↗
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