Qfitlia (fitusiran) / Sanofi, Alnylam - LARVOL DELTA

Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B. (PubMed, Cochrane Database Syst Rev) - "Evidence from RCTs shows that prophylaxis using non-clotting factor therapies compared with on-demand treatment may reduce bleeding events, increase the percentage of individuals with zero bleeds, increase the incidence of non-serious adverse events, and improve HRQoL. Comparative assessments with other prophylaxis regimens, assessment of long-term joint outcomes, and assessment of economic outcomes will improve evidence-based decision-making for the use of these therapies in bleed prevention."

Clinical • Journal • Review • Cystic Fibrosis • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Immunology • Oncology • Pain • Pulmonary Disease • Rare Diseases • Respiratory Diseases

ATLAS-KIDS: A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B (clinicaltrials.gov) - P3 | N=85 | Recruiting | Sponsor: Sanofi

Trial initiation date • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Overview on Management of Breakthrough Bleeds and Major Surgeries Following Bleed Management Guidelines During the Fitusiran Clinical Program in People With Hemophilia (THSNA 2026) - P3 | "Similarly, reduced doses of CFC/BPA were effectively used for perioperative management of 47 (78.3%) major surgeries, and four major surgeries were conducted without perioperative CFC/BPA. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Clinical • Surgery • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases • Thrombosis

Major Orthopedic Surgeries Conducted Under Fitusiran Prophylaxis in People With Hemophilia A and B, With and Without Inhibitors (THSNA 2026) - "Postoperative thrombosis was reported in one participant with pre-existing increased thrombotic risk when dosing exceeded BMG, highlighting the importance of BMG adherence. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Surgery • Cardiovascular • Genetic Disorders • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Musculoskeletal Diseases • Obesity • Orthopedics • Rare Diseases • Thrombosis

Major Dental Surgeries Conducted Under Fitusiran Prophylaxis in People With Hemophilia A and B, With and Without Inhibitors (THSNA 2026) - P3 | "Tranexamic acid, an antifibrinolytic, was administered during four (21%) major dental surgeries. Reversal of the effects of fitusiran with administration of ATIII is not necessary. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Surgery • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases • Thrombosis

Fitusiran: A Novel Antithrombin-Targeting Therapy for Hemophilia A and B, With or Without Inhibitors (THSNA 2026) - "Fitusiran expands prophylactic options for hemophilia A and B, providing sustained bleed protection with infrequent dosing. Integration into practice requires monitoring of antithrombin activity to mitigate thrombotic or bleeding risk, and liver and gallbladder functions for safety. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Transaminase Elevations in the Fitusiran Clinical Development Program With the Antithrombin-Based Dose Regimen (THSNA 2026) - P1/2, P3 | "The majority of liver transaminase elevations >3x ULN were mild to moderate, transient, and resolved without treatment. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Clinical • Gastrointestinal Disorder • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Hepatology • Liver Failure • Metabolic Dysfunction-Associated Steatohepatitis • Rare Diseases

Evaluating the Safety and Tolerability of Switching from Emicizumab to Fitusiran Prophylaxis in Males Aged ≥12 Years with Severe Hemophilia A, with and without Inhibitors: SWITCH Study (THSNA 2026) - P4 | "Study recruitment is ongoing. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Clinical • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Biliary Events in the Fitusiran Clinical Development Program With the Antithrombin-Based Dose Regimen (THSNA 2026) - P1/2, P3 | "Additional studies assessing the AT-DR with fitusiran are ongoing and planned. No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author."

Clinical • Gastroenterology • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Hepatology • Rare Diseases

Lessons from clinical trials on monitoring antithrombin activity in the setting of fitusiran therapy (THSNA 2026) - No abstract available

Clinical

Novel drugs approved by the EMA, the FDA and the MHRA in 2025: A year in review. (PubMed, Br J Pharmacol) - "Among the FIC drugs, it is worth mentioning the Nav1.8 channel inhibitor suzetrigine, the first non-opioid approved to palliate acute pain; the first positive allosteric modulator of transient receptor potential melastatin 8 (TRPM8), acoltremon, that increases basal tear production in dry eye disease, a globally common disorder; lerodalcibep, a 'third generation' adnectin inhibitor of the protease Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) to treat elevated LDL-c; and zoliflodacin and gepotidacin, both innovatively targeting bacterial topoisomerases to treat uncomplicated urinary tract infections. These approaches include: the combination of two FIC drugs, the RAF/MEK clamp avutometinib paired with the FAK/Pyk2 inhibitor defactinib, to block more efficiently the RAS-RAF-MEK-ERK/FAK oncogenic pathway in low-grade serous ovarian cancer; fitusiran, the first RNAi therapy for haemophilia, targeting for the first time the production of the natural..."

European regulatory • FDA event • Journal • Review • Bronchiectasis • Dry Eye Disease • Hematological Disorders • Hemophilia • Infectious Disease • Nephrology • Oncology • Ophthalmology • Ovarian Cancer • Ovarian Serous Adenocarcinoma • Pain • Pulmonary Disease • Rare Diseases • Respiratory Diseases • Solid Tumor • NAV1 • TRPM8

EFC17905: A study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged 1 to less than 12 years with hemophilia A or B. (clinicaltrialsregister.eu) - P2/3 | N=26 | Not yet recruiting | Sponsor: Sanofi-Aventis Recherche & Developpement

New P2/3 trial • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Comparative Efficacy and Safety of Non-Clotting Factor Prophylaxis Versus. on-Demand Therapy in Hemophilia: A Meta-Analysis of Randomized Controlled Trials. (PubMed, Clin Appl Thromb Hemost) - "Prophylaxis increased the likelihood of achieving zero treated bleeds [RR = 4.11; 95% CI: (1.48%, 11.45%), I2 = 88.5%, p < 0.0001]. An exploratory network meta-analysis comparing fitusiran, emicizumab, and concizumab reported no statistically significant difference in the ABR for all treated bleeds.ConclusionCompared to on-demand therapy, non-factor prophylactic therapies significantly reduce bleeding episodes, improve quality of life, and increase the likelihood of zero bleeds in patients with hemophilia."

Journal • Retrospective data • Review • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

COST-EFFECTIVENESS OF FITUSIRAN PROPHYLAXIS VERSUS FACTOR VIII ON-DEMAND OR PROPHYLACTIC THERAPY FOR HEMOPHILIA A PATIENTS WITHOUT INHIBITORS (ISPOR 2026) - "From a US payer perspective, fitusiran prophylaxis was a cost-effective and cost-saving strategy for hemophilia A without inhibitors, providing superior health outcomes at lower overall cost compared with on-demand and prophylactic FVIII therapy."

Clinical • Cost effectiveness • HEOR • Hematological Disorders • Hemophilia • Hemophilia A • Rare Diseases

2025 FDA TIDES (Peptides and Oligonucleotides) Harvest. (PubMed, Pharmaceuticals (Basel)) - "Fitusiran and donidalorsen are the first oligonucleotide therapies approved for antithrombin deficiency and hereditary angioedema, respectively, while plozasiran represents the second approved therapy for familial chylomicronemia syndrome...In 2025, elamipretide further expanded this paradigm by becoming the first disease-specific treatment approved for Barth syndrome. This review provides an overview of TIDES approved in 2025, with emphasis on their chemical structures, medical targets, modes of action, routes of administration, and associated adverse effects."

Journal • Review • Cardiovascular • Complement-mediated Rare Disorders • Familial Chylomicronemia Syndrome • Hereditary Angioedema

Challenges in Balancing Hemostasis and Thrombosis in Therapy Tailoring for Hemophilia: A Narrative Review. (PubMed, Int J Mol Sci) - "Therapeutic approaches, including innovative NRTs, such as emicizumab, or rebalancing agents (e.g., concizumab, marstacimab, fitusiran), offer promising advancements in bleeding prophylaxis but may increase thrombotic risks. FXI inhibition emerges as a paradigm shift in thrombosis management, offering reduced bleeding risks while preserving vascular health. Finally, this review highlights the need for global laboratory assays to personalize treatments, emphasizing strategies to optimize safety and efficacy, particularly as hemophilia patients live longer with complex comorbidity profiles."

Journal • Review • Acute Coronary Syndrome • Atrial Fibrillation • Cardiovascular • Hematological Disorders • Hemophilia • Hemophilia A • Human Immunodeficiency Virus • Infectious Disease • Rare Diseases • Thrombosis

QFitlia (Fitusiran): redefining hemophilia treatment with RNAi therapy. A correspondence. (PubMed, Ann Med Surg (Lond)) - No abstract available

Journal • Hematological Disorders • Hemophilia • Rare Diseases

2025: strong sales and EPS growth. (GlobeNewswire) - "ALTUVIIIO (haemophilia A) sales were €324 million of which 85% was in the US...Rest of World sales of €48 million benefited from the launches in Japan and Taiwan...Rezurock (chronic graft-versus-host disease) sales were €113 million and decreased by 6.1%...Sales in Europe were -€4 million, mainly from a one-time credit in the UK. In Rest of World, sales were €12 million, the overwhelming majority from the launch in China; Cablivi (acquired thrombotic thrombocytopenic purpura) sales were €69 million and increased by 1.4%, driven by more patients being treated in the US and Europe offset by an element of price impact in the US....Wayrilz (immune thrombocytopenia) sales were €6 million, all in the US, following approval in August 2025; Qfitlia (haemophilia A and B) sales were €4 million, all in the US, following approval in March 2025."

Sales • Chronic Graft versus Host Disease • Hemophilia A • Hemophilia B • Immune Thrombocytopenic Purpura

ATLAS-NEO: A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia (clinicaltrials.gov) - P3 | N=91 | Active, not recruiting | Sponsor: Sanofi | Trial completion date: Jun 2028 ➔ Jan 2029

Trial completion date • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Redefining hemophilia management: treatment goals, nonfactor replacement therapies, and the role of fitusiran. (PubMed, Am J Manag Care) - "Current therapeutic guidelines, last updated in 2020, recommend recombinant clotting factor concentrates as the standard replacement therapy, with emicizumab offered as an alternative nonfactor standard of care. As this and other therapeutic advancements redefine the standard of care for patients with hemophilia, managed care and policy frameworks must adapt to ensure timely access and affordability. Likewise, updates to clinical guidelines and care pathways are essential to capture the rapidly evolving therapeutic landscape."

Journal • Hematological Disorders • Hemophilia • Rare Diseases

ATLAS-KIDS: A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B (clinicaltrials.gov) - P3 | N=85 | Recruiting | Sponsor: Sanofi | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Fitusiran (Qfitlia) as a first-in-class RNA interference therapeutic: targeting antithrombin for prophylactic hemostasis in hemophilia. (PubMed, Ann Med Surg (Lond)) - No abstract available

Journal • Hematological Disorders • Hemophilia • Rare Diseases

Rebalancing Hemostasis: Fitusiran as a First-in-Class RNAi Therapy in Hemophilia A and B. (PubMed, Health Sci Rep) - "Its RNAi-based, factor-independent mechanism enables durable bleed protection and improved treatment convenience. While long-term hepatic safety and thrombotic risk require continued surveillance, fitusiran represents a significant advance in hemophilia management and a promising step toward more accessible, individualized, and rebalancing-based therapies."

Journal • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Pain • Rare Diseases

Fitusiran: A Novel Antithrombin-Targeting Therapy for Hemophilia A and B, With or Without Inhibitors. (PubMed, Ann Pharmacother) - "Fitusiran expands prophylactic options for hemophilia A and B, providing sustained bleed protection with infrequent dosing. Integration into practice requires monitoring of antithrombin activity to mitigate thrombotic or bleeding risk, and liver and gallbladder functions for safety."

Journal • Review • Hematological Disorders • Hemophilia • Hemophilia A • Hemophilia B • Rare Diseases

Qfitlia Receives SFDA Registration For Hemophilia A Or B In Saudi Arabia (OneArabia)

Filing • Hemophilia A • Hemophilia B