Fitusiran

Fitusiran is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in people aged twelve years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors (neutralizing antibodies).

The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). The label also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with fitusiran or after a dose increase of fitusiran.

The efficacy and safety of fitusiran were assessed in two multicenter, randomized clinical trials which enrolled a total of 177 adult and pediatric male participants with either hemophilia A or hemophilia B. In one study, participants had inhibitory antibodies to coagulation factor VIII or coagulation factor IX and previously received on-demand treatment with medicines known as "bypassing agents" for bleeding. In the second study, participants did not have inhibitory antibodies to coagulation factor VIII or coagulation factor IX and previously received on-demand treatment with clotting factor concentrates. In the two randomized trials, participants received either a fixed dose of fitusiran monthly or their usual on-demand treatment (bypassing agents or clotting factor concentrates) as needed for nine months. The fixed dose of fitusiran is not approved because it led to excessive clotting in some participants.

The US Food and Drug Administration (FDA) granted the application for fitusiran orphan drug and fast track designations. The FDA granted the approval of Qfitlia to Sanofi.

Fitusiran was approved for medical use in the United States in March 2025.{{cite press release | title=Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors | website=Sanofi | date=28 March 2025 | url=https://www.news.sanofi.us/2025-03-28-Qfitlia-approved-as-the-first-therapy-in-the-US-to-treat-hemophilia-A-or-B-with-or-without-inhibitors | access-date=29 March 2025}}

Fitusiran is the international nonproprietary name.{{cite journal | vauthors = ((World Health Organization)) | year = 2016 | title = International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75 | journal = WHO Drug Information | volume = 30 | issue = 1 | hdl = 10665/331046 | hdl-access = free | author-link = World Health Organization }}

Fitusiran is sold under the brand name Qfitlia.

{{reflist}}

{{refbegin}}

• {{cite journal | vauthors = Srivastava A, Rangarajan S, Kavakli K, Klamroth R, Kenet G, Khoo L, You CW, Xu W, Malan N, Frenzel L, Bagot CN, Stasyshyn O, Chang CY, Poloskey S, Qiu Z, Andersson S, Mei B, Pipe SW | title = Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase 3 trial | journal = The Lancet. Haematology | volume = 10 | issue = 5 | pages = e322–e332 | date = May 2023 | pmid = 37003278 | doi = 10.1016/S2352-3026(23)00037-6 | url = https://eprints.soton.ac.uk/477603/1/Fitusiran_003_ATLAS_INH_revised_4.0_tracked.docx }}
• {{cite journal | vauthors = Young G, Kavakli K, Klamroth R, Matsushita T, Peyvandi F, Pipe SW, Rangarajan S, Shen MC, Srivastava A, Sun J, Tran HA, You CW, Zülfikar BB, Menapace LA, Zhang C, Shen Y, Puurunen M, Demissie M, Kenet G | title = Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study | journal = Blood | volume = | issue = | pages = | date = March 2025 | pmid = 40053895 | doi = 10.1182/blood.2024027008 | doi-access = free }}
• {{cite journal | vauthors = Young G, Srivastava A, Kavakli K, Ross C, Sathar J, You CW, Tran H, Sun J, Wu R, Poloskey S, Qiu Z, Kichou S, Andersson S, Mei B, Rangarajan S | title = Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial | journal = Lancet | volume = 401 | issue = 10386 | pages = 1427–1437 | date = April 2023 | pmid = 37003287 | doi = 10.1016/S0140-6736(23)00284-2 | url = https://eprints.soton.ac.uk/477636/1/Srivastava_Phase_3_ATLAS_AB_manuscript_Lancet_Haem_R2.docx }}

{{refend}}

• {{ClinicalTrialsGov|NCT03417102|A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors (ATLAS-INH)}}
• {{ClinicalTrialsGov|NCT03417245|A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors}}
• {{ClinicalTrialsGov|NCT03754790|Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX (ATLAS-OLE)}}

{{Antithrombotics}}

{{Portal bar | Medicine}}

{{Authority control}}

Category:Anticoagulants

Category:Orphan drugs