Fitusiran
| Clinical data | |
|---|---|
| Trade names | Qfitlia |
| Other names | ALN-AT3SC |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a625058 |
| License data | |
| Routes of administration | Subcutaneous |
| Drug class | Anthithrombin production inhibitor |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C520H636F21N175Na43O309P43S6 |
| Molar mass | 17193.48 g·mol−1 |
Fitusiran, sold under the brand name Qfitlia, is a medication used for the treatment of hemophilia.[1] It is an antithrombin-directed small interfering ribonucleic acid.[1] It is given by subcutaneous injection.[1] Fitusiran reduces the amount of a protein called antithrombin.[2]
The most common side effects include viral infection, common cold symptoms (nasopharyngitis) and bacterial infection.[2]
Fitusiran was approved for medical use in the United States in March 2025.[2] The US Food and Drug Administration considers it to be a first-in-class medication.[3][4]
Medical uses
[edit]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).[1][2]
Adverse effects
[edit]The US prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal).[2] 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.[2]
History
[edit]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.[2] 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.[2] 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.[2] 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.[2] The fixed dose of fitusiran is not approved because it led to excessive clotting in some participants.[2]
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.
Society and culture
[edit]Legal status
[edit]Fitusiran was approved for medical use in the United States in March 2025.[2][5]
Names
[edit]Fitusiran is the international nonproprietary name.[6]
References
[edit]- 1 2 3 4 5 6 "Qfitlia- fitusiran injection, solution". DailyMed. 16 September 2025. Retrieved 22 July 2026.
- 1 2 3 4 5 6 7 8 9 10 11 12 13 "FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors". U.S. Food and Drug Administration. 28 March 2025. Retrieved 29 March 2025.
This article incorporates text from this source, which is in the public domain. - ↑ "Novel Drug Approvals for 2025". U.S. Food and Drug Administration (FDA). 8 July 2026. Retrieved 19 July 2026.
- ↑ "Advancing Health Through Innovation: New Drug Therapy Approvals 2025" (PDF). U.S. Food and Drug Administration (FDA). January 2026.
- ↑ "Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors". Sanofi (Press release). 28 March 2025. Retrieved 29 March 2025.
- ↑ World Health Organization (2016). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 75". WHO Drug Information. 30 (1). hdl:10665/331046.
Further reading
[edit]- Srivastava A, Rangarajan S, Kavakli K, Klamroth R, Kenet G, Khoo L, et al. (May 2023). "Fitusiran prophylaxis in people with severe haemophilia A or haemophilia B without inhibitors (ATLAS-A/B): a multicentre, open-label, randomised, phase 3 trial". The Lancet. Haematology. 10 (5): e322–e332. doi:10.1016/S2352-3026(23)00037-6. PMID 37003278.
- Young G, Kavakli K, Klamroth R, Matsushita T, Peyvandi F, Pipe SW, et al. (March 2025). "Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study". Blood. doi:10.1182/blood.2024027008. PMID 40053895.
- Young G, Srivastava A, Kavakli K, Ross C, Sathar J, You CW, et al. (April 2023). "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". Lancet. 401 (10386): 1427–1437. doi:10.1016/S0140-6736(23)00284-2. PMID 37003287.
External links
[edit]- Clinical trial number NCT03417102 for "A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors (ATLAS-INH)" at ClinicalTrials.gov
- Clinical trial number NCT03417245 for "A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors" at ClinicalTrials.gov
- Clinical trial number NCT03754790 for "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)" at ClinicalTrials.gov
