The first antithrombin-lowering (AT) treatment for routine prophylaxis to prevent or lessen the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B, with or without factor VIII or IX inhibitors, is Qfitlia (fitusiran), which has been approved by the US Food and Drug Administration (FDA). The data from the ATLAS phase 3 studies, which showed clinically significant bleed protection as determined by annualized bleeding rates (ABR) among hemophilia patients with and without inhibitors, served as the basis for the approval.
Current treatment options can make people with hemophilia feel they need to choose between effective bleed control and convenient dosing schedules, leading to trade-offs when it comes to disease management. Qfitlia takes a novel approach to providing protection for people living with hemophilia while reducing the frequency of dosing for patients and their families.
Phil Gattone
In order to help hemophiliacs regain hemostasis, Qfitlia helps boost thrombin production by decreasing AT, a protein that prevents blood clotting. Subcutaneous dosage, low volume injections, and low treatment frequency are made possible by Qfitlia’s use of small-interfering RNA technology.
This approval highlights our commitment to advancing innovation and improving care for the rare blood disorders community. Qfitlia has the potential to meaningfully change the hemophilia landscape through effective bleed protection, infrequent dosing, and simplified administration. Our robust portfolio of hemophilia treatment options continues to grow as we focus on offering protection with reduced treatment burden that best fits an individual’s needs.
Brian Foard
With as little as six injections annually, Qfitlia showed low bleed rates across subgroups in the ATLAS clinical development study.
Significant side effects, such as hepatotoxicity, acute and recurring gallbladder illness, and thrombotic events, are also possible. Bacterial infection, nasopharyngitis, and viral infection are the most frequent adverse effects (incidence >10%).
The FDA approved Siemens Healthineers’ INNOVANCE® Antithrombin assay as a companion diagnostic for Qfitlia to measure AT levels concurrently with the approval of Qfitlia. Patients prescribed Qfitlia will have free access to the FDA-approved companion test to measure AT levels through the Qfitlia Testing Program with Labcorp.
Of all the prophylactic treatments, Qfitlia can provide the fewest dosages, and its cost will be similar to that of other prophylactic hemophilia treatments. Together with Qfitlia, HemAssist is launched to offer full patient support services, such as financial and insurance help and instructional materials. Patients who have been prescribed Qfitlia or other Sanofi hemophilia medications are eligible for this program.
For hemophilia A and B, the FDA awarded Qfitlia Orphan Drug Designation, Fast Track Designation for hemophilia A and B with and without factor VIII or IX inhibitors, and Breakthrough Therapy Designation for hemophilia B with factor IX inhibitors. In Brazil, a regulatory application for Qfitlia is being reviewed for the treatment of hemophilia A or B in adults and adolescents with or without inhibitors. China is anticipated to make a regulatory decision in the latter part of 2025.
ATLAS clinical development program
The ATLAS clinical development program is looking into Qfitlia’s safety and effectiveness. Completed phase 3 studies ATLAS-INH (NCT03417102), ATLAS-A/B (NCT03417245), and ATLAS-PPX (NCT03549871) are included in the program. Three phase 3 trials are now underway: ATLAS-PEDS (NCT03974113), ATLAS-NEO (NCT05662319), and ATLAS OLE (NCT03754790).
In order to assess the safety and effectiveness of Qfitlia with a modified AT-based dosage regimen (AT-DR), which was created to maintain an AT target range of 15%–35% in patients who have already finished a previous phase 3 ATLAS clinical trial, the ATLAS-OLE research is under underway. It is a single-arm, phase 3, open-label investigation. Compared to other Qfitlia research, this one uses lower dosages and less frequent dosing. By contrasting the AT-DR treatment data from ATLAS-OLE with the control data from ATLAS-INH and ATLAS-A/B trials, the effectiveness of Qfitlia AT-DR treatment was evaluated. The intent-to-treat principle is adhered to throughout the analyses.
Qfitlia
For the prophylactic treatment of adults and pediatric patients (aged 12 and older) with hemophilia A and B, with or without factor VIII or IX inhibitors, the US FDA has approved Qfitlia (fitusiran), a first-in-class AT lowering therapy. The 50 mg dose is administered subcutaneously using a convenient, prefilled pen. By reducing AT, a protein that slows blood clotting, Qfitlia promotes the production of thrombin, preventing bleeding and aiding in the rebalancing of hemostasis. Qfitlia is a small interference RNA treatment that makes use of the ESC-GalNAc conjugate technology developed by Alnylam Pharmaceutical Inc.
Source: Sanofi
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Graduated from the University of Kerala with B.Sc. Botany and Biotechnology. Attained Post-Graduation in Biotechnology from the Kerala University of Fisheries and Ocean Science (KUFOS) with the third rank. Conducted various seminars and attended major Science conferences. Done 6 months of internship in ICMR – National Institute of Nutrition, Hyderabad. 5 years of tutoring experience.
