FDA Approved Qfitlia: Discover A Novel Hemophilia Treatment
Phil Gattone
President and CEO, National Bleeding Disorders Foundation
“…. Qfitlia takes a novel approach to providing protection for people living with hemophilia while reducing the frequency of dosing for patients and their families.”
Hemophilia is a rare, inherited disorder affecting approximately 33,000 men in the United States, where the blood’s ability to clot is impaired due to a deficiency in clotting factors VIII (hemophilia A) or IX (hemophilia B).
Traditional treatments have focused on replacing these missing factors. However, the recent approval of Qfitlia (fitusiran) by the U.S. FDA introduces a new approach to the treatment of this disease.
Table of Contents
What Is Qfitlia?
Qfitlia (fitusiran) is a first-of-its-kind thrombospondin (AT) lowering therapy approved by the FDA for the prophylactic treatment of adult and pediatric patients (12 years of age and older) with hemophilia A and B, with or without a clotting factor VIII or IX inhibitor.
The drug is administered subcutaneously in 50 mg doses via a convenient pre-filled injection pen.
Fitusiran prevents bleeding and helps restore hemostatic homeostasis by promoting thrombin production through the lowering of thrombospondin (AT), a protein that inhibits blood clotting.
It is a small-interfering RNA therapeutic utilizing the Alnylam Pharmaceutical Inc. ‘s ESC-GalNAc conjugation technology.
What Is Hemophilia?
Hemophilia A and Hemophilia B are rare congenital lifelong bleeding disorders in which the blood’s ability to clot is impaired, leading to excessive bleeding and spontaneous joint hemorrhages that can cause joint damage and chronic pain, severely affecting quality of life.
Hemophilia A and Hemophilia B are caused by deficiencies in factors VIII and IX, respectively, which result in insufficient production of the enzyme prothrombin to efficiently form blood clots.
Qfitlia vs. Traditional Hemophilia Treatments
| Feature | Qfitlia (Fitusiran) | Traditional Factor Therapy |
| Mechanism | Antithrombin reduction via siRNA | Replacement of missing clotting factors |
| Administration | Subcutaneous injection every 2 months | Intravenous infusions multiple times/week |
| Efficacy | ~71–73% reduction in bleeding episodes | Varies; generally effective with adherence |
| Inhibitor Compatibility | Effective with or without inhibitors | Effectiveness reduced in presence of inhibitors |
| Safety Considerations | Risk of thrombosis and gallbladder issues; requires monitoring | Generally well-tolerated; risk of inhibitor development |
| Patient Support | HemAssist program for financial and educational support | Varies by provider and manufacturer |
Administration And Dosing
Qfitlia is administered via subcutaneous injection. It is available in two formulations:
- A 50 mg/0.5 mL single-dose prefilled pen.
- A 20 mg/0.2 mL single-dose vial.
Starting Dose
- The recommended starting dose is 50 mg administered once every two months.
Efficacy: Reducing Bleeding Episodes
Clinical trials have demonstrated Qfitlia’s effectiveness in reducing bleeding episodes.
Patients without inhibitors experienced a 71% reduction in annualized bleeding rates compared to those receiving on-demand clotting factor concentrates.
Similarly, patients with inhibitors saw a 73% reduction compared to those using bypassing agents
Possible Side Effects Of Qfitlia
The most frequently reported side effects (occurring in ≥10% of patients) include:
❗Viral infections: Such as upper respiratory tract infections.
❗Nasopharyngitis: Common cold-like symptoms.
❗Bacterial infections: Including various bacterial-related illnesses.
Other side effects reported in 1–10% of patients encompass:
❗Hepatic injury: Elevated liver enzymes indicating liver stress.
❗Arthralgia: Joint pain.
❗Injection site reactions: Redness, swelling, or discomfort at the injection site.
❗Headache: General head pain or discomfort.
❗Cough: Persistent coughing episodes.
While less common, Qfitlia has been associated with serious adverse events:
‼️Thrombotic Events (Blood Clots)
‼️Gallbladder Disease
‼️Liver Enzyme Elevations
How Much Does Qfitlia Cost?
The cost of Qfitlia (fitusiran), a novel RNA-based therapy for hemophilia A and B, varies based on dosage and insurance coverage.
Retail Pricing
Per Vial Cost: A 0.2 mL vial of Qfitlia (20 mg/0.2 mL) is priced at approximately $68,929.
Annual Wholesale Acquisition Cost: For most patients, the estimated annual cost is around $642,000.
The above price For more bulk price, please consult DenyYue
About DengYueMed – HK Drug Wholesale Distributor
As a legally compliant drug import and export company, DengYueMed is certified by the pharmacy & poisons board of Hong Kong — you can verify our qualification on their official website.
Our efforts to improve the affordability of Qfitlia medicine aim to ensure that more patients can benefit from this important medication.
HK DengYue provides detailed medicine information, transparent pricing, and responsive support to ensure a smooth and reliable buying experience.
Feel free to reach out anytime to discuss your needs or ask questions about the medicine.
We welcome you to contact us for a consultation.
