Living with hemophilia means constantly facing the risk of uncontrollable bleeding. For those affected by this genetic disorder, even a minor injury can have serious consequences. Now, there's a new development: the Food and Drug Administration (FDA) has approved Qfitlia, a treatment that could change the lives of many people.
What is Hemophilia?
Hemophilia is an inherited condition that prevents blood from clotting properly. Instead of stopping quickly, a cut or bruise can continue bleeding because the body lacks an essential clotting protein. There are two main types:
- Hemophilia A: This is caused by a deficiency of factor VIII.
- Hemophilia B: In this case, the problem is a lack of factor IX.
People with hemophilia must be cautious, as even minor injuries can lead to prolonged bleeding, increasing the risk of long-term damage, especially in muscles and joints.
Qfitlia: A Treatment to Reduce Bleeding Episodes
The FDA has approved Qfitlia (fitusiran) as a preventive therapy to reduce the frequency of bleeding episodes in patients with hemophilia A or B, with or without inhibitors to factors VIII or IX. It is administered via a subcutaneous injection, which can be given as infrequently as once every two months, simplifying treatment compared to more frequent therapies.
How Does Qfitlia Work?
Unlike traditional treatments that replace the missing clotting factor, Qfitlia works differently: it reduces antithrombin, increasing thrombin levels—an enzyme crucial for clotting. This mechanism helps compensate for the deficiency of factors VIII and IX, lowering the risk of bleeding.
What Do Clinical Studies Say?
The approval is based on two clinical trials involving 177 adult and pediatric patients. Results showed that in the group of patients with inhibitors, Qfitlia reduced bleeding episodes by 73% compared to on-demand therapy. In patients without inhibitors, the reduction was 71%.
The dosage is personalized through the INNOVANCE Antithrombin diagnostic test, which helps adjust administration based on antithrombin activity levels in the blood.
Potential Side Effects
Like any medication, Qfitlia carries some risks. It may increase the likelihood of thrombotic events (abnormal blood clots) and gallbladder issues, in some cases requiring surgery. Additionally, it may affect liver function, so patients need specific tests during the first six months of treatment or after a dosage change.
Common side effects include viral infections, cold-like symptoms (nasopharyngitis), and bacterial infections.
Regulatory Status
Qfitlia has received FDA Orphan Drug and Fast Track designations, recognizing its importance for a patient population with unmet medical needs. The drug has been approved for commercialization by pharmaceutical company Sanofi.
Glossary
- Antithrombin: A protein that helps prevent excessive clot formation in the blood.
- Bypassing agents: Medications used in hemophilia patients with inhibitors to circumvent the need for the missing clotting factor.
- Thrombin: An enzyme essential for clotting, which converts fibrinogen into fibrin to form a clot.
Fitusiran: An experimental drug for hemophilia treatment, also known as Qfitlia.
First study: A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors (ATLAS-INH).
Second study: A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors.
Orphan Drug: FDA designation for drugs intended to treat rare diseases.
Fast Track: FDA designation to accelerate the development and review of drugs for serious conditions.
