Fitusiran
Generic Name: fitusiran
Brand Names:
Qfitlia
11 DESCRIPTION QFITLIA injection contains fitusiran, an antithrombin-directed double-stranded small interfering ribonucleic acid (siRNA), which is covalently linked to a ligand containing a triantennary N-acetylgalactosamine (GalNAc) moiety. The molecular formula of fitusiran sodium is C 520 H 636 F 21 N 175 Na 43 O 309 P 43 S 6 and the molecular weight is 17,193 Da. Fitusiran drug substance is a white to pale yellow powder and freely soluble in water and phosphate buffered saline.
Overview
11 DESCRIPTION QFITLIA injection contains fitusiran, an antithrombin-directed double-stranded small interfering ribonucleic acid (siRNA), which is covalently linked to a ligand containing a triantennary N-acetylgalactosamine (GalNAc) moiety. The molecular formula of fitusiran sodium is C 520 H 636 F 21 N 175 Na 43 O 309 P 43 S 6 and the molecular weight is 17,193 Da. Fitusiran drug substance is a white to pale yellow powder and freely soluble in water and phosphate buffered saline.
Uses
1 INDICATIONS AND USAGE QFITLIA is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or B with or without factor VIII or IX inhibitors. QFITLIA is an antithrombin-directed small interfering ribonucleic acid indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or B with or without factor VIII or IX inhibitors. ( 1 )
Dosage
2 DOSAGE AND ADMINISTRATION For subcutaneous use only. Starting dose: 50 mg once every 2 months ( 2.1 ). Monitor AT activity using an FDA-cleared test. Maintain AT activity between 15–35% by adjusting the dose and/or frequency of administration ( 2.2 ). See Full Prescribing Information for important preparation and administration instructions ( 2.4 ). 2.1 Recommended Dosage For subcutaneous use only. Use of QFITLIA is recommended under the supervision of a healthcare professional experienced in the treatment of hemophilia or bleeding disorders. Measure AT activity prior to initiation of QFITLIA. Do not initiate QFITLIA dosing if AT activity is 35% after 6 months, or if the patient has not achieved satisfactory bleed control, dose escalation should be considered.
Side Effects
6 ADVERSE REACTIONS The following clinically significant adverse reactions are described elsewhere in the labeling: Thrombotic Events [see Boxed Warning and Warnings and Precautions (5.1) ] Acute and Recurrent Gallbladder Disease [see Boxed Warning and Warnings and Precautions (5.2) ] Hepatotoxicity [see Warnings and Precautions (5.3) ] Common adverse reactions (incidence >10%) are viral infection, nasopharyngitis, and bacterial infection ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact Genzyme Corporation 1-800-745-4447 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Interactions
7 DRUG INTERACTIONS 7.1 Hypercoagulability with Concomitant Use of CFC or BPA QFITLIA prophylaxis leads to increased thrombin generation with additive increase in peak thrombin when used concomitantly with CFC or BPA [see Boxed Warning , Dosage and Administration (2.3) and Warnings and Precautions (5.1) ] .
Warnings
WARNING: THROMBOTIC EVENTS AND ACUTE AND RECURRENT GALLBLADDER DISEASE WARNING: THROMBOTIC EVENTS and ACUTE AND RECURRENT GALLBLADDER DISEASE See full prescribing information for complete boxed warning. 5 WARNINGS AND PRECAUTIONS Hepatotoxicity: Obtain liver tests at baseline and then monthly for at least 6 months after initiating QFITLIA and after dose increases, and periodically thereafter. Liver test elevations may require QFITLIA interruption or discontinuation ( 5.3 ). 5.1 Thrombotic Events Serious thrombotic events have been reported in QFITLIA-treated patients. Thrombotic events were reported in 2.6% of patients receiving the 80 mg once monthly dose (2.3 events per 100 person-years), including a fatal event of cerebral venous sinus thrombosis. The 80 mg once monthly dose is not approved or recommended for use. 4 CONTRAINDICATIONS None. None ( 4 )
Pregnancy
8.1 Pregnancy Risk Summary There are no available data on QFITLIA use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Reproduction studies in pregnant animals have not been conducted with fitusiran. It is not known whether QFITLIA can cause fetal harm when administered to a pregnant woman or can affect reproduction capacity. QFITLIA should be used during pregnancy only if the potential benefit justifies the potential risks, including those to the fetus.
Storage
16.2 Storage and Handling 50 mg Prefilled Pen Store refrigerated at 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. QFITLIA may be stored at room temperature between 15°C to 30°C (59°F to 86°F) for a single period of up to 3 months within the expiration date printed on the label.
Frequently Asked Questions
What is Fitusiran used for?▼
1 INDICATIONS AND USAGE QFITLIA is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or B with or without factor VIII or IX inhibitors. QFITLIA is an antithrombin-directed small interfering ribonucleic acid indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or B with or without factor VIII or IX inhibitors. ( 1 )
What are the side effects of Fitusiran?▼
6 ADVERSE REACTIONS The following clinically significant adverse reactions are described elsewhere in the labeling: Thrombotic Events [see Boxed Warning and Warnings and Precautions (5.1) ] Acute and Recurrent Gallbladder Disease [see Boxed Warning and Warnings and Precautions (5.2) ] Hepatotoxicity [see Warnings and Precautions (5.3) ] Common adverse reactions (incidence >10%) are viral infection, nasopharyngitis, and bacterial infection ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact Genzyme Corporation 1-800-745-4447 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Can I take Fitusiran during pregnancy?▼
8.1 Pregnancy Risk Summary There are no available data on QFITLIA use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Reproduction studies in pregnant animals have not been conducted with fitusiran. It is not known whether QFITLIA can cause fetal harm when administered to a pregnant woman or can affect reproduction capacity. QFITLIA should be used during pregnancy only if the potential benefit justifies the potential risks, including those to the fetus.
What are the important warnings for Fitusiran?▼
WARNING: THROMBOTIC EVENTS AND ACUTE AND RECURRENT GALLBLADDER DISEASE WARNING: THROMBOTIC EVENTS and ACUTE AND RECURRENT GALLBLADDER DISEASE See full prescribing information for complete boxed warning. 5 WARNINGS AND PRECAUTIONS Hepatotoxicity: Obtain liver tests at baseline and then monthly for at least 6 months after initiating QFITLIA and after dose increases, and periodically thereafter. Liver test elevations may require QFITLIA interruption or discontinuation ( 5.3 ). 5.1 Thrombotic Events Serious thrombotic events have been reported in QFITLIA-treated patients. Thrombotic events were reported in 2.6% of patients receiving the 80 mg once monthly dose (2.3 events per 100 person-years), including a fatal event of cerebral venous sinus thrombosis. The 80 mg once monthly dose is not approved or recommended for use. 4 CONTRAINDICATIONS None. None ( 4 )
Related Medications
Chlorhexidine Hydrochloride
chlorhexidine hydrochloride
Dosage form: POWDER. Active ingredients: CHLORHEXIDINE HYDROCHLORIDE (1 kg/kg). Category: BULK INGREDIENT.
Bacti-gone
bacti-gone
purpose Antiseptic
Dexfosfoserine
dexfosfoserine
Dosage form: POWDER. Active ingredients: DEXFOSFOSERINE (1 kg/kg). Category: BULK INGREDIENT.
Medical Disclaimer
This drug information is for educational purposes only and should not replace professional medical advice. Drug information is sourced from the FDA National Drug Code Directory and Structured Product Labeling. Always consult with a healthcare provider before starting, stopping, or changing any medication.