Qfitlia fitusiran 20 mg/.2mL Injection, Solution, 1 vial
🆔 Identity & classification
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🏷️ RxNorm drug class
This medicine belongs to the Other systemic hemostatics class.
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🏭 Manufacturer & labeler
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🩺 Clinical
Fitusiran is used to prevent or reduce the frequency of bleeding episodes in people with certain types of hemophilia. Fitusiran is in a class of medications called antithrombin-directed small interfering RNA (siRNA). It works by by reducing the amount of a protein in the body to improve blood clotting.
Read the full MedlinePlus article ↗Patient education
Supplement & herbal interactions
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🧪 Inactive Ingredients / Excipients
Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.
💡 Tap an ingredient (hover on desktop) to see what it is and why it’s used.
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UNII E4GA8884NN
Phosphoric acid is a weak mineral acid used in medicines as a buffer and pH adjuster. It helps stabilize the product and control its acidity level.
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0.982 mg / 0.2 mL
UNII 451W47IQ8X
Sodium chloride is common table salt. It's used in medicines as a buffer to maintain proper pH, as a filler to add bulk, or to adjust the osmotic balance in liquid formulations.
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UNII 55X04QC32I
A strong alkaline chemical used to adjust and maintain the pH balance of liquid medicines. It helps keep the medicine stable and ensures it stays effective during storage.
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0.234 mg / 0.2 mL
UNII 70WT22SF4B
A mineral salt that acts as a buffer to maintain the pH balance of the medication. It helps stabilize the drug's potency and prevents unwanted chemical changes during storage.
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0.018 mg / 0.2 mL
UNII 593YOG76RN
A salt made from sodium and phosphate that helps regulate acidity and maintain proper pH balance in the medicine. It also acts as a buffer to keep the product stable.
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UNII 059QF0KO0R
Water is a liquid solvent that dissolves and mixes ingredients together in liquid medicines, syrups, and injections. It helps distribute the active drug evenly throughout the product.
6 inactive ingredients listed in the exact product block matched to this NDC.
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ingredient classCode="IACT" elements from the exact product block matched by this NDC. Label-section narrative from DailyMed / the openFDA label index is shown separately when available.Inactive ingredient FAQ
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💲 Pricing
A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.
| Price system | Per mL | Per package |
|---|---|---|
| Retail pharmacies payNADAC · weekly | Not in the retail survey — common for institutional, discontinued, or low-volume packs. | |
| Medicaid paysCMS SDUD · 12 mo | No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data. | |
| Medicare drug plans payPart D · quarterly | No Part D plan price is available for this NDC in our data. | |
| Medicare Part B allowsASP · J7174 | $131.202 / J7174 unit | — |
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🧾 Billing & reimbursement
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🔁 Therapeutic equivalents
| Product | Labeler | Pack | NADAC/unit | TE | Status | Price vs. this |
|---|---|---|---|---|---|---|
| Qfitlia 20 mg/.2mLthis 58468-0347-01 | Genzyme | 1 vial | — | — | FDA listed | — |
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⏳ Availability & generic status
We did not find an FDA-approved generic match for this exact strength, form and route. Patent/protection dates below may affect future generic timing.
Why the date isn’t exact: Generic timing can change because patents may be challenged, settled, licensed, added, removed, or worked around with a narrower label — and FDA approval does not always mean a pharmacy can get the generic today.
🛈 What do these terms mean?
- Patent
- Legal protection listed in the Orange Book that may delay generic approval or launch. Issued by the U.S. Patent & Trademark Office.
- Substance patent
- Covers the active drug molecule itself — the hardest to design around. A generic generally can’t launch until it expires.
- Formulation (product) patent
- Covers a specific formulation or dosage form. A generic can sometimes work around it with a different formulation.
- Method-of-use patent
- A patent covering one specific approved use of the drug — not necessarily the whole molecule. A generic can sometimes launch with a “skinny label” that carves out the protected use and keeps the others.
- Skinny label
- A generic label that omits a still-patented use when the FDA allows it — letting a generic reach the market for the unprotected uses.
- Exclusivity
- FDA-granted marketing protection, separate from patents — e.g. 5-yr new chemical entity, 7-yr orphan drug, or a +6-month pediatric extension.
- Paragraph IV
- A generic applicant’s formal challenge to a listed patent. It can potentially lead to earlier generic entry, but often involves litigation or a settlement.
- RLD / RS
- Reference Listed Drug — the brand product the FDA uses as the reference for generic applications. Reference Standard — the product the FDA expects generics to compare against in bioequivalence testing.
- TE / AB rating
- FDA therapeutic-equivalence rating. An AB rating generally means the FDA considers a generic therapeutically equivalent to — and substitutable for — the brand.
- LOE (loss of exclusivity)
- The latest patent or exclusivity currently listed — the loss-of-exclusivity / latest-listed-protection date shown on this page. Paragraph-IV challenges and settlements can move the real date earlier; FDA approval and a manufacturer’s decision to market can move it later.
Built from the FDA Orange Book. The bars above are scaled to each protection’s expiry; the red LOE marker is the last one to lapse.
| Patent | Type | Use code | Expires |
|---|---|---|---|
| US 11091759 ↗ | Method of use | U-4165 | Dec 7, 2036 |
| US 11091759 ↗ | Method of use | U-4165 | Dec 7, 2036 |
| US 9376680 ↗ | Drug substance | U-4165 | Mar 15, 2033 |
| US 9376680 ↗ | Drug substance | U-4165 | Mar 15, 2033 |
| US 9127274 ↗ | Drug substance | U-4165 | Mar 22, 2033 |
| US 9127274 ↗ | Drug substance | U-4165 | Mar 22, 2033 |
| Code | What it grants | Expires |
|---|---|---|
| NCE | New Chemical Entity (5-year) | Mar 28, 2030 |
| ODE-510 | Orphan Drug Exclusivity (7-year) | Mar 28, 2032 |
| ODE-511 | Orphan Drug Exclusivity (7-year) | Mar 28, 2032 |
| NCE | New Chemical Entity (5-year) | Mar 28, 2030 |
| ODE-510 | Orphan Drug Exclusivity (7-year) | Mar 28, 2032 |
| ODE-511 | Orphan Drug Exclusivity (7-year) | Mar 28, 2032 |
Is there a generic version of QFITLIA 20 MG/0.2 ML VIAL?
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🔬 Reported adverse events (FAERS)
Top reported reactions
Age at onset
Reporter sex
Serious outcomes
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📦 Packaging — all sizes for this product
| Package NDC | Description | Marketing start | Status |
|---|---|---|---|
| 58468-0347-01 You're viewing this | 1 VIAL in 1 CARTON (58468-0347-1) / .2 mL in 1 VIAL | 2025-03-28 | Active |
🧭 About this NDC listing & data coverage
What data is (and isn’t) available for this NDC — tap to expand
| NDC identity (package / product / labeler codes) | ✓ Available |
| Labeler | ✓ Available |
| Product & package description | ✓ Available |
| Marketing category & status | ✓ Available |
| Active ingredient / dosage form / route | ✓ Available |
| FDA label (SPL via DailyMed) | ✓ Available |
| Package photos | ✓ Available |
| Inactive ingredients (structured) | ✓ Available |
| NADAC pharmacy acquisition price (CMS) | — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey. |
| Orange Book / therapeutic-equivalence data | ✓ Available |
| HCPCS J-code billing crosswalk | ✓ Available |
| Medicaid utilization (CMS SDUD) | — Not published for this NDC CMS reports utilization only for NDCs with Medicaid claims above its privacy threshold. |
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📄 Full prescribing information FDA SPL
🚨 Boxed Warning ▾
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. Thrombotic Events Serious thrombotic events have occurred in QFITLIA-treated patients with risk factors including persistent antithrombin (AT) activity less than 15%, QFITLIA 80 mg once monthly dosing, an indwelling venous catheter, and the post-operative setting when bleed management guidelines were not followed. Interrupt QFITLIA in patients with a thrombotic event and manage as clinically indicated.
( 5.1 ) Acute and Recurrent Gallbladder Disease Gallbladder disease has occurred in QFITLIA-treated patients, some requiring cholecystectomy or developing complications (e.g., pancreatitis). Monitor for signs and symptoms of gallbladder disease. Consider interruption or discontinuation of QFITLIA if gallbladder disease occurs.
Consider alternative treatment for hemophilia if history of symptomatic gallbladder disease. ( 5.2 ) Thrombotic Events Serious thrombotic events have occurred in QFITLIA-treated patients with risk factors for thromboembolism including persistent antithrombin (AT) activity less than 15%, use of QFITLIA 80 mg once monthly, presence of indwelling venous catheters, and in the post-operative setting when bleed management guidelines were not followed. Monitor AT activity using an FDA-cleared test and target AT activity 15–35% to reduce the risk of thrombosis.
Monitor patients for signs and symptoms of thrombotic events. Interrupt QFITLIA in patients with a thrombotic event and manage as clinically indicated. (5.1) Acute and Recurrent Gallbladder Disease Acute and recurrent gallbladder disease, including cholelithiasis and cholecystitis have occurred in QFITLIA-treated patients, some of whom required cholecystectomy or had complications (e.g., pancreatitis) related to gallbladder disease.
Monitor patients for signs and symptoms of acute and recurrent gallbladder disease. Consider interruption or discontinuation of QFITLIA if gallbladder disease occurs. Consider alternative treatment for hemophilia in patients with a history of symptomatic gallbladder disease.
(5.2)
🎯 Indications and Usage ▾
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 and Administration ▾
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 ).
2.1Recommended 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 <60%. Monitor AT activity using an FDA-cleared test. Information on FDA-cleared tests for AT activity is available at http://www.fda.gov/CompanionDiagnostics.
After QFITLIA is initiated, patients may continue their prior clotting factor concentrates (CFC) or bypassing agent (BPA) prophylaxis for the first 7 days of treatment. Discontinue CFC or BPA prophylaxis no later than 7 days after the initial dose of QFITLIA. The starting dose of QFITLIA is 50 mg once subcutaneously every two months.
Adjust the dose and/or dosing interval, if needed, to maintain AT activity between 15–35% [see Boxed Warning , Dosage and Administration (2.2) and Warnings and Precautions (5.1) ] .
2.2Dosage Modification Measure AT activity using an FDA-cleared test at Weeks 4 (Month 1), 12 (Month 3), 20 (Month 5) and 24 (Month 6) following the starting dose and after any dose modification. If any AT activity is <15%, a dose reduction is required. The lower dose should be initiated 3 months after the prior dose.
AT measurements should be restarted after a dose reduction. If AT activity is >35% after 6 months, or if the patient has not achieved satisfactory bleed control, dose escalation should be considered. AT measurements should be restarted after a dose escalation.
Refer to Table 1 below for modified dosage based on AT activity levels. Table 1: Dose Modification Based on Antithrombin Activity Levels Last Dosage Administered Antithrombin Activity Level Dose Modification 50 mg every 2 months Less than 15% 20 mg every 2 months 15% to 35% Continue current dosage Greater than 35% after 6 months 50 mg every month 20 mg every 2 months Less than 15% 10 mg every 2 months 15% to 35% Continue current dosage Greater than 35% after 6 months 20 mg every month 10 mg every 2 months Less than 15% Discontinue QFITLIA 15% to 35% Continue current dosage Greater than 35% after 6 months 10 mg every month Once the patient's target dose is identified based on AT activity 15–35%, measure AT activity annually.
Additional AT measurements can be considered if bleeding control is not adequate. After cessation of QFITLIA dosing, routine AT monitoring is not needed unless the patient is bleeding and treatment with CFC/BPA is required. Based on data from the clinical studies, a majority of patients have AT activity >60% by 6 months after the last QFITLIA dose, after which standard doses of CFC/BPA may be used.
Missed dose If a dose of QFITLIA is missed, administer as soon as possible; thereafter, resume the patient's usual dosing schedule of either once every month or once every two months, as applicable, from the last dose.
2.3Bleed Management Breakthrough Bleed Management If breakthrough bleeding requiring on-demand treatment with CFC or BPA occurs during the first 7 days after QFITLIA initiation, manage the bleed using the patient's prior dosing regimen of CFC or BPA. If breakthrough bleeding occurs after 7 days from the first QFITLIA dose, bleeds should be managed with a reduced dose and frequency of CFC/BPA to minimize the risk of thrombotic events. Initially, the weight-based dose of a CFC/BPA should be reduced, and the dosing interval doubled compared to the standard dose.
This reduced dosing is shown in Table 2. If adequate hemostatic control is not achieved, higher doses may be used based on clinical judgement. Combination use of antif…
💊 Dosage Forms and Strengths ▾
3 DOSAGE FORMS AND STRENGTHS QFITLIA is a clear, colorless to pale yellow solution in a single-dose: Injection: 50 mg/0.5 mL prefilled pen Injection: 20 mg/0.2 mL vial Injection: 50 mg/0.5 mL (100 mg/mL) in a single-dose prefilled pen 20 mg/0.2 mL (100 mg/mL) in a single-dose vial ( 3 )
⛔ Contraindications ▾
4 CONTRAINDICATIONS None. None ( 4 )
⚠️ Warnings and Cautions ▾
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 ).
5.1Thrombotic 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.
Thrombotic events were reported in 1.4% of patients receiving QFITLIA prophylaxis using the antithrombin-based dose regimen (AT-DR) that targeted AT activity 15-35% (0.8 events per 100 person-years). Participants with established thrombophilia or a history of thrombosis were generally excluded from studies with QFITLIA. The risk of thrombosis is greater in patients with persistent AT activity <15%, with comorbidities that predispose to thrombosis, when bleed management guidelines are not followed in the post-operative setting, when there is an indwelling venous catheter, and with use of the 80 mg once monthly (non-AT-based) dose.
Treatment of breakthrough bleeding episodes with CFC or BPA at a dose greater or more frequent than recommended may also increase thrombotic risk [see Dosage and Administration (2.3) ] . The decision to utilize higher dosing regimens of CFC or BPA in the setting of inadequate hemostasis requires an assessment of the benefits and risks and close clinical monitoring. Monitor AT activity using an FDA-cleared test and target AT activity 15–35% to reduce the risk of thrombosis [see Dosage and Administration (2.1 , 2.2) and Warnings and Precautions (5.1) ] .
Monitor patients for signs and symptoms of thrombotic events. Interrupt QFITLIA prophylaxis in patients with a thrombotic event and manage as clinically indicated. Inform patients treated with QFITLIA to monitor for and report signs and symptoms of thrombotic events.
Consider the benefits and risks of resuming QFITLIA prophylaxis following resolution of the thrombotic event.
5.2Acute and Recurrent Gallbladder Disease Treatment with QFITLIA is associated with an increased occurrence of acute and recurrent gallbladder disease including cholelithiasis and cholecystitis. QFITLIA at a fixed dose (including 80 mg once monthly) is not approved or recommended for use. In the 270 patients in the QFITLIA clinical studies who received the fixed dose (non-AT-based dose) once monthly regimen, 17% experienced gallbladder events and 4% (11 patients) underwent cholecystectomy.
In 286 patients who received the AT-DR, 3.8% experienced gallbladder events and 0.3% (1 patient) underwent cholecystectomy. All but one of the patients who underwent cholecystectomy resumed QFITLIA after surgery. One patient who started on fixed dosing experienced cholangitis and pancreatitis caused by gallstone disease more than a year after cholecystectomy while receiving AT-DR.
Patients diagnosed with acute or recurrent gallbladder disease most commonly presented with epigastric pain, generalized abdominal pain, indigestion, nausea and/or vomiting. If gallbladder disease is suspected, appropriate imaging and clinical follow-up are indicated. Consider alternative treatment for hemophilia in patients with a history of symptomatic gallbladder disease.
Consider interruption or discontinuation of QFITLIA if gallbladder disease occurs.
5.3Hepatotoxicity In the two randomized studies testing QFITLIA 80 mg once monthly, serum alanine transaminase (ALT) and aspartate transaminase (AST) elevations above 3 times the upper limit of normal (ULN) occurred in 32% of patients with hemophilia with inhibitors and 18% of patients with hemophilia without inhibitors compared to no events of AST or ALT elevation greater than 3× ULN in the control groups. There was one case of moderate hepatic injury…
🤒 Adverse Reactions ▾
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.
6.1Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The pooled safety population described in the WARNINGS AND PRECAUTIONS reflect exposure to QFITLIA as fixed doses and AT-DR (N=335). The safety of the QFITLIA AT-DR was assessed in 286 adult and pediatric male patients with hemophilia A or B with or without inhibitors [see Clinical Studies (14) ] .
Among patients who received the AT-DR, 93% were exposed for 6 months or longer and 83% were exposed for 12 months or longer. The median duration of exposure across the studies was 674 days (with a maximum of 896 days). Serious adverse reactions occurred in 4/286 (1.4%) patients who received the AT-DR, two of whom had serious adverse reactions of cholecystitis.
Permanent discontinuation of QFITLIA due to an adverse reaction occurred in 4/286 (1.4%) patients receiving the AT-DR and included liver injury, post-operative deep vein thrombosis, cerebral infarction and pruritus. Dosage interruptions of QFITLIA due to an adverse reaction occurred in 2/286 (0.7%) patients receiving the AT-DR and included increased serum transaminases. The most common adverse reactions (≥10%) reported in patients treated with the AT-DR were viral infection, nasopharyngitis, and bacterial infection.
Table 3: Adverse Reactions Reported in ≥5% of Patients from Pooled Clinical Studies with QFITLIA AT-DR Adverse Reaction Number of Patients (%) N=286 Viral infection Includes similar terms. 29 Nasopharyngitis 26 Bacterial infection 11 Hepatic Injury Hepatic injury includes: alanine aminotransferase increased, aspartate aminotransferase increased, liver injury. 8 Arthralgia 8 Prothrombin fragment 1.2 increased 7 Injection site reaction injection site reactions: includes injection site bruising, injection site erythema, injection site pain, injection site hematoma, injection site atrophy, injection site hemorrhage, injection site discomfort, injection site swelling, injection site discoloration, injection site pruritus, injection site induration, injection site nodule, injection site mass, injection site vesicles, injection site deformation, injection site rash, injection site joint pain and application site erythema.
6 Headache 5 Cough 5 Clinically relevant adverse reactions in less than 5% of patients include: Dyspepsia Abdominal pain
🔄 Drug Interactions ▾
7 DRUG INTERACTIONS
7.1Hypercoagulability 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) ] .
👥 Use in Specific Populations ▾
8 USE IN SPECIFIC POPULATIONS Avoid use in patients with established hepatic impairment (Child-Pugh Class A, B and C) ( 8.6 ). To report SUSPECTED ADVERSE REACTIONS, contact Genzyme Corporation 1-800-745-4447 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
8.1Pregnancy 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. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes.
In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2–4% and 15–20%, respectively.
8.2Lactation Risk Summary There are no data on the presence of fitusiran or its metabolite in human milk, the effects on the breastfed child, or the effects on milk production. It is not known whether QFITLIA is safe for use during breastfeeding. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for QFITLIA and any potential adverse effects on the breastfed infant from QFITLIA or from the underlying maternal condition.
8.3Females and Males of Reproductive Potential Contraception Females Use of QFITLIA in women using hormonal contraceptives may increase the risk of thrombotic events. Estrogen based hormonal contraceptives are an established risk factor for thrombosis in women with inherited AT deficiency. Advise patients using hormonal contraceptives to use an alternative non-hormonal contraception prior to starting treatment with and while receiving QFITLIA.
8.4Pediatric Use The safety and effectiveness of QFITLIA for the treatment of hemophilia A or B with or without factor VIII or IX inhibitors have been established in pediatric patients aged 12 years and older. Use of QFITLIA in pediatric patients with hemophilia A and B is supported by evidence from adequate and well-controlled studies in adult and pediatric patients [see Adverse Reactions (6.1) and Clinical Studies (14) ] . A total of 60 pediatric patients ages 12 to 17 were treated with QFITLIA in the clinical studies.
The safety and effectiveness of QFITLIA have not been established in pediatric patients below 12 years of age.
8.5Geriatric Use There were 3 patients with hemophilia 65 years of age and older in the clinical studies on QFITLIA. Clinical studies of QFITLIA did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects.
8.6Hepatic Impairment Serum transaminase elevations have been observed in the clinical studies [see Warnings and Precautions (5.3) ] . Avoid use of QFITLIA in patients with established hepatic impairment (Child-Pugh Class A, B and C).
🤰 Pregnancy ▾
8.1Pregnancy 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. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes.
In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2–4% and 15–20%, respectively.
🧒 Pediatric Use ▾
8.4Pediatric Use The safety and effectiveness of QFITLIA for the treatment of hemophilia A or B with or without factor VIII or IX inhibitors have been established in pediatric patients aged 12 years and older. Use of QFITLIA in pediatric patients with hemophilia A and B is supported by evidence from adequate and well-controlled studies in adult and pediatric patients [see Adverse Reactions (6.1) and Clinical Studies (14) ] . A total of 60 pediatric patients ages 12 to 17 were treated with QFITLIA in the clinical studies.
The safety and effectiveness of QFITLIA have not been established in pediatric patients below 12 years of age.
🧓 Geriatric Use ▾
8.5Geriatric Use There were 3 patients with hemophilia 65 years of age and older in the clinical studies on QFITLIA. Clinical studies of QFITLIA did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects.
🧬 Clinical Pharmacology ▾
12 CLINICAL PHARMACOLOGY
12.1Mechanism of Action QFITLIA is a double-stranded siRNA that causes degradation of AT messenger RNA (mRNA) through RNA interference, reducing plasma AT levels.
12.2Pharmacodynamics In clinical studies with QFITLIA in hemophilia patients, the primary pharmacodynamic (PD) measure was plasma AT activity. Lower AT activity levels were associated with lower annualized bleeding rates (ABR); however, persistent AT activity <15% is a risk factor for thrombotic events. In Study LTE15174, the mean (SD) AT activity on the AT-DR was 24% (4.64).
Simulations to estimate time for AT activity to reach steady state found that regardless of the dose regimen, more than 99% of participants reached steady state AT levels (defined as <10% variability among consecutive measures) within 23 weeks following initiation of QFITLIA dosing or following dose escalation or de-escalation. The duration of persistent AT activity <60% (non-negligible impact of QFITLIA on coagulation) following QFITLIA treatment discontinuation was estimated to be approximately 6 months, after which standard doses of CFC/BPA may be administered.
In patients with AT activity <15%, the time required prior to initiation of a lower dose was estimated to be 12 weeks to maximize the AT activity recovery to the target range of 15% to 35%. Extent of AT reduction with QFITLIA did not differ between patients with hemophilia A or B, with or without inhibitors.
12.3Pharmacokinetics The pharmacokinetic (PK) properties of fitusiran were evaluated following administration of QFITLIA in patients with hemophilia A or B, with or without inhibitors as summarized in Table 4. QFITLIA PD is driven by liver PK rather than plasma PK. The QFITLIA dosing strategy is based on maintaining plasma AT activity levels between 15–35% with 10, 20 or 50 mg dosing every month or every two months.
Table 4: Pharmacokinetic Parameters of QFITLIA PK Parameters Unless otherwise indicated, the PK parameters in this table are from Study LTE15174. All PK parameters are estimated using non-compartmental analysis and are presented as Mean (SD) [CV%], except for t max which is presented as median (range) QFITLIA 20 mg QFITLIA 50 mg AUC = area under the plasma concentration-time curve extrapolated to infinity; CL/F = apparent clearance; C max = maximum plasma concentration; CV = coefficient of variation; PK = pharmacokinetic(s); SD = standard deviation; t 1/2 = terminal elimination half-life; t max = time to maximum concentration; Vss/F = apparent volume of distribution General Information Steady State Exposure: C max (ng/mL) 34.4 (10.1) [29%] 84.1 (58.7) [70%] AUC (ng ∙ h/mL) 491 (83.8) [17%] 1290 (377) [29%] Dose Proportionality QFITLIA exhibited a dose proportional increase in plasma exposure after SC administration.
Accumulation Study TDR14767 (ALN-AT3SC-001) No accumulation of QFITLIA was observed following multiple once monthly dosing. Absorption t max (h) 2.88 (0.5 – 4.33) 3.78 (0.42 – 11.9) Distribution QFITLIA distributes primarily to the liver after subcutaneous injection. Vss/F 431 (135) [31%] 570 (713) [125%] Protein Binding In vitro data 96.6% at clinically relevant exposure (0.5 microgram/ml) Elimination t 1/2 (h) 5.57 (2.36) [42%] 7.98 (4.74) [59%] CL/F 41.9 (8.39) [20%] 50.8 (71.1) [140%] Metabolism Primary Pathway QFITLIA is metabolized by endo- and exo-nucleases to oligonucleotides of varying lengths, which are further metabolized to oligonucleotides of even shorter lengths.
QFITLIA is not a substrate for CYP450 or transporters. Main Metabolite AS(N-1)3' Excretion Primary Pathway Not evaluated 14.6% (mean) of the administered 50 mg dose of QFITLIA is recovered unchanged in 0–24 hour urine Specific Populations QFITLIA has only been studied in male patients. The pharmacokinetics of fitusiran is not influenced by race.
QFITLIA has not been studied in children <12 years old. No clinical study to evaluate the effect of hepatic impairment or renal impairment on the pharmacokinetics of…
🧬 Mechanism of Action ▾
12.1Mechanism of Action QFITLIA is a double-stranded siRNA that causes degradation of AT messenger RNA (mRNA) through RNA interference, reducing plasma AT levels.
📦 How Supplied / Storage and Handling ▾
16 HOW SUPPLIED/STORAGE AND HANDLING
16.1How Supplied QFITLIA (fitusiran) is a clear, colorless to pale yellow solution supplied in a single-dose prefilled pen or a single-dose vial. Each prefilled pen is designed to deliver 50 mg of QFITLIA in 0.5 mL (NDC 58468-0348-1). Each vial is designed to deliver 20 mg of QFITLIA in 0.2 mL (NDC 58468-0347-1). QFITLIA is available in cartons containing 1 prefilled pen or 1 vial.
16.2Storage 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. Discard no later than 3 months after removal from the refrigerator or at the expiration date, whichever comes first.
After storage at room temperature, do not return the product to the refrigerator. 20 mg Vial Store QFITLIA either in the refrigerator at 2°C to 8°C (36°F to 46°F) or at room temperature between 15°C to 30°C (59°F to 86°F), in the original carton to protect from light. After storage at room temperature, do not return the product to the refrigerator.
Do not shake QFITLIA at any time. Do not heat QFITLIA. Do not freeze.
Do not put into direct sunlight.
📦 Storage and Handling ▾
16.2Storage 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. Discard no later than 3 months after removal from the refrigerator or at the expiration date, whichever comes first.
After storage at room temperature, do not return the product to the refrigerator. 20 mg Vial Store QFITLIA either in the refrigerator at 2°C to 8°C (36°F to 46°F) or at room temperature between 15°C to 30°C (59°F to 86°F), in the original carton to protect from light. After storage at room temperature, do not return the product to the refrigerator.
Do not shake QFITLIA at any time. Do not heat QFITLIA. Do not freeze.
Do not put into direct sunlight.
📋 Description ▾
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.
The pH of a 1% aqueous solution of fitusiran drug substance in 50 mM KCl is 5.4 (4.4–7.3). It has the following structural formula: QFITLIA is supplied as a sterile, preservative-free, clear, colorless to pale yellow solution for subcutaneous administration. Each 50 mg single-dose prefilled pen delivers 50 mg fitusiran (equivalent to 53.0 mg fitusiran sodium) in 0.5 mL, and also contains dibasic sodium phosphate (0.585 mg), monobasic sodium phosphate (0.044 mg), sodium chloride (2.455 mg), and Water for Injection, USP.
Phosphoric acid (concentrated) and sodium hydroxide may be added to adjust to pH 7.0. Each 20 mg single-dose vial delivers 20 mg fitusiran (equivalent to 21.2 mg fitusiran sodium) in 0.2 mL, and also contains dibasic sodium phosphate (0.234 mg), monobasic sodium phosphate (0.018 mg), sodium chloride (0.982 mg), and Water for Injection, USP. Phosphoric acid (concentrated) and sodium hydroxide may be added to adjust to pH 7.0.
Chemical Structure
💬 Information for Patients ▾
17 PATIENT COUNSELING INFORMATION Advise the patient to read the FDA-approved patient labeling (Medication Guide and Instructions for Use). Use of BPAs or CFCs Advise the patient and/or caregiver to discontinue prophylactic use of BPA or CFC no later than 7 days after starting QFITLIA to reduce the risk of thrombotic events. Discuss the appropriate dosing and frequency of BPA or CFC for breakthrough bleed management with the patient and/or caregiver prior to starting QFITLIA prophylaxis [see Dosage and Administration (2.3) ] .
Thrombotic Events Advise the patient and/or caregiver of the risk of thrombotic events while receiving QFITLIA. Inform the patient and/or caregiver of the need for periodic measurements of AT activity that may result in changes to the QFITLIA dose and/or frequency of administration to reduce the risk for thrombosis. Educate patients on the signs and symptoms of thrombotic events and to seek immediate medical attention if new symptoms of thrombotic events occur [see Warnings and Precautions (5.1) ] .
Acute and Recurrent Gallbladder Disease Inform the patient and/or caregiver of the risk of acute and recurrent gallbladder disease while receiving QFITLIA. Educate patients on the signs and symptoms of gallbladder disease and to seek medical attention if new symptoms of gallbladder disease occur [see Warnings and Precautions (5.2) ] . Hepatotoxicity Inform the patient and/or caregiver of the risk of hepatotoxicity and that blood tests to monitor for this risk will be obtained before starting QFITLIA and periodically during treatment.
Inform patients to seek medical attention if symptoms of hepatotoxicity occur [see Warnings and Precautions (5.3) ] . Administration Instructions Provide training to the patient and/or caregiver on proper subcutaneous injection technique, including aseptic technique, and the preparation and administration of QFITLIA prior to use [see Dosage and Administration (2.4) ] .
💬 Medication Guide ▾
This Medication Guide has been approved by the U.S. Food and Drug Administration. Issued: September 2025 MEDICATION GUIDE QFITLIA ® (kew fit lee ah) (fitusiran) injection, for subcutaneous use What is the most important information I should know about QFITLIA?
QFITLIA helps your blood form clots. Do not stop using QFITLIA without talking to your healthcare provider. If you miss doses or stop using QFITLIA, you may no longer be protected against bleeding.
Use of a clotting factor concentrate (CFC) or bypassing agent (BPA) to help protect against bleeding must be stopped within 7 days after your first dose of QFITLIA. Your healthcare provider may prescribe on demand CFC (factor VIII or factor IX products) or BPA if you bleed during treatment with QFITLIA. Carefully follow your healthcare provider's instructions regarding when to use on-demand treatment with CFC or BPA, including the prescribed dose and timing of the CFC or BPA.
QFITLIA can cause serious side effects, including: Abnormal blood clotting (thrombotic events). Serious blood clots have happened in people treated with QFITLIA. QFITLIA can cause blood clots to form in blood vessels in your arms, legs, lungs, heart, brain, eyes, or head.
Your risk of blood clots is greater if your antithrombin (AT) blood level is persistently less than 15% or if you have certain other conditions. Get medical help right away if you get any of these signs or symptoms of blood clots during or after treatment with QFITLIA: swelling, pain or redness in arms or legs coughing up blood shortness of breath severe chest pain or tightness of the chest fast heart rate feeling faint or passing out severe or persistent headache difficulty speaking or understanding language feel confused numbness or weakness in your face, arms or legs sudden loss or changes in your vision, eye pain or swelling Gallbladder disease.
QFITLIA can cause gallstones (cholelithiasis) and inflammation of your gallbladder (cholecystitis), which might require surgery to remove your gallbladder. Tell your healthcare provider right away if you develop stomach (abdomen) pain, indigestion, nausea or vomiting. Your healthcare provider may temporarily or permanently stop your treatment with QFITLIA if you develop any of these symptoms.
Liver problems. QFITLIA can cause an increase in your blood liver enzymes. Your healthcare provider will do blood tests to check your liver function before and during treatment with QFITLIA.
See " What are the possible side effects of QFITLIA? " for more information about side effects. What is QFITLIA? QFITLIA is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children 12 years and older with hemophilia A or B with or without factor VIII or IX inhibitors.
It is not known if QFITLIA is safe and effective in children younger than 12 years of age. Before receiving QFITLIA, tell your healthcare provider about all of your medical conditions, including if you: have liver problems have a history of gallbladder disease are pregnant or plan to become pregnant. It is not known if QFITLIA may harm your unborn baby.
Females who are able to become pregnant: Hormonal birth control (contraception) may increase your risk of blood clots if used during treatment with QFITLIA. If you use hormonal birth control (contraception), talk to your healthcare provider about effective forms of non-hormonal birth control (contraception) options you can use before starting and during treatment with QFITLIA. are breastfeeding or plan to breastfeed. It is not known if QFITLIA passes into your breast milk.
Tell your healthcare provider about all the medicines you take , including prescription and over-the-counter medicines, vitamins, and herbal supplements. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine. How should I use QFITLIA?
See the detailed "Instructions for Use" that comes with your QFITLIA for information on…