Pronunciation: BECK-vess
Generic name: fidanacogene elaparvovec-dzkt
Dosage form: Injectable for intravenous infusion
Drug class: Miscellaneous coagulation modifiers

Medically reviewed by Carmen Pope, BPharm. Last updated on May 1, 2024.

What is Beqvez?

Beqvez (fidanacogene elaparvovec-dzkt) is a one-time, injectable, viral vector-based gene therapy that may be used to treat moderate to severe hemophilia B (congenital factor IX deficiency) in adult males who:

• Currently use factor IX prophylaxis therapy, or
• Have current or a history of life-threatening hemorrhage, or
• Have repeated, serious spontaneous bleeding episodes, and,
• Do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.

Beqvez (fidanacogene elaparvovec-dzkt) contains a bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity variant of human coagulation Factor IX (FIX) gene. The viral vector component of Beqvez can insert itself into liver cells, which is where factor IX is made. It delivers a functional copy of the factor IX gene encoding the high-activity FIX variant gene. A single, 60-minute, infusion of Beqvez enables patients with hemophilia B to produce the factor IX themselves, rather than needing the regular intravenous infusions.

Beqvez was FDA approved on April 26, 2024.

Beqvez side effects

Beqvez can cause serious side effects, such as liver toxicity, hypersensitivity reactions, infusion reactions, and an increased risk of liver cancer. See the warnings below for more information.

The most common side effect of Beqvez affecting 5% or more patients who take it is an increased risk of transaminases, a type of liver enzyme.

This is not a complete list of side effects. See the Beqvez Package Insert for more information about side effects. To report suspected side effects contact Pfizer Inc. at 1-800-438-1985 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Related/similar drugs

Warnings

Beqvez may cause liver toxicity and an increase in liver transaminases. These elevations, if seen within the first 4 months of treatment, are likely to be due to an immune system-mediated injury to the liver cells and may reduce the effectiveness of Beqvez. Your healthcare provider will monitor your liver transaminases and factor IX activity levels once or twice weekly for at least 4 months after Beqvez administration and treat if necessary.

Infusion reactions, including hypersensitivity reactions and anaphylaxis, may occur during the infusion or within 3 hours after the infusion. Your healthcare provider will monitor you during this time but report any symptoms such as fever, heart palpitations, nausea, vomiting, chills, headache, or signs of low blood pressure (such as dizziness on standing) to your healthcare provider immediately. Your healthcare provider may administer treatments or adjust the infusion rate if a reaction occurs.

Beqvez may increase the risk of liver cancer, especially in people with other risk factors, such as hepatitis B or C, non-alcoholic fatty liver disease, long-term alcohol use, non-alcoholic steatohepatitis, or older age. Your healthcare provider will perform regular liver ultrasounds and alpha-fetoprotein testing every year for 5 years following administration. Report any incidence of liver cancer that occurs after treatment to Pfizer Inc. at 1-800-438-1985. No malignancies were observed to date in clinical studies, but you should be enrolled in a 15-year registry to evaluate the long-term efficacy and safety of hemophilia treatments.

There may be some inter-laboratory variability with regard to reagents used for determining factor IX activity which may affect results. The same laboratory should be used for monitoring over time.

Not all patients will respond to Beqvez and it is currently not possible to predict who will respond and how long the treatment response will continue. Talk to your healthcare provider about this. Some patients may need to re-instate prophylactic use of factor IX concentrates/hemostatic agents.

Not intended for use in females. The safety and effectiveness of Beqvez in children has not been established.

Before taking

Your healthcare provider will perform pre-infusion blood tests to look for factor IX inhibitors and detect pre-existing antibodies to AAVRh74var. If these tests are positive you will not be a candidate for Beqvez.

Before receiving Beqvez, tell your healthcare provider about all your medical conditions, including if you:

• Have liver disease or liver cancer
• Have low albumin levels
• Have HIV or hepatitis
• Have allergies
• Drink alcohol
• Take other medications that may also affect your liver, such as acetaminophen, NSAIDs, statins, or herbal supplements.

Pregnancy

Beqvez is not intended for use in women and there is no data regarding its use in pregnant women.

Male patients should use a condom or be abstinent if they have a female partner, and refrain from donating sperm for up to 6 months after receiving Beqvez.

Breastfeeding

There is no data regarding the use of Beqvez during breastfeeding. Beqvez is not intended for use in women

How is Beqvez administered?

Beqvez is administered by a healthcare provider as a one-time infusion.

• The recommended dosage of Beqvez is 5 x 10¹¹ vector genomes per kg of body weight.

Your healthcare provider will determine the right dose for you and will wear personal protective equipment (such as gloves, safety goggles, and a long-sleeved laboratory coat) while preparing and during the infusion.

• Your healthcare provider will monitor you for infusion reactions during and for at least 3 hours after infusion (see warnings above).
• It is important to maintain or improve hepatic health after receiving Beqvez.
• Potential hepatotoxic medicinal substances, herbal supplements, and alcohol may reduce the efficacy of Beqvez, and increase the risk of serious hepatic reactions.
• If bleeding occurs following administration of Beqvez, then blood tests will be performed for factor IX activity and factor IX inhibitors.

Following Beqvez administration

Because temporary viral vector shedding occurs primarily through urine and feces, and to some extent saliva, mucus, and semen following Beqvez administration, you, your caregivers, or family members should avoid exposure to this human waste and practice good hand hygiene. Talk to your healthcare providers about the proper handling of any materials that have come into contact with patient bodily waste or fluids; recommended procedures include storage of waste material in sealed bags before disposal into regular trash.

These precautions should be followed for 6 months after a Beqvez infusion.

Vaccinations

Adjustments to your vaccination schedule may be necessary if you have also received corticosteroids. Talk to your healthcare provider about this.

What should I avoid after Beqvez?

For the first year after receiving Beqvez, you should limit or avoid alcohol, because alcohol may increase liver enzyme levels and potentially reduce factor IX activity over time.

Avoid the use of herbal supplements that may affect the liver. Talk to your healthcare provider or pharmacist before you take any other medications, because some may have liver side effects.

Viral vector DNA has been detected in all blood and tissues, including semen, following Beqvez administration. Do not donate blood, organs, tissues, or cells for transplantation.

What other drugs will affect Beqvez?

Other drugs may interact with Beqvez, including prescription and over-the-counter medicines, vitamins, and herbal products. No formal interaction studies have been conducted with Beqvez but there is the potential for interactions with:

• Other medications or herbal supplements that affect the liver, such as acetaminophen, NSAIDs, or statins
• Factor IX concentrates/hemostatic agents
• Vaccinations
• Alcohol.

Tell your pharmacist or healthcare provider about all the medicines you take, including any prescription and over-the-counter medicines, vitamins, or herbal supplements. See the prescribing information for a full list of interactions.

Storage

Beqvez is shipped and delivered frozen at between −100 °C to −60 °C (−148 °F to −76 °F) and stored by your healthcare provider at between −90 °C to −60 °C (−130 °F to −76 °F).

Frozen vials take about 1 hour to thaw. The total time at room temperature between removing vials from frozen storage until the beginning of dose preparation should be no more than 3 hours. Once thawed, the medicinal product should not be re-frozen and may be stored refrigerated at 2 °C to 8 °C (36 °F to 46 °F) in the inner carton for up to 24 hours.

Following dilution in 0.9% sodium chloride with 0.25% HSA, stability has been demonstrated for 24 hours at 2 °C to 30 °C (36 °F to 86 °F).

Ingredients

Bio-engineered adeno-associated virus (AAV) capsid (protein shell) with high activity FIX variant gene (FIX-R338L, hFIX Padua).

Beqvez consists of a customized kit containing the number of vials needed for each patient.

Manufacturer

Pfizer Inc.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer