Beqvez: Uses, Side Effects and Dosage

What is BEQVEZ used for?

BEQVEZ is used to treat people with Hemophilia B who have a gene that is not functioning to allow the body to make enough of a protein called Factor IX. This protein is important for blood to clot and stop bleeding. BEQVEZ is given to help make enough working Factor IX protein to help prevent bleeding in patients with Hemophilia B.

How does BEQVEZ work?

BEQVEZ is a type of medicine called a ‘gene therapy.’ The active substance in BEQVEZ, fidanacogene elaparvovec, is based on a virus that does not cause disease in humans. This virus cannot spread in the body but can deliver a copy of the Factor IX gene into your cells. This allows the body to produce Factor IX protein and increase the levels of working Factor IX in the blood to help the blood to clot and prevent or reduce bleeding episodes in patients with Hemophilia B.

What are the ingredients in BEQVEZ?

Medicinal ingredient: fidanacogene elaparvovec

Non-medicinal ingredients: disodium phosphate heptahydrate, monosodium phosphate monohydrate, poloxamer 188, sodium chloride, water for injection.

This medicine contains recombinant adeno-associated viral vectors.

BEQVEZ comes in the following dosage forms:

BEQVEZ is supplied in a plastic vial. When thawed, BEQVEZ is a clear to slightly opalescent, colourless to slightly brown solution.

Do not use BEQVEZ if:

• You are allergic to fidanacogene elaparvovec or to any of the other ingredients of this medicine.

To help avoid side effects and ensure proper use, talk to your healthcare professional before you are given BEQVEZ. Talk about any health conditions or problems you may have, including if you:

• Have or had liver or kidney problems
• Have an infection
• Are planning on having children

For your personal safety, the treatment with BEQVEZ will take place under the supervision of your healthcare professional in a clinical setting.

Before treatment with BEQVEZ

Your healthcare professional will perform several tests before you are given BEQVEZ treatment.

• Antibody blood tests: Your healthcare professional will conduct blood tests to check for certain antibodies (proteins) before treatment with BEQVEZ, including:

  • Blood tests to see if you have antibodies directed against the type of virus used to make BEQVEZ. If you have these antibodies, you will not receive BEQVEZ.
  • Blood tests to check for the presence of antibodies in your blood directed against the human Factor IX protein (Factor IX inhibitors). If you test positive for these antibodies, another test will be performed in approximately 2 weeks. If both the initial test and re-test results are positive, you will not receive BEQVEZ.

• Liver health: If you have poor liver health you may not receive BEQVEZ. Your healthcare professional will check the status of your liver health before you start treatment with BEQVEZ and perform:

  • Blood tests to check your liver enzymes in your blood
  • Liver ultrasound
  • Tests to check for scarring or thickening of your liver (fibrosis assessment).

During or shortly after receiving BEQVEZ

• Infusion-related side effects can occur during or shortly after you are given BEQVEZ infusion (drip). Your healthcare professional will monitor you during BEQVEZ infusion and for at least 3 hours after. You may experience symptoms such as, but not limited to, hypotension (low blood pressure), fever, palpitation (fast or irregular heartbeat), nausea, vomiting, chills or headache. Tell your healthcare professional immediately if you experience these or any other symptoms during or shortly after the treatment infusion.
• Depending on your symptoms, your infusion may be interrupted. If the infusion is interrupted, your healthcare professional may choose to restart it at a slower rate. Your healthcare professional may also consider if you should be given another medicine to help manage your symptoms.

After treatment with BEQVEZ

After treatment with BEQVEZ, your healthcare professional will continue to check your health. It is important that you discuss the schedule for these blood tests with your healthcare professional so that they can be carried out as necessary.

• Liver enzymes: BEQVEZ will trigger a response within your immune system that could lead to an increased level of certain liver enzymes in your blood called transaminases (transaminitis). Your healthcare professional will regularly monitor your liver enzyme levels to ensure that the medicine is working as it should:

  • In the first 3 months you will have blood tests twice per week to monitor your liver enzyme levels.

    • If you experience an increase in liver enzymes, you may have more frequent blood tests to check the levels of your liver enzymes, until they return to normal. You may also need to take another medicine (corticosteroids) to manage these side effects. Corticosteroids may cause side effects when you take

      • depending on your blood test results.
    • Your healthcare professional may also perform additional tests to exclude other causes for the increase in your liver enzymes, if needed, in consultation with a healthcare professional experienced in liver diseases (hepatologist).
  • Your healthcare professional will continue to regularly monitor your liver enzyme levels over time following BEQVEZ administration.

• Factor IX levels: Your healthcare professional will regularly check your Factor IX levels to see if treatment with BEQVEZ was successful.

  • In at least the first 3 months after you are given BEQVEZ, you will have blood tests twice per week to check your Factor IX levels. Your healthcare professional will continue to monitor your Factor IX levels at regular intervals over time following BEQVEZ administration.

Discontinuation of other Hemophilia B treatments:

Talk to your healthcare professional about if or when you should stop your other Hemophilia B treatments and develop a treatment plan of what to do in case of surgery, trauma, bleeds, or any procedures that will increase your risk of bleeding. It is important to continue your monitoring and keep your healthcare professional visits.

Risk of liver cancer (hepatocellular carcinoma) potentially associated with BEQVEZ

BEQVEZ will insert into cells in your body and it could possibly insert into your DNA. This could contribute to a risk of cancer, such as liver cancer. Although there is no evidence of this in the clinical trials so far, this remains possible because of the nature of the medicine. You should therefore discuss this with your healthcare professional.

If you are a patient with risk factors for liver cancer (you have liver cirrhosis or scarring and thickening of the liver, or Hepatitis B, Hepatitis C or fatty liver), your healthcare professional will monitor your liver health yearly for at least 5 years after BEQVEZ administration and perform the following tests:

• Annual liver ultrasound
• Annual blood tests to check for increases in protein (alpha-fetoprotein).

Avoiding blood donations and donations for transplantations

To ensure BEQVEZ DNA is not transferred from you to another person, you will not be able to donate blood, organs, tissues, or cells after you have been treated with BEQVEZ.

Abnormal clotting of blood (thromboembolic events)

After treatment with BEQVEZ, your Factor IX protein level may increase. Although not observed in clinical trials with BEQVEZ, Factor IX protein could increase to levels above the normal range. Unusually high Factor IX levels may cause your blood to clot abnormally, increasing the risk of blood clots. You may be at increased risk for abnormal blood clotting if you have pre-existing problems with your heart and blood vessels (e.g., a history of heart disease, high blood pressure, or if you are diabetic). Your healthcare professional will regularly monitor your blood for potential abnormalities in Factor IX levels. Consult your doctor immediately, if you observe signs of abnormal clotting, such as sudden chest pain, shortness of breath, sudden onset of muscle weakness, loss of sensation and/or balance, decreased alertness, difficulty in speaking, or swelling of one or both legs.

Receiving gene therapy again in the future

After receiving BEQVEZ, your immune system will produce antibodies to the shell of the AAV vector. It is not yet known whether or under which conditions therapy with BEQVEZ may be repeated. It is also not yet known whether or under which conditions subsequent use of another gene therapy may be possible.

Long-term follow-up

After receiving this treatment, you are expected to be enrolled in a registry to follow Hemophilia patients. This is to help understand the long-term safety and how well it continues to work.

Other warnings you should know about:

• Children and adolescents: BEQVEZ is not recommended for children or adolescents under the age of 18.

• Pregnancy, breast-feeding and fertility: BEQVEZ is not intended for use in women and there are no data regarding BEQVEZ in pregnant or breast-feeding women. There is no information on the effect of BEQVEZ on female or male fertility.

• Driving and using machines: Some side effects of BEQVEZ may affect your ability to drive or use machines. You should wait until the side effects go away before you drive or use machines.

• Hygiene precautions after receiving BEQVEZ: The active substance in BEQVEZ may be transmitted to persons other than the patient receiving the treatment through blood, semen and other bodily waste and fluids; this is called ‘shedding’. You, and your caregivers, should take precautions and should practice proper hand hygiene when coming into direct contact with patient bodily waste and fluids. These precautions should be followed for 6 months after BEQVEZ infusion, especially in case of pregnancy or close contact with a person who has a weakened immune system.
• Use of contraception: It is recommended that you and your female partner use appropriate barrier contraception for 1 year after receiving BEQVEZ to prevent DNA to be transferred to a child. For the same reasons, you must not donate semen.

Tell your healthcare professional about all the medicines you take, including any drugs, vitamins, minerals, natural supplements or alternative medicines.

Medicines and herbal supplements which affect the liver and alcohol may impact the response to this medicine and may increase the risk of liver damage.

How BEQVEZ is given:

BEQVEZ will be given to you in a hospital setting under the direction of a healthcare professional experienced and trained in the treatment of Hemophilia B. Your healthcare professional will administer the treatment dose based on your weight. Treatment with BEQVEZ consists of a one-time single infusion (drip) into a vein.

If you have any questions on the use of BEQVEZ ask your healthcare professional.

Usual dose:

Your healthcare professional will determine the correct dose for you based on your body weight. The dose is 5 x 10¹¹ genome copies, the unit BEQVEZ is measured in, per kg of your body weight.

Overdose:

There is no experience of overdose with BEQVEZ.

Missed dose:

BEQVEZ is administered only once.

What are possible side effects from using BEQVEZ?

Like all medicines, BEQVEZ can cause side effects, although not everybody gets them.

Talk to your healthcare professional if you develop any side effects. These can include:

Very common (may affect more than 1 in 10 people)

• Increased levels of transaminases (liver enzymes) seen in blood tests.
• Headache
• Joint pain
• Respiratory infections

Common (may affect more than 1 in 100 and up to 1 in 10 people)

• Anemia (decreased red blood cells)
• Abdominal (stomach) pain
• Diarrhea
• Dyspepsia (upset stomach)
• Gastrointestinal reflux disease (acid reflux, heartburn)
• Abnormal liver function, fatty liver
• Gastroenteritis (stomach flu)
• Sprain
• Muscle strain
• Coagulation Factor X level decreased
• Back pain
• Joint swelling
• Muscle aches
• Pain in extremity
• Dizziness
• Insomnia
• Cough
• Acne
• High blood pressure

These are not all the possible side effects you may have when taking BEQVEZ. If you experience any side effect that is not listed here or becomes bad enough to interfere with your daily activities, tell your healthcare professional.

Reporting Side Effects

Storage:

BEQVEZ will be stored by the healthcare professionals at your healthcare facility. You will not store BEQVEZ yourself.

If you want more information about BEQVEZ:

• Talk to your healthcare professional
• Find the full product monograph that is prepared for healthcare professionals and includes this Patient Medication Information by visiting the Health Canada website: (https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/drug-product-database.html; the manufacturer’s website [www.pfizer.ca], or by calling 1-800-463-6001 (Pfizer Medical Information).

Last Revised December 20, 2023