Why is this medication prescribed?

Etranacogene dezaparvovec-drlb injection is prescribed for the treatment of hemophilia B in adult and adolescent patients with factor IX (FIX) deficiency. Hemophilia B is a genetic bleeding disorder caused by a deficiency of clotting factor IX, leading to prolonged bleeding episodes and difficulty in clot formation.

Etranacogene dezaparvovec-drlb is a gene therapy product designed to deliver a functional copy of the Factor IX gene to the patient’s liver cells. Once administered, it enables the patient’s liver to produce Factor IX, thus reducing the frequency of bleeding episodes and the need for exogenous Factor IX replacement therapy.

How should this medicine be used?

Etranacogene dezaparvovec-drlb injection is typically administered by a healthcare professional in a clinical setting due to its nature as a gene therapy product. The injection is delivered directly into the bloodstream via intravenous infusion.

Here’s a general outline of how Etranacogene dezaparvovec-drlb injection is typically used:

• Preparation: The healthcare professional will prepare the injection according to the manufacturer’s instructions. This may involve diluting the product with a compatible solution and ensuring proper handling to maintain sterility.
• Administration: The prepared injection is administered through a vein (intravenous infusion). This process may take some time, during which the patient will be monitored closely for any adverse reactions.
• Post-administration monitoring: After the infusion is complete, the patient will be observed for a period of time to monitor for any immediate reactions or side effects.
• Follow-up: Patients receiving Etranacogene dezaparvovec-drlb injection will require regular follow-up appointments with their healthcare provider to assess the effectiveness of treatment, monitor for any long-term side effects, and adjust the treatment plan as needed.

It’s important for patients to adhere to their healthcare provider’s instructions regarding the administration and monitoring of Etranacogene dezaparvovec-drlb injection. Additionally, patients should report any unusual symptoms or side effects to their healthcare provider promptly.

Other uses for this medicine

Etranacogene dezaparvovec-drlb injection is primarily indicated for the treatment of hemophilia B in adult and adolescent patients with factor IX deficiency. It is a gene therapy product specifically designed to address this condition by delivering a functional copy of the Factor IX gene to liver cells.

What special precautions should I follow?

Regarding special precautions for Etranacogene dezaparvovec-drlb injection, here are some key points:

• Administration by healthcare professionals: Etranacogene dezaparvovec-drlb injection should be administered by healthcare professionals who are experienced in the treatment of hemophilia B and gene therapy products. They should follow proper protocols for preparation, administration, and monitoring.
• Patient selection: Patients should be carefully selected for treatment with Etranacogene dezaparvovec-drlb injection based on factors such as the severity of hemophilia B, previous treatment history, and potential risks and benefits of gene therapy.
• Monitoring: Patients receiving Etranacogene dezaparvovec-drlb injection require regular monitoring to assess the effectiveness of treatment, detect any adverse reactions or complications, and manage them appropriately.
• Risk of immune response: As with any gene therapy product, there is a risk of immune response to Etranacogene dezaparvovec-drlb injection. Patients should be monitored for signs of immune reactions, and appropriate measures should be taken if such reactions occur.
• Potential for thrombotic events: There is a theoretical risk of thrombotic events associated with gene therapy products. Patients should be monitored for signs and symptoms of thrombosis, and appropriate antithrombotic measures should be taken if necessary.
• Patient counseling: Patients and caregivers should be provided with information about the treatment, including its benefits, risks, and potential side effects. They should also be educated about the signs and symptoms of complications and when to seek medical attention.

It’s important for patients to discuss any questions or concerns about Etranacogene dezaparvovec-drlb injection with their healthcare provider before starting treatment. Additionally, they should report any unexpected symptoms or side effects to their healthcare provider promptly.

What special dietary instructions should I follow?

There are no specific dietary restrictions associated with etranacogene dezaparvovec-drlb injection. However, maintaining a balanced diet as recommended by your healthcare provider is essential for overall health and well-being.

What should I do if I forget a dose?

• If you miss a dose of etranacogene dezaparvovec-drlb, contact your healthcare provider as soon as possible for guidance.
• Do not double the dose to catch up for the missed dose unless directed by your healthcare provider.
• Follow your healthcare provider’s instructions for resuming the dosing schedule after a missed dose.

What side effects can this medication cause?

Etranacogene dezaparvovec-drlb injection, like any medication or medical intervention, can cause side effects. Since it’s a gene therapy product used in the treatment of hemophilia B, it may carry specific risks and side effects. It’s important to note that the information provided here is not exhaustive, and patients should consult their healthcare provider for a comprehensive understanding of potential side effects. Some possible side effects of Etranacogene dezaparvovec-drlb injection may include:

• Immune response: There is a risk of immune responses to the therapy, which could manifest as allergic reactions, inflammation, or autoimmune disorders.
• Thrombotic events: There’s a theoretical risk of thrombotic events associated with gene therapy products, including Etranacogene dezaparvovec-drlb injection. This could include blood clots forming in the bloodstream, potentially leading to serious complications such as stroke or heart attack.
• Liver abnormalities: Since Etranacogene dezaparvovec-drlb injection targets the liver for gene therapy, liver abnormalities or enzyme level elevations may occur. Monitoring liver function is essential during treatment.
• Flu-like symptoms: Some patients may experience mild to moderate flu-like symptoms such as fever, chills, fatigue, and muscle aches after receiving the injection.
• Injection site reactions: Pain, redness, swelling, or irritation at the injection site may occur.
• Bleeding: While the therapy aims to improve clotting factor levels in patients with hemophilia B, there is still a risk of bleeding episodes. Patients should be monitored for any signs of increased bleeding tendency.
• Other potential side effects: Other potential side effects may include headache, nausea, diarrhea, and changes in blood pressure.

It’s important for patients to discuss potential side effects and risks with their healthcare provider before starting treatment with Etranacogene dezaparvovec-drlb injection. Additionally, patients should report any unexpected or concerning symptoms to their healthcare provider promptly. Regular monitoring and follow-up appointments will help manage potential side effects and ensure the effectiveness of treatment.

What should I know about storage and disposal of this medication?

Storage and disposal:

• Storage: Etranacogene dezaparvovec-drlb injection should be stored according to the manufacturer’s instructions, which typically involve refrigeration. It is essential to keep the medication at the recommended temperature and protect it from light. Do not freeze the medication.
• Disposal: Unused or expired Etranacogene dezaparvovec-drlb injection should be disposed of properly according to local regulations or guidelines provided by your healthcare provider or pharmacist. Do not dispose of medications in household trash or wastewater unless specifically instructed to do so.

In case of emergency/overdose

• Seek medical attention: If you suspect an overdose or experience severe adverse reactions after receiving Etranacogene dezaparvovec-drlb injection, seek immediate medical assistance. Contact emergency services or go to the nearest hospital emergency room.
• Inform healthcare providers: Provide healthcare providers with as much information as possible about the medication, including the dosage received and the timing of administration. This information will help them assess the situation and provide appropriate treatment.

What other information should I know?

• Follow-up appointments: Patients receiving Etranacogene dezaparvovec-drlb injection will require regular follow-up appointments with their healthcare provider to monitor treatment effectiveness, assess for any side effects or complications, and adjust the treatment plan as needed.
• Patient counseling: Patients and caregivers should receive thorough counseling from healthcare providers regarding the benefits, risks, and potential side effects of Etranacogene dezaparvovec-drlb injection. They should also be educated about the signs and symptoms of adverse reactions and instructed on when to seek medical attention.
• Genetic counseling: Given that Etranacogene dezaparvovec-drlb injection is a gene therapy product, patients and their families may benefit from genetic counseling to understand the implications of gene therapy and potential hereditary factors associated with hemophilia B.

It’s important to adhere to all instructions provided by healthcare providers regarding the storage, administration, and disposal of Etranacogene dezaparvovec-drlb injection. Additionally, promptly report any concerns or adverse reactions to healthcare providers for appropriate management.