Etranacogene dezaparvovec

Etranacogene dezaparvovec is used to treat a bleeding disorder called Hemophilia B in adults who are getting preventive treatment for the condition, have repeated serious bleeding episodes, or have had life-threatening bleeding events in the past or present.

Etranacogene dezaparvovec may also be used for reasons not mentioned in this medication guide.

Your doctor will do medical tests before starting treatment with etranacogene dezaparvovec to ensure it is the right treatment for you.

Etranacogene dezaparvovec is given as an injection into a vein by a healthcare provider.

You will be monitored during and for at least 3 hours after the infusion to make sure you do not have a reaction.

Doses are determined by your weight. Your dose may change if your weight goes up or down.

You will need weekly blood tests to check your liver function for 3 months after treatment.

You may be given other medications to help manage abnormal liver function tests. Continue taking these medicines for as long as your doctor has prescribed.

You may need additional medical tests for a longer time if you have certain conditions or if you experience certain side effects.

Tell your doctor if you use other medicines or have other medical conditions or allergies.

Tell your doctor if you have or have ever had:

• unusual bleeding, any bleeding that will not stop;
• hepatitis B or C;
• if you drink large amounts of alcohol; or
• cirrhosis or other liver disease.

etranacogene dezaparvovec is not intended for use by women.

Get emergency medical help if you have signs of an allergic reaction: hives, difficulty breathing, swelling of your face, lips, tongue, or throat.

Some side effects may happen during or right after the injection. Tell your medical caregiver if you feel dizzy, nauseous, light-headed, itchy, sweaty, or if you have a headache, chest tightness, back pain, trouble breathing, unusual bleeding, or swelling in your face.

Common side effects of etranacogene dezaparvovec may include:

• abnormal blood tests;
• headache;
• flu-like symptoms;
• a reaction after receiving the injection; or
• feeling weak or tired, body aches.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Usual Adult Dose for Hemophilia B:

Usual dose: 2 x 10(13) genome copies per kilogram of body weight (or 2 mL/kg body weight) as a single IV infusion Comments:-Refer to manufacturer product information for detailed dose calculation.-Patients with any of the following criteria are eligible for this treatment:---Currently on Factor IX prophylaxis therapy---Have current or historical life-threatening hemorrhage---Have repeated, serious spontaneous bleeding episodes-The following tests should be performed for screening patients before treatment:---Factor IX inhibitor titer testing: A re-test should be performed within 2 weeks in case of initial positive test results. If both tests are positive, this therapy should not be administered.---Liver health assessments: Enzyme testing (alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase and total bilirubin), hepatic ultrasound and elastography-Consult hepatologist in case of radiological liver abnormalities and/or sustained liver enzyme elevations to assess appropriateness of this treatment.Use: Treatment of adults with Hemophilia B (congenital Factor IX deficiency)

Call your doctor for instructions if you miss an appointment for your Etranacogene dezaparvovec injection.

In a medical setting, an overdose would be treated quickly.

Do not donate blood, organs, tissues, or cells for transplantation after receiving etranacogene dezaparvovec.

Other drugs may impact etranacogene dezaparvovec, including both prescription and over-the-counter medications, vitamins, and herbal products. Inform your doctor about all other medicines you are taking.