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HEMGENIX®-logo-bg HEMGENIX® (etranacogene dezaparvovec)

Safety Profile For Hemgenix

In clinical studies, HEMGENIX was safe and effective, with no treatment-related serious adverse events and no development of factor IX inhibitors reported.

Adverse Events With HEMGENIX

Adverse Reactions ≥5%
Subjects (%) (N=57)*

Alanine aminotransferase increased

24 (42%)

Headache

10 (18%)

Blood creatine kinase increased

24 (42%)

Flu-like symptoms

8 (14%)

Infusion-related reactions† (see below)

19† (33%)

Hypersensitivity

2‡ (4%)

Fatigue

7 (12%)

Aspartate aminotransferase increased

24 (42%)

Nausea

4 (7%)

Malaise

7 (12%)

*N=57 patients (N=3 patients from a Phase 2b and N=54 patients from a Phase 3 clinical study).

†Infusion-related reaction: In 7 subjects, symptoms occurred during infusion; in 12 subjects, after infusion. Symptoms occurring in ≥5% of subjects were: Dizziness, flu-like symptoms and headache. Symptoms occurring in <5% of subjects were: Abdominal pain, abdominal discomfort, chest discomfort, chills, eye pruritus, fever (pyrexia), flushing, hives (urticaria), infusion site reaction, and tachycardia. Eleven subjects recovered on the day or day one after infusion. Eight subjects recovered within 8 days after infusion.

‡One of 2 hypersensitivity reactions: 12 minutes after initiation of administration of HEMGENIX, the patient experienced high blood pressure, red eyes, feeling warm, dizziness, coughing, dyspnea, elevated heart rate, shivering, and leg cramps. Infusion was stopped and not restarted. Only 10% of the dose of HEMGENIX was administered. The patient recovered on the same day after treatment with intravenous diphenhydramine and intramuscular epinephrine.

2 of 2 hypersensitivity reactions: 10 minutes after initiation of administration of HEMGENIX, the patient experienced itching, tightness of throat, and swelling of the right side of the neck. The dose of HEMGENIX was not interrupted and administered in full. All symptoms resolved on the same day without treatment.

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Safety Considerations

Infusion-related
reactions

Transaminitis

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Infusion-related reactions (IRRs) occurred in 19/57 (33%) of patients. The majority (58%) of patients recovered on the day of or one day after infusion.

In the event of an IRR during administration, the infusion may be slowed or temporarily stopped and restarted at a slower rate once the infusion reaction is resolved. Treatment with a corticosteroid or antihistamine may be considered for management of IRR.

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In the clinical studies, ALT elevation occurred in 24/57 (42%) of patients.

  • The onset of ALT elevations ranged from day 8–day 731 post-dose
  • Nine patients with ALT elevations received a tapered course of corticosteroid. The mean duration of corticosteroid treatment for the elevated ALT was 81.4 days
  • Prophylactic steroids to prevent ALT elevation were not required
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IMPORTANT SAFETY INFORMATION

Patient Selection

Perform baseline testing to select patients, including titer testing for Factor IX (FIX) inhibitor presence. Do not administer HEMGENIX®, etranacogene dezaparvovec-drlb, to patients with FIX inhibitors or a history of FIX inhibitors. Perform liver health assessments, consulting with a hepatologist if needed. Also perform laboratory tests to evaluate hepatitis B and C, and postpone treatment if patient has active infection, as this may reduce the efficacy of HEMGENIX and/or increase the risk of adverse reactions.

Warnings and Precautions

Hypersensitivity and Infusion Reactions

Infusion reactions, including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during administration and for at least 3 hours after end of infusion. If symptoms occur, slow or interrupt administration. When symptoms have resolved, restart administration at a slower infusion rate.

Hepatotoxicity/Hepatocellular Carcinogenicity

Hepatotoxicity with elevated liver transaminase has occurred after HEMGENIX treatment. Monitor ALT levels once per week for 3 months and thereafter monthly up to 1 year after administration. Consider corticosteroid treatment should elevations occur and as clinically indicated.

The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. For patients with preexisting risk factors for hepatocellular carcinogenicity, consider liver ultrasound and alpha-fetoprotein testing following administration, and monitor for hepatocellular carcinomas for five years following administration of HEMGENIX.

Immune-Mediated Neutralization of the AAV5 vector capsid

Preexisting neutralizing anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with HEMGENIX, all patients developed neutralizing anti-AAV5 antibodies to AAV5 vector capsid.

Monitoring Laboratory Tests

Monitor patients regularly for FIX activity (eg, weekly for 3 months), especially when exogenous FIX is administered, as it may take several weeks following HEMGENIX administration before hemostatic control becomes apparent. Hemostatic support may be needed for some patients. Monitor patients through appropriate clinical observations and laboratory tests for the development of inhibitors to FIX.

Adverse Reactions

The most common adverse reactions (incidence ≥5% in clinical trials) are elevated ALT, headache, blood creatine kinase elevations, flu-like symptoms, infusion-related reactions, fatigue, nausea, malaise, and elevated AST.

Indication

HEMGENIX is indicated for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who:

  • Currently use Factor IX prophylaxis therapy, or
  • Have current or historical life-threatening hemorrhage, or
  • Have repeated, serious spontaneous bleeding episodes.

HEMGENIX is for single-use intravenous infusion only.

Please see full prescribing information for HEMGENIX.

To report SUSPECTED ADVERSE REACTIONS, contact the CSL Behring Pharmacovigilance Department at 1-866-915-6958 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.