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BLOOD AND BLOOD FORMING ORGANS › ANTIHEMORRHAGICS › VITAMIN K AND OTHER HEMOSTATICS › Blood coagulation factors
etranacogene dezaparvovec
B02BD16
Forms & Strengths
- Suspension for intravenous infusion: 1 x 1E13 gc/mL in single-use vials
Adult Dosing
- Single dose of 2 x 1E13 gc/kg of body weight administered as a single intravenous infusion
Pediatric Dosing
- Safety and efficacy have not been established in pediatric patients under 18 years of age
Indications
- Treatment of severe and moderately severe hemophilia B (congenital Factor IX deficiency) in adult patients currently using Factor IX prophylaxis, or with a history of life-threatening hemorrhage, or severe spontaneous bleeding episodes
Mechanism of Action
- AAV5-based gene therapy containing a codon-optimized Padua variant human factor IX gene under the control of a liver-specific promoter, designed to increase circulating Factor IX activity and reduce bleeding episodes
Contraindications
- Active acute Hepatitis B or C infection
- Advanced hepatic fibrosis (Ishak score > 3) or cirrhosis
- Known hypersensitivity to etranacogene dezaparvovec-drlb or any of its excipients
Adverse Reactions
- Transaminases elevation (ALT/AST increases)
- Headache
- Flu-like symptoms (infusion-related reactions)
- Fatigue
- Nausea
- Malaise
Drug Interactions
- Concomitant use of hepatotoxic drugs may increase the risk of liver enzyme elevations and potential loss of transgene expression
- Coadministration with systemic immunosuppressants (e.g., corticosteroids) may be required to manage immune-mediated transaminase elevations
Curated Content: Needs Vetting Before Put to Clinical Use