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eplontersen
N07XX21
Forms & Strengths
- Subcutaneous injection: 45 mg/2 mL in a single-dose pre-filled syringe or pre-filled pen
Adult Dosing
- Polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN): 45 mg subcutaneously once monthly
Pediatric Dosing
- Safety and efficacy in pediatric patients have not been established
Indications
- Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Mechanism of Action
- Antisense oligonucleotide (ASO) conjugated to N-acetylgalactosamine (GalNAc) that targets transthyretin (TTR) mRNA
- Binds to TTR mRNA, leading to its degradation by RNase H-1
- Reduces the synthesis of both mutant and wild-type TTR protein in the liver
Contraindications
- History of severe hypersensitivity reaction to eplontersen or any of its components
Adverse Reactions
- Injection site reactions
- Vitamin A decrease
- Nausea
- Vomiting
- Pyrexia
- Rash
Drug Interactions
- No formal clinical drug-drug interaction studies have been conducted
- Potential for reduced serum Vitamin A levels requires concurrent dietary supplementation
Curated Content: Needs Vetting Before Put to Clinical Use