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New Drug Introduction: Exondys 51 / eteplirsen Pharmacology Manufacturer Approval Date Indications Contraindications Black Box Warnings Warnings Precautions Pregnancy/Lactation Pharmacokinetics Drug Interactions Adverse Effects (Treatment%) [Placebo%] Monitoring Efficacy Monitoring Toxicity Dosing – Initial/Max Renal Adjustment Hepatic Adjustment Antisense Oligonucleotide Sarepta Therapeutics, Inc. 09/19/2016 Treatment of Duchenne muscular dystrophy (DMD) in patients with a mutation of the DMD gene that is amenable to exon 51 skipping None None None None No human or animal data A – Cmax occurs near the end of infusion (1.1-1.2 hours) and follows linear kinetics D – Volume of distribution= 600 ml/kg and is between 6-17% protein bound M – Not metabolized by hepatic microsomes E – t1/2= 3-4 hours. Clearance was 339 ml/hr/kg following 12 weeks of therapy Does not significantly inhibit CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, or CYP3A4/5 Does not induce CYP2B6, CYP3A4, or CYP1A2 Not a substrate or inhibitor for OAT1, OAT3, OCT1, OCT2, OATP1B1, OATP1B3, P-gp, BCRP, MRP2, and BSEP Balance disorder (38%) (0%) Vomiting (38%) (0%) Contact dermatitis (25%) (0%) 6-minute walk test, dystrophin levels in muscle tissue Monitor for adverse reactions 30 mg/kg once weekly as a 35-60 minute IV infusion No studies have been done in patients with renal impairment No studies have been done in patients with hepatic impairment Cost: Source: MedWatch 10/24/2016 Exondys 51 – Eteplirsen Exondys 51 Prednisone Dose(s) Yearly cost 100 mg/2 ml solution in a single-dose vial 500 mg/10 ml solution in a single-dose vial 50 mg ~$300,000/yr ~$300,000/yr ~$900/yr Summary Exondys 51, eteplirsen, is an antisense oligonucleotide and is the first drug approved to treat patients with Duchenne muscular dystrophy under accelerated approval. Exondys 51 is dosed as a 30 mg/kg once weekly IV infusion over 35-60 minutes. The most common adverse reactions seen with eteplirsen include balance disorders, vomiting, and contact dermatitis. Eteplirsen has a low potential for drug-drug interactions. References: 1. www.exondys51.com 2. Exondys 51 (eteplirsen) package insert. Sarepta Therapeutics, Inc. September. 2016. 3. Eteplirsen. UpToDate. Accessed 10/24/2016. 4. Mendell JR, et al. Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol. 2013;74(5):637-47. Date Prepared: 11/3/2016 Editor: Peter G. Koval, Pharm.D., BCPS Author: Meredith Tilley, Pharm.D. Candidate, UNC Eshelman School of Pharmacy