Download Exondys 51

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