For: Duchenne Muscular Dystrophy
Exondys 51 (eteplirsen) is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. EXONDYS 51- eteplirsen injection
Sarepta Therapeutics, Inc.
1 INDICATIONS AND USAGE
EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51 [see Clinical Studies (14)]. A clinical benefit of EXONDYS 51 has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
2 DOSAGE AND ADMINISTRATION
2.1 Dosing Information
The recommended dose of EXONDYS 51 is 30 milligrams per kilogram administered once weekly as a 35 to 60 minute intravenous infusion.
If a dose of EXONDYS 51 is missed, it may be administered as soon as possible after the scheduled time.
2.2 Preparation Instructions
EXONDYS 51 is supplied in single-dose vials as a preservative-free concentrated solution that requires dilution prior to administration. Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. Use aseptic technique.
Calculate the total dose of EXONDYS 51 to be administered based on the patient's weight and the recommended dose of 30 milligrams per kilogram. Determine the volume of EXONDYS 51 needed and the correct number of vials to supply the full calculated dose.
Allow vials to warm to room temperature. Mix the contents of each vial by gently inverting 2 or 3 times. Do not shake.
Visually inspect each vial of EXONDYS 51. EXONDYS 51 is a clear, colorless solution that may have some opalescence. Do not use if the solution in the vials is discolored or particulate matter is present.
With a syringe fitted with a 21-gauge or smaller non-coring needle, withdraw the calculated volume of EXONDYS 51 from the appropriate number of vials.
Dilute the withdrawn EXONDYS 51 in 0.9% Sodium Chloride Injection, USP, to make a total volume of 100-150 mL. Visually inspect the diluted solution for particulates.
EXONDYS 51 contains no preservatives and should be administered immediately after dilution. Complete infusion of diluted EXONDYS 51 solution within 4 hours of dilution. If immediate use is not possible, the diluted solution may be stored for up to 24 hours at 2�C to 8�C (36�F to 46�F). Do not freeze. Discard unused EXONDYS 51.
2.3 Administration Instructions
Application of a topical anesthetic cream to the infusion site prior to administration of EXONDYS 51 may be considered.
EXONDYS 51 is administered via intravenous infusion. Flush the intravenous access line with 0.9% Sodium Chloride Injection, USP, prior to and after infusion.
Infuse the diluted EXONDYS 51 solution over 35 to 60 minutes. Do not mix other medications with EXONDYS 51 or infuse other medications concomitantly via the same intravenous access line.
3 DOSAGE FORMS AND STRENGTHS
EXONDYS 51 is a clear and colorless solution that may have some opalescence, and is available as follows:
Injection: 100 mg/2 mL (50 mg/mL) solution in a single-dose vial
Injection: 500 mg/10 mL (50 mg/mL) solution in a single-dose vial
