STORAGE AND HANDLING.1
Once you receive EXONDYS 51 at your facility, be sure to store it according to proper procedures.
Store EXONDYS 51 at 2°C to 8°C (36°F to 46°F)
Protect from light and store in the original carton until ready for use
Do not freeze
To infuse EXONDYS 51, you will need:
EXONDYS 51 10 mL or 2 mL single-dose vials
0.2 micron filter
A syringe fitted with a 21-gauge or smaller non-coring needle
PREPARING EXONDYS 51.1
Using aseptic technique, prepare EXONDYS 51 according to these steps:
EXONDYS 51 is administered intravenously via an in-line 0.2 micron filter. Flush the patient’s intravenous access with sodium chloride 0.9% injection, USP, prior to and after infusion.
Application of a topical anesthetic cream to the infusion site prior to administration of EXONDYS 51 may be considered.
Assess the patient’s IV or port for patency prior to removing any vials from the refrigerator. If IV access is adequate, remove the appropriate number of vials from the refrigerator and allow them to warm to room temperature. Do not microwave vials.
Once at 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, and may contain trace amounts of small, white to off-white amorphous particles. Do not use if the solution in the vials is cloudy, discolored or contains extraneous particulate matter other than trace amounts of small, white to off-white amorphous particles. If there is an issue with the solution, please report the issue to Sarepta at 1-888-SAREPTA (1-888-727-3782).
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.
Before adding the calculated volume of EXONDYS 51 to the saline bag, remove an equal volume of normal saline solution from the bag. Next, inject the bag with the syringe containing the calculated patient dose of EXONDYS 51. Gently invert infusion bag to ensure equal distribution of product. Contents can be mixed through 2-3 gentle inversions. Avoid agitation during preparation. Do not shake. Visually inspect the diluted solution. Do not use if the solution is cloudy, discolored or contains extraneous particulate matter other than trace amounts of small, white to off-white amorphous particles. EXONDYS 51 contains no preservatives and should be administered immediately after dilution. Complete infusion within 4 hours of dilution. If immediate use is not possible, the diluted product may be stored for up to 24 hours at 2° C to 8° C (36° F to 46° F).
DMD PATIENT WITH
DELETION OF EXON 50
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EXONDYS 51 (eteplirsen) 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. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia and urticaria, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.
Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.
The most common adverse reactions from observational clinical studies (N=163) seen in greater than 10% of patients were headache, cough, rash, and vomiting.
Please see the full Prescribing Information for EXONDYS 51 (eteplirsen).
EXONDYS 51 [package insert]. Cambridge, MA: Sarepta Therapeutics Inc.