Exondys 51 50 mg/mL
eteplirsen · INJECTION · Sarepta Therapeutics, Inc.
Exondys 51 is a injection containing eteplirsen at 50 mg/mL, taken intravenous. Manufactured by Sarepta Therapeutics, Inc..
Key Facts
- Brand Name
- Exondys 51
- Generic Name
- eteplirsen
- NDC Code (Product)
60923-284- Manufacturer
- Sarepta Therapeutics, Inc.
- Strength
- 50 mg/mL
- Dosage Form
- INJECTION
- Route
- INTRAVENOUS
- Marketing Status
- Application #
- NDA206488
- Drug Class
- Antisense Oligonucleotide [EPC]
- Marketing Start
- 09/19/2016
Recall History
No Recall HistorySide Effects Reported to FDA
FDA FAERS database · These are reported events, not confirmed side effects
Full Prescribing Information
Indications & Usage
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 )] . Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. EXONDYS 51 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. 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 )]. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. ( 1 )
Dosage & Administration
2 DOSAGE AND ADMINISTRATION 30 milligrams per kilogram of body weight once weekly ( 2.1 ) Administer as an intravenous infusion over 35 to 60 minutes via an in-line 0.2 micron filter ( 2.1 , 2.3 ) Dilution required prior to administration ( 2.2 ) 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 via an in-line 0.2 micron filter. 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…
Contraindications
4 CONTRAINDICATIONS None. None ( 4 )
Adverse Reactions
6 ADVERSE REACTIONS The most common adverse reactions (incidence ≥35% and higher than placebo) were balance disorder and vomiting ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. EXONDYS 51 was studied in a double-blind, placebo-controlled study for 24 weeks (Study 1), followed by an open label extension (Study 2). In Study 1, 12 patients were randomized to receive weekly intravenous infusions of EXONDYS 51 (n=8) or placebo (n=4) for 24 weeks. All 12 patients continued in Study 2 and received open-label EXONDYS 51 weekly for up to 208 weeks. In Study 1, 4 patients received placebo, 4 patients received EXONDYS 51 30 mg/kg, and 4 patients received EXONDYS 51 50 mg/kg (1.7 times the recommended dosage). In Study 2, 6 patients received EXONDYS 51 30 mg/kg/week and 6 patients received …
Frequently Asked Questions
What is Exondys 51 used for?
Exondys 51 contains eteplirsen. It is a injection taken intravenous. Consult your doctor for specific uses.
Is Exondys 51 a controlled substance?
Exondys 51 is not classified as a controlled substance by the DEA.
What is the generic name for Exondys 51?
The generic name for Exondys 51 is eteplirsen. There are no other listed brand versions of eteplirsen.
What is the NDC code for Exondys 51 50 mg/mL?
The NDC (National Drug Code) for Exondys 51 50 mg/mL is 60923-284, listed by Sarepta Therapeutics, Inc..
Other Exondys 51 Dosages
Not medical advice. Always consult your doctor or pharmacist before making any medication decisions.
Data from openFDA · Public domain (CC0 1.0)