Eteplirsen
Generic Name: eteplirsen
Brand Names:
Exondys 51
11 DESCRIPTION EXONDYS 51 (eteplirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. EXONDYS 51 is clear and colorless, and may have some opalescence, and may contain white to off-white amorphous particles. EXONDYS 51 is supplied in single dose vials containing 100 mg or 500 mg eteplirsen (50 mg/mL).
Overview
11 DESCRIPTION EXONDYS 51 (eteplirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. EXONDYS 51 is clear and colorless, and may have some opalescence, and may contain white to off-white amorphous particles. EXONDYS 51 is supplied in single dose vials containing 100 mg or 500 mg eteplirsen (50 mg/mL).
Uses
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.
Dosage
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.
Side Effects
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).
Warnings
5 WARNINGS AND PRECAUTIONS Hypersensitivity Reactions: Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria, have occurred in patients treated with EXONDYS 51. If hypersensitivity reactions occur, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. ( 2.3 , 5.1 ) 5.1 Hypersensitivity Reactions 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 [see Dosage and Administration ( 2.3 )] . 4 CONTRAINDICATIONS None. None ( 4 )
Pregnancy
8.1 Pregnancy Risk Summary There are no human or animal data available to assess the use of EXONDYS 51 during pregnancy. In the U.S. general population, major birth defects occur in 2 to 4% and miscarriage occurs in 15 to 20% of clinically recognized pregnancies.
Storage
16.2 Storage and Handling Store EXONDYS 51 at 2°C to 8°C (36°F to 46°F). Do not freeze. Protect from light and store EXONDYS 51 in the original carton until ready for use.
Frequently Asked Questions
What is Eteplirsen used for?▼
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.
What are the side effects of Eteplirsen?▼
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).
Can I take Eteplirsen during pregnancy?▼
8.1 Pregnancy Risk Summary There are no human or animal data available to assess the use of EXONDYS 51 during pregnancy. In the U.S. general population, major birth defects occur in 2 to 4% and miscarriage occurs in 15 to 20% of clinically recognized pregnancies.
What are the important warnings for Eteplirsen?▼
5 WARNINGS AND PRECAUTIONS Hypersensitivity Reactions: Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria, have occurred in patients treated with EXONDYS 51. If hypersensitivity reactions occur, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. ( 2.3 , 5.1 ) 5.1 Hypersensitivity Reactions 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 [see Dosage and Administration ( 2.3 )] . 4 CONTRAINDICATIONS None. None ( 4 )
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Medical Disclaimer
This drug information is for educational purposes only and should not replace professional medical advice. Drug information is sourced from the FDA National Drug Code Directory and Structured Product Labeling. Always consult with a healthcare provider before starting, stopping, or changing any medication.