CLINICAL TRIALS EXPERIENCE
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.1
|Trial type||double-blind, placebo-controlled|
|Trial length||24 weeks|
|Received weekly infusions of EXONDYS 51||n=8|
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 dose). All 12 patients continued in Study 2.
|Trial type||Open label extension of Study 1|
|Trial length||up to 208 weeks|
|Received weekly infusions of EXONDYS 51||n=12|
6 patients received EXONDYS 51 30 mg/kg/week, and 6 patients received EXONDYS 51 50 mg/kg/week.
STUDY 1: ADVERSE REACTIONS1
In DMD patients treated with 30 or 50 mg/kg/week EXONDYS 51
with incidence at least 25% more than placebo:
|ADVERSE REACTIONS||EXONDYS 51 | N=8 (%)||PLACEBO | N=4 (%)|
30-mg/kg and 50-mg/kg groups were pooled. 50 mg/kg/week = 1.7 times the recommended dosage.
Because of the small numbers of patients, these values represent crude frequencies that may not reflect the frequencies observed in practice.1
The 50mg/kg once-weekly dosing requirement of EXONDYS 51 is not recommended.1
OBSERVATIONAL CLINICAL STUDIES
In open-label observational studies, 163 patients received at least one intravenous dose of EXONDYS 51, with doses ranging between 0.5 mg/kg (0.017 times the recommended dosage) and 50 mg/kg (1.7 times the recommended dosage). All patients were male and had genetically confirmed Duchenne muscular dystrophy. Age at study entry was 6 months to 19 years. Most (85%) patients were Caucasian.1
The most common adverse reactions seen in greater than 10% of the study population were headache, cough, rash, and vomiting.1
Postmarketing adverse reactions that occurred during infusion include bronchospasm, cyanosis of the lips, and malaise. The following adverse reactions have also been reported in patients receiving EXONDYS 51: pyrexia, flushing, protein urine present, and dehydration.1
DMD PATIENT WITH
DELETIONS OF EXONS 48-50
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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.