|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 patiemts 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 50 mg/kg once-weekly dosing regimen of EXONDYS 51 is not recommended.1
- In the 88 patients who received ≥30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in ≥10% of patients and occurred more frequently than with the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.1
- Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension 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.1
DMD PATIENT WITH
DELETIONS OF EXONS 48-50
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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. 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.
IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, 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.
EXONDYS 51 [package insert]. Cambridge, MA: Sarepta Therapeutics Inc; October 2018.