AMONDYS 45 (casimersen)

The pharmacokinetics of casimersen was evaluated in DMD patients following administration of intravenous (IV) doses ranging from 4 mg/kg/week to 30 mg/kg/week (i.e., recommended dosage). Following a single IV dose of casimersen, C max was reached at the end of infusion. Casimersen exposure increased in a proportional manner with dose increment. No accumulation of casimersen was observed in plasma following once weekly dosing. Inter-subject variability (as %CV) for casimersen C max and AUC ranged...

The effect of AMONDYS 45 on dystrophin production was evaluated in one study in male DMD patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping (Study 1; NCT02500381). Study 1 is an ongoing, double-blind, placebo-controlled, multicenter study designed to evaluate the safety and efficacy of AMONDYS 45 in ambulatory patients. The study is planned to enroll a total of 111 patients, age 7 to 13 years, randomized to AMONDYS 45 or placebo in a 2 to 1 ratio. Patients were required to have been on a stable dose of oral corticosteroids for at least 24 weeks prior to...

The following clinically significant adverse reactions are described elsewhere in the labeling: Hypersensitivity reactions [see Warnings and Precautions ( 5.1 )] The most common adverse reactions (incidence >20% and at least 5% higher than placebo) were upper respiratory tract infection, cough, pyrexia, headache, arthralgia, and oropharyngeal pain. ( 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.f...

AMONDYS 45 is contraindicated in patients with known serious hypersensitivity to casimersen or to any of the inactive ingredients in AMONDYS 45. Instances of hypersensitivity, including angioedema and anaphylaxis, have occurred in patients receiving AMONDYS 45 [see Warnings and Precautions ( 5.1 )] . AMONDYS 45 is contraindicated in patients with serious hypersensitivity to casimersen or to any of...

Hypersensitivity Reactions: Hypersensitivity reactions, including angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion, interrupting or discontinuing the AMONDYS 45 infusion ( 2.4 , 5.1 ) Kidney Toxicity: Based on animal data, may cause kidney toxicity. Kidney function should be monitored; creatinine may not be a reliable measure of renal function in...