Casimersen

AMONDYS 45 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 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. AMONDYS 45 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 45 skipping.

Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon 45 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 45 skipping.

Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting AMONDYS 45 30 milligrams per kilogram of body weight once weekly. Administer as an intravenous (IV) infusion over 35 to 60 minutes via an in-line 0.2 micron filter. Dilution required prior to administration

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. AMONDYS 45 is contraindicated in patients with serious hypersensitivity to casimersen or to any of the inactive ingredients in AMONDYS 45.

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. 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 DMD patients.

The following clinically significant adverse reactions are described elsewhere in the labeling: Hypersensitivity reactions 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.

Store AMONDYS 45 at 2°C to 8°C (36°F to 46°F). Do not freeze. Store in original carton until ready for use to protect from light.