EXONDYS 51 DOSING CALCULATIONS.1
The required dosage can be calculated using the following formula:
STEP 1: DOSE
STEP 2: VOLUME
STEP 3: VIALS
EXAMPLE CALCULATION BASED ON A 33.5 KG PATIENT:
Step 1: 33.5 kg x 30 mg/kg = 1,005 mg
Step 2: 1,005 ÷ 50 mg/mL = 20.1 mL = 21 mL of EXONDYS 51
(rounded to next higher mL)
Step 3: 21 mL ÷ 10 = two 10 mL vials + one 2 mL vial
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.