ELEVIDYS

2 DOSAGE AND ADMINISTRATION For single-dose intravenous infusion only. ELEVIDYS is for single-dose intravenous infusion only. Select patients for treatment with ELEVIDYS with anti-AAVrh74 total binding antibody titers = 3 times baseline, total bilirubin > ULN) following ELEVIDYS infusion. Consider IV bolus corticosteroids instead of oral corticosteroids for GGT or bilirubin elevations that do not respond after 1 week of increased oral corticosteroids. Consult with a specialist experienced in immunosuppressive therapy for additional interventions as needed. Obtain prompt consultation with a specialist (e.g., gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected. Taper the additional peri-ELEVIDYS corticosteroids for patients previously taking corticosteroids at baseline back to baseline dose over 2 weeks, or longer as needed. Taper the peri-ELEVIDYS corticosteroids for patients not previously taking corticosteroids at baseline back to no corticosteroids over 4 weeks, or longer as needed. Do not stop corticosteroids abruptly. Table 2: Recommended oral corticosteroid regimen dose modification for liver function abnormalities following ELEVIDYS infusion a Peri-ELEVIDYS infusion corticosteroid dosing Modified oral corticosteroid dose following ELEVIDYS infusion (prednisone equivalent) b c Recommended maximum total daily oral dose (prednisone equivalent) b c a GGT >= 3 times baseline and/or other clinically significant liver function abnormalities (e.g., total bilirubin > ULN) following infusion. b Consider IV bolus corticosteroids instead of oral corticosteroids for GGT or bilirubin elevations that do not respond after 1 week of increased oral corticosteroids. Consult with a specialist experienced in immunosuppressive therapy for additional interventions as needed. c Corticosteroids other than prednisone and prednisolone have not been studied for use as a peri-ELEVIDYS infusion corticosteroid. Baseline + 1 mg/kg/day Increase to 2 mg/kg/day (and continue baseline dose) 120 mg/day Baseline + 1 mg/kg/day taken on days without high-dose corticosteroid treatment Increase to 2 mg/kg/day taken on days without high-dose corticosteroid treatment (and continue baseline dose) 120 mg/day 1.5 mg/kg/day Increase from 1.5 mg/kg/day to 2.5 mg/kg/day 120 mg/day 2.3 Preparation General precautions Prepare ELEVIDYS using aseptic technique. Verify the required dose of ELEVIDYS based on the patient's body weight. Confirm that the kit contains sufficient number of vials to prepare the ELEVIDYS infusion for the patient. Visually inspect parenteral drug products for particulate matter and discoloration prior to administration, whenever suspension and container permit. ELEVIDYS may contain white to off-white particles. Recommended supplies and materials: 60 mL siliconized polypropylene syringes 21-gauge maximum or smaller stainless steel needles Preparing ELEVIDYS infusion Thaw ELEVIDYS before use. When thawed in the refrigerator, ELEVIDYS vials are stable for up to 14 days in the refrigerator (2°C to 8°C [36º F to 46º F]) when stored in the upright position. Frozen ELEVIDYS vials will thaw in approximately 2 hours when placed at room temperature (up to 25°C [77ºF]) when removed from original packaging. Thawed ELEVIDYS is stable for up to 24 hours at room temperature (up to 25°C [77ºF]). Inspect vials to ensure no ice crystals are present prior to preparation. When thawed, swirl gently. Do not shake. Do not refreeze. Do not place back in the refrigerator. Visually inspect each vial of ELEVIDYS. ELEVIDYS is a clear, colorless liquid that may have some opalescence. ELEVIDYS may contain white to off-white particles. Do not use if the suspension in the vials is cloudy or discolored. Remove the plastic flip-off cap from the vials and disinfect the rubber stopper with a sterilizing agent (e.g., alcohol wipes). Withdraw 10 mL of ELEVIDYS from each vial provided in the customized ELEVIDYS kit (refer to Table 10 ). Do not use filter needles during preparation of ELEVIDYS. Multiple syringes will be required to withdraw the required volume. Remove air from the syringes and cap the syringes. Maintain syringes at room temperature prior to and during administration. 2.4 Administration Recommended supplies and materials: Syringe infusion pump 0.2-micron PES* in-line filter with a large surface area. To avoid the risk of occlusions, the use of smaller pediatric in-line filters (e.g., less than 10 cm 2 surface area) is not recommended. PVC* (non-DEHP*) IV infusion tubing, and polyurethane catheter *PVC = Polyvinyl chloride, DEHP = Di(2-ethylhexyl) phthalate, PES = Polyether sulfone Administer ELEVIDYS as a single-dose intravenous infusion through a peripheral venous catheter: ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available [see Warnings and Precautions ( 5.4 )] . Do not infuse ELEVIDYS at a rate of 10 mL/kg/hour or faster. Consider application of a topical anesthetic to the infusion site prior to administration of IV insertion. Recommend inserting a back-up catheter. Flush the intravenous access line with 0.9% Sodium Chloride Injection prior to the ELEVIDYS infusion at the same infusion rate. Administer ELEVIDYS via intravenous infusion using a syringe infusion pump with an in-line 0.2-micron filter at a duration of approximately 1 to 2 hours, or longer at care team discretion, through a peripheral limb vein. Infuse at a rate of less than 10 mL/kg/hour. Do not administer ELEVIDYS as an intravenous push. Do not infuse ELEVIDYS in the same intravenous access line with any other product. Use ELEVIDYS within 12 hours after drawing into syringe. Discard the ELEVIDYS-containing syringe(s) if infusion of the drug has not been completed within the 12-hour timeframe. In the event of an infusion-related reaction during administration [see Warnings and Precautions ( 5.4 )]: Slow or stop the infusion based on patient's clinical presentation. Discontinue infusion for anaphylaxis. Administer treatment as needed to manage infusion-related reaction. ELEVIDYS infusion may be restarted at a lower rate after the infusion-related reaction has resolved at the discretion of the physician, based on severity of patient's clinical presentation. If the ELEVIDYS infusion needs to be stopped and restarted, ELEVIDYS should be infused within 12 hours after drawing into the syringe [see How Supplied/Storage and Handling ( 16.2 )] . Flush the intravenous access line with 0.9% Sodium Chloride Injection after the ELEVIDYS infusion. Discard unused ELEVIDYS [see How Supplied/Storage and Handling ( 16.2 )]. Dispose of the needle and syringe [see How Supplied/Storage and Handling ( 16.2 )]. Monitoring Post-ELEVIDYS Administration Assess liver function (clinical exam, AST, ALT, GGT, albumin, aPTT, INR, and total bilirubin) weekly for the first 3 months. Continue monitoring if clinically indicated, until results are unremarkable (e.g., normal clinical exam, GGT and total bilirubin levels return to near baseline levels) [see Warnings and Precautions ( 5.1 ), Specific Populations ( 8.6 )] . Obtain platelet counts weekly for the first two weeks [see Adverse Reactions ( 6.1 )] . Continue monitoring if clinically indicated. Measure troponin-I weekly for the first month [see Warnings and Precautions ( 5.3 )] . Continue monitoring if clinically indicated, until results return to near baseline levels or stabilize.

See official label

1 INDICATIONS AND USAGE ELEVIDYS is indicated for the treatment of patients 4 years of age and older with Duchenne muscular dystrophy (DMD), who are ambulatory and have a confirmed mutation in the DMD gene [see Clinical Pharmacology ( 12.2 ), Clinical Studies ( 14 )] .