Brand Name
Elevidys
Generic Name
Delandistrogene Moxeparvovec-Rokl
View Brand Information FDA approval date: June 22, 2023
Form: Kit
What is Elevidys (Delandistrogene Moxeparvovec-Rokl)?
ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy in individuals at least 4 years of age: For patients who are ambulatory and have a confirmed mutation in the DMD gene [see Clinical Pharmacology ( 1.
Approved To Treat
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Brand Information
ELEVIDYS (delandistrogene moxeparvovec-rokl)
WARNING: ACUTE SERIOUS LIVER INJURY AND ACUTE LIVER FAILURE
- Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred with ELEVIDYS[see Warnings and Precautions (
- Patients with preexisting liver impairment may be at higher risk[see Warnings and Precautions (
- Prior to infusion, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroids before and after ELEVIDYS infusion. Continue to monitor liver function weekly for the first 3 months after infusion and continue until results are unremarkable[see Dosage and Administration (2.1, 2.2, 2.4)].
- Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion[see Dosage and Administration (
- Obtain prompt consultation with a specialist (e.g., gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected[see Dosage and Administration (
1INDICATIONS 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 (.
2DOSAGE AND ADMINISTRATION
For single-dose intravenous infusion only.
2.1Critical Dosing Information
- Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion.
- Prior to ELEVIDYS infusion:
- Select patients for treatment with ELEVIDYS with anti-AAVrh74 total binding antibody titers <1:400. An FDA-authorized test for the detection of anti-AAVrh74 total binding antibodies is not currently available. Currently available tests may vary in accuracy and design.
- Avoid ELEVIDYS administration in patients with elevated anti-AAVrh74 total binding antibody titers (≥1:400)
- Due to the increased risk of serious systemic immune response, postpone ELEVIDYS in patients with active or recent (within 4 weeks) infections [see Warnings and Precautions (
- Assess liver function (clinical examination and laboratory testing including aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma-glutamyl transferase (GGT), albumin, activated partial thromboplastin time (aPTT), international normalized ratio (INR), and total bilirubin) [see Dosage and Administration (.
- Obtain platelet count and troponin-I levels
- Do not re-administer ELEVIDYS.
2.2Recommended Dose
The recommended dose of ELEVIDYS is 1.33 × 10
For the number of vials required, refer to
Calculate the dose as follows:
ELEVIDYS dose (in mL) = patient body weight (rounded to the nearest kilogram) x 10
The multiplication factor 10 represents the per kilogram dose (1.33 × 1014vg/kg) divided by the amount of vector genome copies per mL of the ELEVIDYS suspension (1.33 × 1013vg/mL).
Number of ELEVIDYS vials needed = ELEVIDYS dose (in mL) divided by 10.
Example: Calculation of volume needed for a 19.5 kg patient
Number of ELEVIDYS vials needed = 200 divided by 10, rounded to the nearest number of vials = 20 vials
Administer corticosteroids to reduce the risk of immune responses to the AAVrh74 vector after administration of ELEVIDYS
Modify oral corticosteroid doses according to Table 2 for patients with liver function abnormalities (e.g., GGT >= 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.
2.3Preparation
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.
- Inspect vials to ensure no ice crystals are present prior to preparation.
- When thawed, swirl gently.
- 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.
- 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
- Maintain syringes at room temperature prior to and during administration.
3DOSAGE FORMS AND STRENGTHS
ELEVIDYS is a preservative-free, sterile, clear, colorless liquid that may have some opalescence and may contain white to off-white particles.
ELEVIDYS is a suspension for intravenous infusion with a nominal concentration of 1.33 × 10
ELEVIDYS is provided in a customized kit containing ten to seventy 10 mL single-dose vials, with each kit constituting a dosage unit based on the patient's body weight
4CONTRAINDICATIONS
ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene [see Warnings and Precautions (.
5DRUG INTERACTIONS
Prior to initiating the corticosteroid regimen required before ELEVIDYS administration, consider the patient's vaccination status. Patients should, if possible, be brought up to date with all immunizations in agreement with current immunization guidelines. Vaccinations should be completed at least 4 weeks prior to initiation of the corticosteroid regimen.
6DESCRIPTION
ELEVIDYS (delandistrogene moxeparvovec-rokl) is a recombinant gene therapy designed to deliver the gene encoding the ELEVIDYS micro-dystrophin protein. ELEVIDYS is a non-replicating, recombinant, adeno-associated virus serotype rh74 (AAVrh74) based vector containing the ELEVIDYS micro-dystrophin transgene under the control of the MHCK7 promoter. The genome within the ELEVIDYS AAVrh74 vector contains no viral genes and consequently is incapable of replication or reversion to a replicating form. The micro-dystrophin protein expressed by ELEVIDYS is a shortened version (138 kDa, compared to 427 kDa size of dystrophin expressed in normal muscle cells) that contains selected domains of dystrophin expressed in normal muscle cells.
ELEVIDYS is a preservative-free, sterile, clear, colorless liquid that may have some opalescence and may contain white to off-white particles. ELEVIDYS is a suspension for intravenous infusion with a nominal concentration of 1.33 ×10
7CLINICAL STUDIES
The efficacy of ELEVIDYS was evaluated in two double-blind, placebo-controlled studies (Study 1 [NCT 03769116] and Study 3 [NCT 05096221]) and one open-label study (Study 2 [NCT 04626674]) in which a total of 214 male patients with a confirmed disease-causing mutation in the
8PATIENT COUNSELING INFORMATION
Advise the patient to read the FDA-approved Medication Guide.
Provide a copy of the Medication Guide and review the contents with the patient.