Delandistrogene Moxeparvovec

Delandistrogene Moxeparvovec is a prescription medication indicated for Duchenne muscular dystrophy (DMD) in ambulatory or nonambulatory pediatric patients aged 4 years and older with a confirmed mutation in the DMD gene.

• Delandistrogene Moxeparvovec is available under the following different brand names: Elevidys, delandistrogene Moxeparvovec-rokl.

Common side effects of Delandistrogene Moxeparvovec include:

• vomiting 
• nausea
• acute liver injury
• pyrexia
• thrombocytopenia (abnormally low platelet count in the blood)
• upset stomach or throwing up

Serious side effects of Delandistrogene Moxeparvovec include:

• muscle weakness, including myocarditis

Rare side effects of Delandistrogene Moxeparvovec include:

• none 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, coordination loss, unsteady, very stiff muscles, high fever, profuse sweating, or tremors.
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights.
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Pediatric dosage

Suspension for IV infusion

• 1.33 × 1013 vector genomes (vg)/mL
• Provided in customized kit containing ten to seventy 10-mL single-dose vials, with each kit constituting a dosage unit based on patient’s body weight

Duchenne muscular dystrophy

Pediatric dosage

• Single-dose IV infusion
  • Children weighing less than 70 kg: 1.33 x 1014 vector genomes (vg)/kg of body weight (or 10 mL/kg)
  • Children weighing 70 kg and more (fixed dose): 9.31 x 1015 vg/kg of body weight
• Calculate dose (less than 70 kg)
  • Dose (in mL) = patient body weight (in kg) × 10
  • The multiplication factor 10 represents the per kilogram dose (1.33 × 1014 vg/kg) divided by the amount of vector genome copies per mL of the suspension (1.33 × 1013 vg/mL)
  • Number of vials needed = dose (in mL) divided by 10 (round to nearest number of vials)
  • Example: Calculation of volume for 19.6 kg patient
    • 19.6 kg × 10 = 196 mL
    • Number of vials needed = 196 divided by 10, rounded to the nearest number of vials = 20 vials
• Pre- and post-infusion corticosteroid dosing
  • Not to exceed prednisone (or prednisone equivalent) total daily dose (TDD) of 60 mg/day
  • Deflazacort is not recommended for use as a peri-infusion corticosteroid.
  • Baseline corticosteroids use daily or intermittently.
  • Start 1 day before the infusion.
  • 1 mg/kg/day (and continue baseline dose)
  • Baseline high-dose corticosteroids 2 days/week
  • Not to exceed 60 mg/day
  • Not on corticosteroids
    • Start 1 week before infusion.
    • 1.5 mg/kg per day
    • Not to exceed 60 mg/day
• Recommended corticosteroid regimen dose modification for liver function abnormalities following infusion.
  • GGT above 150 U/L and/or other clinically significant liver function abnormalities (. eg, total bilirubin above 2 × ULN) following infusion; for GGT or bilirubin elevations that do not respond to these oral corticosteroid increases, IV bolus corticosteroids may be considered; deflazacort is not recommended for use as a peri-infusion corticosteroid.
  • Not to exceed prednisone (or prednisone equivalent) TDD of 120 mg/day.
  • Peri-infusion baseline + 1 mg/kg/day: Increase to 2 mg/kg/day (and continue baseline dose)
  • Peri-infusion baseline + 1 mg/kg/day (on days without high-dose corticosteroids): Increase to 2 mg/kg/day on days without high-dose corticosteroids.
  • Peri-infusion 1.5 mg/kg/day: Increase to 2.5 mg/kg/day.

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Delandistrogene Moxeparvovec has severe interactions with no other drugs
• Delandistrogene Moxeparvovec has serious interactions with no other drugs
• Delandistrogene Moxeparvovec has moderate interactions with the following drugs:
  • diphtheria & tetanus toxoids
  • diphtheria & tetanus toxoids/ acellular pertussis vaccine
  • diphtheria & tetanus toxoids/acellular pertussis/poliovirus, inactivated vaccine
  • haemophilus influenzae type b vaccine
  • hepatitis b vaccine
  • influenza virus vaccine quadrivalent
  • influenza virus vaccine quadrivalent, cell-cultured
  • measles mumps and rubella vaccine, live
  • meningococcal A C Y and W polysaccharide tetanus toxoid conjugate vaccine
  • meningococcal C and Y/haemophilus influenza type B vaccine
  • pneumococcal vaccine 13-valent
  • pneumococcal vaccine 15-valent
  • pneumococcal vaccine 20-valent
  • poliovirus vaccine inactivated
  • rotavirus oral vaccine, live
  • tetanus & reduced diphtheria toxoids/ acellular pertussis vaccine
  • varicella virus vaccine live
  • varicella zoster immune globulin, human
• Delandistrogene Moxeparvovec has minor interactions with no other drugs

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all the products you use. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

• Contraindications
• Patients with any deletion in exon 8 and/or exon 9 in the DMD gene

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Delandistrogene Moxeparvovec?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Delandistrogene Moxeparvovec?”
• Cautions
• Infusion-related reactions

  • Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following administration

  • Closely monitor during and for at least 3 hours after completing infusion for signs and symptoms including tachycardia, tachypnea, lip swelling, difficulty breathing, nasal flaring, urticaria, flushing, lip pruritus, rash, cheilitis, vomiting, nausea, rigors, and pyrexia

  • Administer in setting where treatment for infusion-related reactions is immediately available

  • If infusion-related reaction occurs, may slow or stops infusion based on severity

  • Administer treatment as needed to manage reactions based on the severity

  • If infusion stopped, may restart at lower rate once symptoms have resolved, at the discretion of the physician

• Acute serious liver injury
  • Acute serious liver injury observed; administration may result in elevated liver enzymes (eg, GGT, ALT) and total bilirubin, typically seen within 8 weeks; if suspected, consultation with a specialist is recommended
  • Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (eg, acute hepatic viral infection) may be at higher risk of acute serious liver injury
  • Postpone administration in patients with acute liver disease until resolved or controlled
  • Patients with hepatic impairment, acute liver disease, chronic hepatic condition, or elevated GGT have not been studied in clinical trials
  • In clinical studies, liver function test increases (including increases in GGT, GLDH, ALT, AST, or total bilirubin) were commonly reported, typically within 8 weeks following infusion; majority of cases were asymptomatic
  • Cases observed resolved spontaneously or with systemic corticosteroids and resolved without clinical sequelae within 2 months; no cases of liver failure were reported
  • Before administration, perform liver enzyme testing, then monitor liver function (clinical exam, GGT, and total bilirubin) weekly × 3 months following infusion
  • Continue monitoring if clinically indicated, until results are unremarkable
  • Systemic corticosteroid treatment is recommended for patients before and after infusion; adjust corticosteroid regimen when indicated
• Immune-mediated myositis
  • Immune-mediated myositis was observed approximately 1 month following infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene
  • Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed
  • In a life-threatening case of immune-mediated myositis, symptoms resolved during hospitalization following additional immunomodulatory treatment; muscle strength gradually improved but did not return to the baseline level
  • Immune reactions may be due to a T-cell-based response from lack of self-tolerance to a specific region encoded by the transgene corresponding to exons 1-17 of the DMD gene
  • Limited data are available regarding Delandistrogene Moxeparvovec treatment in patients with mutations in the DMD gene in exons 1-17 and/or exons 59-71; patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction
  • Contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene due to increased risk for severe immune-mediated myositis
  • Advise caregivers to contact a physician immediately if a child experiences any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia as these may be symptoms of myositis
  • Consider additional immunomodulatory treatment (immunosuppressants (eg, calcineurin-inhibitor] in addition to corticosteroids) based on the patient’s clinical presentation and medical history if these symptoms occur
  • Myocarditis
    • Acute serious myocarditis and troponin-I elevations were observed following infusion
    • Monitor troponin-I before infusion and weekly for the first month following infusion
    • Continue monitoring if clinically indicated; more frequent monitoring may be warranted in the presence of cardiac symptoms (eg, chest pain, shortness of breath)
    • Advise caregivers to contact a physician immediately if a child experience cardiac symptom
    • Pre-existing immunity against AAVhr74
    • In AAV-vector-based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels
    • Following treatment all subjects developed anti-AAVrh74 antibodies
    • Perform baseline testing for the presence of anti-AAVrh74 total binding antibodies before administration
    • Not recommended in patients with elevated anti-AAVrh74 total binding antibody titers (more than 1:400)
  • Drug interaction overview
    • Vaccinations
      • Complete vaccinations at least 4 weeks before initiating the pretreatment corticosteroid regimen

Pregnancy and Lactation

• Not intended for use in pregnant women
• Lactation
  • Not intended for use in lactating women