Elevidys (delandistrogene moxeparvovec-rokl)

It isessential that the manufacturer's labeling be consulted for more detailed information on dosage and administration of this drug. Dosage summary:

*Delandistrogene moxeparvovec-rokl is for single-dose IV infusion only.

*Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by thehealthcare provider, for at least 2 months following delandistrogene moxeparvovec-rokl infusion.

*Select patients for treatment with delandistrogene moxeparvovec-rokl with anti-AAVrh74 total binding antibody titers <1:400. An FDA-authorized test for the detection of anti-AAVrh74 total binding antibodies is not currentlyavailable. Currently available tests may vary in accuracy and design. Avoid administration of delandistrogene moxeparvovec-rokl in patients with elevated anti-AAVrh74 total binding antibody titers (>=1:400).

*Due to the increased risk of serious systemic immune response, postpone delandistrogene moxeparvovec-rok in patientswith active or recent (within 4 weeks) infections.

*Assess liver function (clinical examination and laboratory testing including AST, ALT, GGT,albumin, aPTT, INR, and totalbilirubin).

*Recommended dosage in patients weighing 10 to 70 kg: 1.33 x1014 vector genomes (vg) per kg (vg/kg) of body weight (or 10 mL/kg body weight).

*Recommended dosage in patients weighing 70 kg or greater: 9.31 x1015 vg total fixed dose.

*Calculate the dose as follows: dose (in mL) = patient body weight (rounded to the nearest kg) x 10. The multiplication factor 10 represents the per kg dose (1.33 x 1014 vg/kg) divided by the amount of vector genome copies per mL of the suspension (1.33 x 1013 vg/mL). Number of vials needed = dose (in mL) divided by 10 (round to the nearest number of vials).

*Administer as an IV infusion using a syringe infusion pump with an in-line 0.2-micron filter over approximately 1-2 hours through a peripheral limb vein. Infuse at a rate of less than 10 mL/kg/hour. Do not administer as an IV push and do not infuse into the same IV access line with any other product. Consider application of a topical anesthetic to the infusion site prior to administration of IV insertion.

*Administer in a setting where treatment for infusion-related reactions is immediately available.

*Assess liver function, platelet counts, and troponin-I levels post-administration of delandistrogene moxeparvovec-rokl.

*To reduce the risk of an immune response, administer a corticosteroid regimen one day prior to infusion and continue for a minimum of 60 days after the infusion. Corticosteroid dose modifications are recommended for patients with liver function abnormalities.

*See full prescribing information for additional information on preparation and administration, and instructions on how to manage infusion-related reactions.

No dosing information is available for this tab.

No dosing information is available for this tab.

No dosing information is available for this tab.

There is no enhanced messaging for this drug.

There are no contraindication interactions for this drug.

There are no severe interactions for this drug.

There are no moderate interactions for this drug.

*Patients with any deletion in exon 8 and/or exon 9, including a deletion of anyportion or the entirety of these exons, in the DMD gene.

There are no contraindication contraindications for this drug.

• Disease of liver
• Infection

• Left ventricular failure

Most common adverse reactions (incidence >=5%) were vomiting and nausea, liver injury, pyrexia, thrombocytopenia, and increased troponin-I.

More Frequent

• Fever

Less Frequent

• Drug-induced hepatitis
• Dyspnea
• Hepatic failure
• Myocarditis
• Myositis

Rare or Very Rare

• Acute occlusion of mesenteric vein
• Anaphylaxis
• Lower respiratory infection
• Upper respiratory infection

More Frequent

• Abnormal hepatic function tests
• Nausea
• Thrombocytopenic disorder
• Vomiting

Less Frequent

• Dysphagia
• Myalgia
• Voice change

Rare or Very Rare

• Hypersensitivity drug reaction

No enhanced messaging available.

The safety and effectiveness of delandistrogene moxeparvovec-rokl for the treatment of Duchenne muscular dystrophy have been established in pediatric patients at least 4 years of age with a confirmed mutation in the DMD gene. The use ofdelandistrogene moxeparvovec-rokl in pediatric patients was supported by evidence from three adequate and well controlled clinicalstudies which included 144 pediatric patients 4 years of age and older.

Delandistrogene moxeparvovec-rokl is not intended for use in pregnant women.

In the U.S. general population, the estimated background risks of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

There is no information available on the presence of delandistrogene moxeparvovec-rokl in human milk, the effects on the breastfed infant, or the effects on milk production.

The safety and efficacy of delandistrogene moxeparvovec-rokl in geriatric patients with DMD have not been studied.