Is Delandistrogene moxeparvovec approved by the FDA?

Delandistrogene moxeparvovec, known by the brand name Elevidys, received FDA approval for the treatment of Duchenne muscular dystrophy in children on June 22, 2023. This approval provides a new treatment option for a condition characterized by progressive muscle degeneration and weakness due to alterations of a protein called dystrophin that helps keep muscle cells intact.

How Delandistrogene Moxeparvovec Works

Delandistrogene moxeparvovec delivers a functional copy of the dystrophin gene to muscle cells, which can help produce the dystrophin protein that patients with DMD are lacking. This treatment aims to improve muscle function and slow the progression of the disease.

Administration

Dosage Form: Intravenous kit (rokl)
Dosing and Administration: Given as a single dose, administered through an infusion pump into a vein by a healthcare provider. The dosage is weight-based, and patients will undergo frequent blood tests before and after the treatment.
Supportive Medications: Patients will be prescribed other medications to prevent serious side effects before and after receiving delandistrogene moxeparvovec. These medications must be taken as directed by the healthcare provider.

Side Effects

Common side effects of delandistrogene moxeparvovec include:

Fever
Low platelet levels
Nausea and vomiting
Abnormal liver function tests

Serious side effects can occur weeks or months after treatment, such as:

Liver problems (e.g., jaundice, dark urine)
Respiratory issues (e.g., shortness of breath)
Muscular issues (e.g., increased muscle pain)

Patients should seek immediate medical attention if they experience any severe symptoms.

Precautions

Gene Mutation: The child's specific gene mutation must be confirmed before starting treatment.
Infections and Antibodies: Inform the doctor about any recent infections or high levels of anti-AAVrh74 antibodies.
Liver and Heart Conditions: Patients with a history of liver or heart problems should discuss these conditions with their doctor.
Vaccinations: Ensure the child is up to date with all vaccines at least four weeks before starting treatment.

Storage and Handling

Store vials in the refrigerator at 2°C to 8°C (36°F to 46°F) and protect from light.
Do not freeze or shake the vials.
Unopened vials may be kept at room temperature for up to three days before use and should be returned to refrigeration if not used immediately.

Delandistrogene moxeparvovec represents a significant advancement in the treatment of Duchenne muscular dystrophy, offering hope to patients and families affected by this debilitating condition. Its FDA approval marks an important milestone in the availability of gene therapy for rare genetic disorders.

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