Is Vamorolone approved by the FDA?

Vamorolone, marketed under the brand name Agamree, was approved by the FDA on October 26, 2023. It is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 2 years and older.

What is Vamorolone?

Vamorolone is a steroid medication used to treat Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration and weakness. It helps manage the symptoms and slows the progression of the disease. The medication is suitable for patients aged 2 years and older.

How to Use Vamorolone

Dosage: Vamorolone is usually taken once a day with a meal. Dosage is based on weight, and adjustments may be necessary if there are significant changes in weight.
Preparation: Shake the oral suspension for about 30 seconds before use. Measure the dose with the supplied device and do not mix it with any other liquids.
Administration: Follow the prescription label and doctor’s instructions carefully. Do not stop using vamorolone suddenly; taper the dose gradually as per the doctor's instructions to avoid adrenal insufficiency.

Common Side Effects

Weight gain and swelling
Mood or behavior changes
Acne and increased facial or body hair growth
Vomiting and tiredness
Low blood vitamin D levels

Serious Side Effects

Seek immediate medical attention if you experience:

Allergic reactions: hives, difficulty breathing, swelling of face, lips, tongue, or throat
Signs of infection: fever, chills, sore throat, mouth sores, swollen gums, weakness, diarrhea, burning during urination
Increased adrenal gland hormones: hunger, weight gain, swelling, skin discoloration, slow wound healing, sweating, acne, increased body hair, tiredness, mood changes, missed menstrual periods, sexual changes
Blood clot symptoms: chest pain, sudden cough, shortness of breath, dizziness, coughing up blood, pain, swelling, or warmth in one leg
Hepatitis reactivation: right-sided upper stomach pain, vomiting, loss of appetite, yellowing of skin or eyes
Electrolyte imbalances: muscle spasms, cramps, numbness or tingling, constipation, irregular heartbeats
High blood sugar: increased thirst, increased urination, dry mouth, fruity breath odor
Mental health changes: new or worsening depression or anxiety, panic attacks, trouble sleeping, impulsive or aggressive behavior
Any skin lesion, muscle weakness, vision changes, or severe headache

Warnings and Precautions

Infection Risk: Vamorolone can weaken the immune system, increasing the risk of infections or worsening existing infections.
Medical Conditions: Inform your doctor if you have any medical history including high blood pressure, stomach or intestinal disorders, heart disease, recent vaccinations, eye infections, thyroid or adrenal gland problems, diabetes, blood clotting disorders, nerve-muscle disorders, liver or kidney disease.
Pregnancy and Breastfeeding: It is not known if vamorolone will harm an unborn baby. Consult your doctor if you are pregnant or plan to become pregnant. Discuss breastfeeding safety with your doctor.

Drug Interactions

Vamorolone can interact with various medications. Inform your doctor about all the medicines you are taking, especially:

Itraconazole
Muscle relaxers like rocuronium or pancuronium

This is not a complete list of potential interactions. Always consult your doctor before starting or stopping any medications while on vamorolone.

Storage

Before opening: Store upright in the original container at room temperature, away from moisture and heat.
After opening: Store in the refrigerator, do not freeze. Discard any remaining suspension 3 months after opening.

Conclusion

Vamorolone (Agamree) was FDA-approved on October 26, 2023, for the treatment of Duchenne muscular dystrophy in patients aged 2 years and older. As a glucocorticoid, it offers a therapeutic option for managing symptoms and slowing disease progression. If you are considering vamorolone, follow your healthcare provider's instructions and be aware of potential side effects and interactions.

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