Is Avalglucosidase approved by the FDA?

Yes, Avalglucosidase alfa is FDA approved. The U.S. Food and Drug Administration (FDA) approved Avalglucosidase alfa for the treatment of late-onset Pompe disease (LOPD) on August 6, 2021. This approval was a significant milestone for patients with Pompe disease, providing a new therapeutic option for managing this genetic disorder.

Uses and Benefits

Avalglucosidase alfa is used to replace the enzyme alpha-glucosidase that is deficient in individuals with Pompe disease. Pompe disease is a rare genetic disorder that results in the buildup of glycogen in the body's cells, leading to muscle weakness and respiratory difficulties. Avalglucosidase alfa helps break down glycogen, thereby managing the symptoms of the disease.

Administration and Dosage

Avalglucosidase alfa is administered as follows:

• Dosage Form: Intravenous injection (100 mg)
• Usual Adult Dose for Pompe Disease:
  • Less than 30 kg: 40 mg/kg IV every 2 weeks
  • At least 30 kg: 20 mg/kg IV every 2 weeks
• Usual Pediatric Dose for Pompe Disease:
  • 1 year or older:
    • Less than 30 kg: 40 mg/kg IV every 2 weeks
    • At least 30 kg: 20 mg/kg IV every 2 weeks

The medication is given by a healthcare provider through an intravenous infusion, usually once every two weeks. It must be administered slowly over 4 to 5 hours.

Side Effects

Avalglucosidase alfa can cause side effects, some of which may be serious. Common side effects include:

• Headache
• Dizziness
• Nausea and vomiting
• Diarrhea
• Itching, rash, redness
• Muscle or joint pain
• Trouble breathing
• Numbness, tingling, or burning pain

Serious side effects that require immediate medical attention include:

• Allergic reactions (hives, itching, difficulty breathing, swelling of face, lips, tongue, or throat)
• Reactions during the injection (wheezing, cough, chest discomfort, trouble breathing, pale skin, blue lips or fingernails, swelling in the tongue, trouble swallowing, dizziness, rash, or flushing)

Precautions

Before starting treatment with Avalglucosidase alfa, consider the following precautions:

• Medical Conditions: Inform your doctor if you have heart disease, lung disease, or breathing problems.
• Pregnancy and Breastfeeding: Discuss with your doctor if you are pregnant, planning to become pregnant, or breastfeeding. The benefits of treating Pompe disease during pregnancy may outweigh potential risks.
• Pompe Registry: Your name may be listed on the Pompe Registry to track the progress of your disease and the outcome of your treatment with Avalglucosidase alfa.

Conclusion

Avalglucosidase alfa offers a crucial treatment option for patients with late-onset Pompe disease. It is important to follow the prescribed administration guidelines and take necessary precautions to minimize side effects and ensure effective treatment. Always consult with a healthcare provider for personalized medical advice and treatment plans.

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