Is Trofinetide approved by the FDA?

Yes, Trofinetide, marketed under the brand name Daybue, is FDA approved. The FDA approved Daybue on March 10, 2023, for the treatment of Rett syndrome in adults and children aged 2 years and older.

Indications: 

Daybue (trofinetide) is indicated for:

• The treatment of Rett syndrome, a rare, severe neurodevelopmental disorder that predominantly affects females and leads to severe cognitive and physical impairments.

How Does Trofinetide Work? 

Trofinetide is designed to address the underlying pathophysiology of Rett syndrome by modifying the neuroinflammation and synaptic dysfunction associated with the condition. The exact mechanism of action is not fully understood, but it is believed to improve the symptoms and overall function in patients with Rett syndrome.

Usage Instructions:

• Dosage: Trofinetide is usually taken twice per day, in the morning and evening, with or without food. Doses are based on weight.
• Administration: Trofinetide can be given orally or through a gastrostomy feeding tube. Follow your doctor's instructions carefully. Measure the liquid medicine with the supplied measuring device (not a kitchen spoon).
• Storage: Store tightly closed in an upright position in the refrigerator. Do not freeze. Discard the medicine 14 days after first opening the bottle, even if there is still medicine left inside.

Trofinetide Dosing Information:

Adults and Adolescents (18 years and older):

• Weight 9 to less than 12 kg: 5,000 mg (25 mL) orally twice a day
• Weight 12 to less than 20 kg: 6,000 mg (30 mL) orally twice a day
• Weight 20 to less than 35 kg: 8,000 mg (40 mL) orally twice a day
• Weight 35 to less than 50 kg: 10,000 mg (50 mL) orally twice a day
• Weight 50 kg or more: 12,000 mg (60 mL) orally twice a day

Children (2 to less than 18 years):

• Weight 9 to less than 12 kg: 5,000 mg (25 mL) orally twice a day
• Weight 12 to less than 20 kg: 6,000 mg (30 mL) orally twice a day
• Weight 20 to less than 35 kg: 8,000 mg (40 mL) orally twice a day
• Weight 35 to less than 50 kg: 10,000 mg (50 mL) orally twice a day
• Weight 50 kg or more: 12,000 mg (60 mL) orally twice a day

What to Do If You Miss a Dose:

• Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.

What to Do In Case of Overdose:

• Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Side Effects: 

Common side effects may include:

• Diarrhea
• Vomiting

Serious side effects requiring immediate medical attention include:

• Severe diarrhea
• Weight loss
• Dehydration symptoms (e.g., dizziness, confusion, feeling very thirsty, less urination)

Patients experiencing any severe side effects should contact their healthcare provider promptly. Side effects can be reported to the FDA at 1-800-FDA-1088.

Warnings and Precautions:

• Laxatives: Patients using laxatives should stop taking them before starting trofinetide.
• Pregnancy and Breastfeeding: It is not known if trofinetide will harm an unborn baby. Patients should inform their doctor if they are pregnant or plan to become pregnant. Consult a doctor to determine if it is safe to breastfeed while using trofinetide.
• Pre-existing Conditions: Patients should inform their doctor if they have kidney disease or use a laxative.

Drug Interactions: 

Other drugs may affect trofinetide, including prescription and over-the-counter medicines, vitamins, and herbal products. Patients should inform their doctor about all other medicines they are currently using to avoid potential interactions.

Conclusion: 

Daybue (trofinetide) is an FDA-approved oral liquid medication for the treatment of Rett syndrome in individuals aged 2 years and older. Approved on March 10, 2023, it provides a significant therapeutic option for managing this severe neurodevelopmental disorder. Patients prescribed this medication should adhere to their healthcare provider's instructions and be mindful of potential side effects and drug interactions.

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