Trofinetide 200 mg/mL Suppliers & Bulk Manufacturers

Available Forms: Oral Solution

Available Strengths: 200 mg/mL

Reference Brands: Daybue (USA)

Category: Orphan Drugs

Trofinetide is a synthetic analog of the naturally occurring tripeptide glycine-proline-glutamate (GPE) that is used for treating Rett syndrome in patients aged 2 years and older. It is taken orally as a solution twice daily, and aims to improve neurological function by supporting synaptic maturation and reducing neuroinflammation. It represents one of the first therapies approved specifically for Rett syndrome.

Product Description:

Trofinetide is a synthetic analogue of glypromate, a tripeptide fragment derived naturally from IGF-1 in the brain. In Rett syndrome, mutations in the MECP2 gene disrupt synapse formation and maintenance, and there is excessive neuroinflammation with abnormal glial activity. Trofinetide is thought to reduce that neuroinflammation and support synaptic function, although the precise mechanism is not established.

It is used for Rett syndrome in patients aged two and over, and it is the first drug approved for the condition anywhere — until it, care was entirely supportive. Rett syndrome affects girls almost exclusively, who develop normally for six to eighteen months and then regress, losing speech and purposeful hand use, with breathing abnormalities and seizures. In its pivotal trial it produced modest but measurable improvements on caregiver and clinician scales. It is taken twice daily as an oral solution, dosed by weight, often through a gastrostomy tube. Diarrhoea and vomiting are very common and are the main reason for stopping.

Sourcing notes. Originator orphan product, on patent, no generic. High volume per dose for an oral solution means shipping weight is significant. Registration is US-led; elsewhere, named-patient routes apply. Paediatric neurology centres are the point of care.