Trofinetide

Overview

Trofinetide is a novel synthetic analog of glypromate, also known as glycine–proline–glutamate (GPE), a naturally occurring protein in the brain and the N-terminal tripeptide of insulin-like growth factor 1 (IGF-1). Trofinetide was approved by the FDA on March 10, 2023, for the treatment of Rett syndrome, which is an X-linked neurodevelopmental disorder characterized by a range of cognitive, motor, and autonomic symptoms. Trofinetide is believed to work by reducing inflammation and apoptosis of neurons.

Indication

Trofinetide is indicated for the treatment of Rett syndrome in adults and pediatric patients two years of age and older.

Associated Conditions

Rett Syndrome

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome	2021/08/04	NCT04988867	Phase 2	Completed	ACADIA Pharmaceuticals Inc.
Open-Label Extension Study of Trofinetide for Rett Syndrome	2021/03/02	NCT04776746	Phase 3	Completed	ACADIA Pharmaceuticals Inc.
Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome	2020/02/21	NCT04279314	Phase 3	Completed	ACADIA Pharmaceuticals Inc.
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)	2019/11/29	NCT04181723	Phase 3	Completed	ACADIA Pharmaceuticals Inc.
A Safety Study of NNZ-2566 in Pediatric Rett Syndrome	2016/03/22	NCT02715115	Phase 2	Completed	Neuren Pharmaceuticals Limited
A Safety and Efficacy Study of NNZ-2566 in Patients With Mild Traumatic Brain Injury (mTBI)	2014/03/31	NCT02100150	Phase 2	Terminated	Neuren Pharmaceuticals Limited
A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome	2013/07/10	NCT01894958	Phase 2	Completed	Neuren Pharmaceuticals Limited
A Safety Study of NNZ-2566 in Patients With Rett Syndrome	2012/10/10	NCT01703533	Phase 2	Completed	Neuren Pharmaceuticals Limited
Safety Study of NNZ-2566 in Healthy Subjects, Following Oral Administration	2011/08/19	NCT01420042	Phase 1	Completed	Neuren Pharmaceuticals Limited
Study of NNZ-2566 in Patients With Traumatic Brain Injury Under EFIC	2011/06/06	NCT01366820	Phase 2	Completed	Neuren Pharmaceuticals Limited

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
Daybue	Acadia Pharmaceuticals Inc.	63090-660	ORAL	200 mg in 1 mL	3/16/2023

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
No EMA approvals found for this drug.

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
No TGA approvals found for this drug.

Related News

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23 days ago

Acadia Pharmaceuticals outlined an ambitious multi-year strategy with a pipeline potentially generating up to $12 billion in annual peak sales across eight clinical programs.

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Acadia Pharmaceuticals has appointed Allyson McMillan-Youngblood as Senior Vice President, Rare Disease Franchise, bringing over 20 years of pharmaceutical industry experience.

FDA Approves Trofinetide as First-Ever Treatment for Rett Syndrome, Opening New Era for Rare Genetic Disorder

3 months ago

The FDA has approved trofinetide (developed by ACADIA Pharmaceuticals) as the first-ever treatment for Rett syndrome, a rare genetic disorder primarily affecting females that causes severe developmental delays and neurological symptoms.

Marvel Biosciences to Seek Orphan Drug Designation for Novel Rett Syndrome Treatment

4 months ago

Marvel Biosciences plans to file for Orphan Drug Designation with the FDA for MB-204 as a potential treatment for Rett Syndrome following strong preclinical data that outperformed Trofinetide, the only FDA-approved treatment.

Daybue Clinical Trials Show Significant Communication and Motor Improvements in Rett Syndrome Patients

5 months ago

• Caregivers reported marked improvements in communication abilities, including new sounds and words, and enhanced eye contact in Rett syndrome patients treated with Daybue in clinical trials. • Nearly all caregivers (32 out of 33) observed meaningful improvements in patients' hand function, mobility, and motor skills, leading to greater independence in daily activities. • The study, funded by Acadia Pharmaceuticals, demonstrated high satisfaction rates among caregivers, with most reporting significant positive impact on patients' social and physical functioning.

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