Trofinetide: First Approval
Related Experiment Video
Updated: Jul 30, 2025

The NeuroStar TMS Device: Conducting the FDA Approved Protocol for Treatment of Depression
Published on: November 12, 2010
1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.
Abstract:
Trofinetide (DAYBUE™), an oral, small molecule, synthetic analog of glycine-proline-glutamate [GPE; the N-terminal tripeptide derivative of insulin like growth factor-1 (IGF-1)], is being developed by Neuren Pharmaceuticals and Acadia Pharmaceuticals for the treatment of rare childhood neurodevelopmental disorders. Trofinetide was approved in March 2023 in the USA for the treatment of Rett syndrome in adult and pediatric patients 2 years of age and older. This article summarizes the milestones in the development of trofinetide leading to this first approval for Rett syndrome.
More Related Videos
11:06Subretinal Implantation of RPE on a Carrier in Minipigs: Guidelines for Preoperative Preparations, Surgical Techniques, and Postoperative Care
Published on: November 11, 2022
13:10Combined Near-infrared Fluorescent Imaging and Micro-computed Tomography for Directly Visualizing Cerebral Thromboemboli
Published on: September 25, 2016
Related Concept Videos
Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
Treatment for Pulmonary Arterial Hypertension: Prostacyclin Receptor Agonists
These agonists bind to the IPR receptor situated on the plasma membrane of the pulmonary artery smooth muscle cells. This binding triggers a cascade of reactions known as the GS-AC-cAMP-PKA pathway. This pathway results in the relaxation of smooth muscle...
Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers
TKIs, such as imatinib (Gleevec), are particularly effective in tackling the growth and mitogenic factors that become upregulated in PAH patients. These factors contribute to the...
Clinical Trials: Overview
Drugs for Treatment of Crohn's Disease in IBD Using Biologic Agents: Anti-TNF
Drug Administration and Therapy Phases: Overview
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...