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Trofinetide
Pharmaceutical drug From Wikipedia, the free encyclopedia
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Trofinetide, sold under the brand name Daybue, is a medication used for the treatment of Rett syndrome.[4] It is taken by mouth.[4]
The most common adverse reactions include diarrhea and vomiting.[5]
Trofinetide was approved for medical use in the United States in March 2023.[4][5][6][7] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[8]
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Medical uses
Trofinetide is indicated for the treatment of Rett syndrome in people two years of age and older.[4][9]
Rett syndrome is a rare, genetic neurological and developmental disorder that affects the way the brain develops.[5] People with Rett syndrome experience a progressive loss of motor skills and language.[5] Most babies with Rett syndrome seem to develop as expected for the first six months of life.[5] These babies then lose skills they previously had attained at approximately six to 18 months of age — such as the ability to crawl, walk, communicate, or use their hands.[5] The hallmark of Rett syndrome is near constant repetitive hand movements, such as rubbing or clapping.[5] Rett syndrome leads to severe impairments affecting nearly every aspect of life, including the ability to speak, walk, eat, and breathe.[5]
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History
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It was developed by Neuren Pharmaceuticals that acts as an analogue of the neuropeptide (1-3) IGF-1, which is a simple tripeptide with sequence Gly-Pro-Glu obtained by enzymatic cleavage of the growth factor IGF-1 within the brain. Trofinetide has anti-inflammatory properties and was originally developed as a potential treatment for stroke,[10][11] but has subsequently been developed for other applications and is approved by the FDA as an oral solution. It has successfully completed phase III clinical trial against Rett syndrome.[12] Trofinetide has also had a successful phase II trial against Fragile X syndrome.[13][14][15] The drug is manufactured by Acadia Pharmaceuticals.[4][16]
The US Food and Drug Administration (FDA) evaluated the efficacy and safety of trofinetide based on a randomized, double-blind, placebo-controlled, 12-week study (Study 1; NCT04181723) of participants with Rett syndrome five to 20 years of age.[5] Participants were randomized to receive trofinetide (N=93) or matching placebo (N=94) for 12 weeks.[5] The dose of trofinetide was based on participant weight to achieve similar exposure in all participants.[5] The FDA granted the application for trofinetide priority review, orphan drug, and fast track designations.[5]
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References
External links
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