Clinical data | |
---|---|
Trade names | Daybue |
Other names | NNZ-2566 |
AHFS/ Drugs.com | Monograph |
MedlinePlus | a623019 |
License data |
|
Routes of administration | By mouth, feeding tube (gastrostomy tube) |
ATC code |
|
Legal status | |
Legal status | |
Pharmacokinetic data | |
Bioavailability | 84% |
Metabolism | Insignificant |
Elimination half-life | ~ 1.5 h |
Excretion | Urine |
Identifiers | |
| |
CAS Number | |
PubChem CID | |
DrugBank | |
ChemSpider | |
UNII | |
KEGG | |
ChEBI | |
ChEMBL | |
Chemical and physical data | |
Formula | C13H21N3O6 |
Molar mass | 315.326 g·mol−1 |
3D model ( JSmol) | |
| |
|
Trofinetide, sold under the brand name Daybue, is a medication used for the treatment of Rett syndrome. [1] It is taken by mouth. [1]
The most common adverse reactions include diarrhea and vomiting. [2]
Trofinetide was approved for medical use in the United States in March 2023. [1] [2] [3] [4] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [5]
Trofinetide is indicated for the treatment of Rett syndrome in people two years of age and older. [1] [6]
Rett syndrome is a rare, genetic neurological and developmental disorder that affects the way the brain develops. [2] People with Rett syndrome experience a progressive loss of motor skills and language. [2] Most babies with Rett syndrome seem to develop as expected for the first six months of life. [2] 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. [2] The hallmark of Rett syndrome is near constant repetitive hand movements, such as rubbing or clapping. [2] Rett syndrome leads to severe impairments affecting nearly every aspect of life, including the ability to speak, walk, eat, and breathe. [2]
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, [7] [8] 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. [9] Trofinetide has also had a successful phase II trial against Fragile X syndrome. [10] [11] [12] The drug is manufactured by Acadia Pharmaceuticals.[ citation needed]
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. [2] Participants were randomized to receive trofinetide (N=93) or matching placebo (N=94) for 12 weeks. [2] The dose of trofinetide was based on participant weight to achieve similar exposure in all participants. [2]
The FDA granted the application for trofinetide priority review, orphan drug, and fast track designations. [2]
Clinical data | |
---|---|
Trade names | Daybue |
Other names | NNZ-2566 |
AHFS/ Drugs.com | Monograph |
MedlinePlus | a623019 |
License data |
|
Routes of administration | By mouth, feeding tube (gastrostomy tube) |
ATC code |
|
Legal status | |
Legal status | |
Pharmacokinetic data | |
Bioavailability | 84% |
Metabolism | Insignificant |
Elimination half-life | ~ 1.5 h |
Excretion | Urine |
Identifiers | |
| |
CAS Number | |
PubChem CID | |
DrugBank | |
ChemSpider | |
UNII | |
KEGG | |
ChEBI | |
ChEMBL | |
Chemical and physical data | |
Formula | C13H21N3O6 |
Molar mass | 315.326 g·mol−1 |
3D model ( JSmol) | |
| |
|
Trofinetide, sold under the brand name Daybue, is a medication used for the treatment of Rett syndrome. [1] It is taken by mouth. [1]
The most common adverse reactions include diarrhea and vomiting. [2]
Trofinetide was approved for medical use in the United States in March 2023. [1] [2] [3] [4] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [5]
Trofinetide is indicated for the treatment of Rett syndrome in people two years of age and older. [1] [6]
Rett syndrome is a rare, genetic neurological and developmental disorder that affects the way the brain develops. [2] People with Rett syndrome experience a progressive loss of motor skills and language. [2] Most babies with Rett syndrome seem to develop as expected for the first six months of life. [2] 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. [2] The hallmark of Rett syndrome is near constant repetitive hand movements, such as rubbing or clapping. [2] Rett syndrome leads to severe impairments affecting nearly every aspect of life, including the ability to speak, walk, eat, and breathe. [2]
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, [7] [8] 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. [9] Trofinetide has also had a successful phase II trial against Fragile X syndrome. [10] [11] [12] The drug is manufactured by Acadia Pharmaceuticals.[ citation needed]
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. [2] Participants were randomized to receive trofinetide (N=93) or matching placebo (N=94) for 12 weeks. [2] The dose of trofinetide was based on participant weight to achieve similar exposure in all participants. [2]
The FDA granted the application for trofinetide priority review, orphan drug, and fast track designations. [2]