Rett Syndrome, a rare genetic disorder is responsible for affecting brain development, particularly in the areas that control cognitive, sensory, and motor functions. The disease is found in girls and is caused by the mutation in the MECP2 gene of the X chromosome in females.
The disease manifests via significant developmental delays for girls as early as 6 to 18 months of age. It includes problems in speech and language, motor skills such as repetitive hand movements, difficulty in coordination and balance, and seizures. Current treatment available includes only physical and occupational therapy, medication for seizures and other symptoms, and specialized education and behavioral therapy.
But parents with children who have Rett Syndrome gets new hope when the U.S. Food and Drug Administration (FDA) approved on the 3rd week of March this year, the first-ever treatment for Rett Syndrome called “Trofinetide”. The drug is a synthetic analog insulin-like growth factor 1 (IGF-1) peptide, commercially named Daybue. Despite Rett commonly affecting females, the drug also indicates treatment for both male and female patients aged 2 and up.
Source: Acadia PharmaceuticalsThe drug was developed way back in the early 2000s but has completed a phase 3 trial prior to product licensing under Acadia Pharmaceuticals in 2018. In the study, Trofinetide was found to impact statistically significant improvements in patients with Rett, as measured by Rett Syndrome Behavior Questionnaire and the Clinical Global Impression Scale-Improvement.
The current challenge of the drug, however, is on the gastrointestinal side effects observed during the trial as 85 percent of people taking Daybue during the phase 3 trial experience diarrhea, although it was mild to moderate accounting for 96 percent of the said cases. FDA has made clear indications for labels in Daybue to provide advice on diarrhea management, including discontinuing drug use for constipation prior to starting Daybue and taking anti-diarrheal medication if needed.
Acadia announced that the drug will be available for commercial sale by the end of April 2023. The drug is anticipated to be on the market at a price of $21.10 per milliliter, with a dose designed according to the person’s weight and given twice a day.
Kathie Bishop, Acadia’s senior vice president, believed that Daybue is just the beginning of Rett treatments. Daybue’s FDA approval will likely accelerate drug development for the condition and will show others what’s possible.
Other companies and communities as per the International Rett Syndrome Foundation as gearing toward future developments in the treatment of Rett including an ongoing phase 3 trial for new drug development, and plans for gene therapy, and gene editing trials this year.
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