Powder form of Rett treatment Daybue now widely available in US
Limited launch showed families liked flexibility, portability of Stix version
Written by |
Daybue Stix, a powder formulation of Daybue (trofinetide), is now widely available for eligible people with Rett syndrome in the U.S., according to Acadia Pharmaceuticals, which markets both therapies.
“The availability of DAYBUE STIX gives us an additional, flexible way to administer trofinetide, which allows us more options to address unique patient and caregiver needs,” Arthur Beisang, MD, a pediatrician at Gillette Children’s Specialty Healthcare in St. Paul, Minnesota, said in a press release from Acadia.
The original version of Daybue is available as a strawberry-flavored liquid. Daybue Stix is a dye- and preservative-free powder that can be dissolved in water or water-based beverages, allowing families to customize both the amount of liquid and its flavor.
“This patient-centered approach aligns with recently published expert consensus recommendations, which advocate for the integration of [Daybue] as part of the standard of care and comprehensive Rett syndrome management,” Beisang said.
Guidelines published last year recommended Daybue as part of the standard treatment for Rett syndrome. Acadia helped fund the development of those guidelines, and Beisang was a co-author.
Company notes positive feedback from limited launch
Beisang said that having Daybue Stix in addition to the original formulation “provides additional customization, supporting individualized care for people with Rett syndrome.”
Daybue Stix was approved by the U.S. Food and Drug Administration (FDA) late last year, and it has since been available to a limited number of patients.
“Initial feedback from a small group of caregivers following the limited launch revealed that more than 80% of early users reported satisfaction with DAYBUE STIX, highlighting the added flexibility and portability of this new formulation,” said Tom Garner, Acadia’s chief commercial officer. “We are hearing that the new formulation may allow for more customized care in real-world settings. Ongoing evaluation from patients and caregivers remains a priority; their perspectives are essential as we identify ways to better assist families managing this complex condition.”
Rett syndrome is a genetic disorder marked by abnormalities in nervous system development. Both Daybue and Daybue Stix contain trofinetide, a therapy that’s thought to help reduce inflammation in the brain and boost the health of connections between nerve cells. Both formulations are approved in the U.S. to treat Rett patients ages 2 and older.
Daybue was the first treatment for Rett syndrome to win FDA approval, and it remains the only approved therapy for the disease in the U.S. The FDA’s initial approval was based primarily on data from a Phase 3 clinical trial called LAVENDER (NCT04181723), which tested the therapy against a placebo in 187 Rett patients, ages 5 to 20. Results showed the therapy eased behavioral symptoms and improved communication abilities. Similar effects were reported in a separate study called DAFFODIL (NCT04988867) that tested Daybue in children with Rett as young as 2, and in a real-world study sponsored by Acadia.
The FDA’s decision to approve Daybue Stix was based on data from a pharmacological study showing that the new powder formulation and the original liquid formulation deliver an equivalent amount of trofinetide to the body.
European Union regulators recently recommended against approving trofinetide for Rett syndrome, saying available evidence isn’t sufficient to conclude the therapy is effective. Acadia has announced plans to challenge that recommendation.
