Daybu wins approval in EU as region’s first Rett syndrome treatment
Liquid med eased behavioral and communication symptoms in clinical trial
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The European Commission has approved Acadia Pharmaceuticals’ Daybu (trofinetide) — sold elsewhere as Daybue — as the first treatment authorized in the European Union for disease-related behavioral, emotional, and communication issues in people with Rett syndrome, ages 5 and older.
The oral therapy, taken by mouth or via a feeding tube, was shown in clinical trials to reduce the severity of neurobehavioral issues seen with Rett. Children in one study saw improvements in core symptoms, such as being able to respond to a choice when asked by parents, or experiencing more freedom from repetitive hand movements.
The decision by the EU’s executive body follows a recent positive opinion issued by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). That recommendation for the drug’s approval was based on a reexamination of data.
The new approval allows Daybu to be marketed across all 27 EU member states, as well as Iceland, Liechtenstein, and Norway. Acadia will now work with national authorities on pricing and reimbursement agreements. The therapy, given as a liquid, is already approved in the U.S., Canada, and Israel under the Daybue name. A powder formulation, called Daybue Stix, is also available in the U.S.
“The approval of DAYBU marks a significant milestone for the Rett syndrome community in the EU and advances our mission to bring this innovative treatment to patients and families who have long faced a profound unmet medical need,” Catherine Owen Adams, Acadia’s CEO, said in a company press release.
A rare genetic disorder, Rett is chiefly caused by mutations in the MECP2 gene that result in low levels of a protein of the same name. MECP2 is important for nerve cell communication, as well as brain development and function. Rett is marked by symptoms that include loss of motor skills as well as cognitive and behavioral issues and difficulty communicating.
Daybu’s active ingredient, trofinetide, is a lab-made version of insulin-like growth factor-1, a signaling molecule involved in brain development. Trofinetide is thought to help support nerve cell function and communication, potentially easing the condition’s neurobehavioral symptoms.
Daybu shown in LAVENDER trial to ease Rett symptoms
The treatment’s approval was based on results from a Phase 3 clinical trial, called LAVENDER (NCT04181723), that enrolled 187 girls and young women with Rett. The participants, who ranged in age from 5 to 20, were given either Daybu or a placebo for 12 weeks, or about three months. The dose of the therapy was based on the participants’ body weight.
The results showed that participants given Daybu experienced significant reductions in neurobehavioral symptoms compared with those receiving the placebo. The treated patients also saw improvements in communication skills. Further, the therapy was generally well tolerated.
Participants who completed LAVENDER could continue to receive Daybu in the open-label extension studies LILAC-1 (NCT04279314) and LILAC-2 (NCT04776746), which spanned nearly four years. Long-term results from these studies indicated continual benefits.
In addition, results from a Phase 2/3 study called DAFFODIL (NCT04988867) — which tested Daybu in 15 girls, ages 2 to 4, with Rett — further showed benefits in overall health and quality of life.
[This approval] provides clinicians with a new treatment option to help address the neurobehavioral symptoms of Rett syndrome and represents progress for the Rett community.
Nadia Bahi, MD, PhD, a pediatric neurologist at Necker-Enfants Malades University Hospital in France, said Daybu’s EU approval “is an important milestone that recognizes the significant unmet medical need in this rare disease.”
According to Bahi, “it provides clinicians with a new treatment option to help address the neurobehavioral symptoms of Rett syndrome and represents progress for the Rett community.”
The CHMP had initially recommended against approving Daybu when Acadia first sought regulatory clearance in the EU, saying that available clinical data did not sufficiently demonstrate the treatment’s benefits. The company then requested a reexamination, which resulted in the positive opinion.
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