European regulators unlikely to recommend approval of Daybue
Developer already planning to ask for reexamination of application
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Acadia Pharmaceuticals has hit a roadblock in its quest for European approval of trofinetide, the company’s oral therapy for Rett syndrome, which is already approved in the U.S., Canada, and Israel under the name Daybue.
Acadia submitted an application seeking approval of trofinetide in the European Union (EU) last year. Applications for new drugs in the EU are reviewed by the Committee for Medicinal Products for Human Use (CHMP), a branch of the European Medicines Agency (EMA) tasked with recommending for or against approval. Drug approvals in the EU are ultimately up to the European Commission, which almost always follows the CHMP’s recommendations.
Acadia has now announced that it has been orally informed of a negative trend vote on the trofinetide application, essentially meaning the CHMP is likely to recommend against approving the therapy.
“While the negative trend vote is disappointing and not what we hoped for, we believe the strong data that supported the approval of trofinetide for the treatment of Rett syndrome in the United States, Canada, and Israel speak to the meaningful benefits that trofinetide can deliver,” said Catherine Owen Adams, CEO of Acadia, in a company press release.
Once the CHMP’s final decision is announced, Acadia said it intends to ask the committee to reexamine the application. Under EU law, drug developers can submit such applications within 15 days of receiving the CHMP’s formal decision. Acadia would then have about two months to submit its reason for requesting reexamination, and the CHMP would have about two months after that to reexamine its decision.
“We look forward to working with the EMA and other stakeholders to advance trofinetide as an important potential treatment option in the EU,” Adams said. “Our commitment to the Rett syndrome community in the EU remains steadfast, and we are fully dedicated to making trofinetide available to individuals and families who urgently need a new therapeutic option.”
Daybue aims to help reduce brain inflammation
Rett syndrome is a genetic disorder characterized by developmental abnormalities due to problems in the nervous system. Trofinetide is thought to improve the health of synapses, the connections between nerve cells, as well as help reduce brain inflammation.
In 2023, the therapy became the first Rett treatment approved in the U.S. The following year, it was approved in Canada, and just a few weeks ago, Acadia announced its approval in Israel. In all of those countries, trofinetide (sold as Daybue) is indicated for Rett patients ages 2 and older.
These approvals were based on data from several clinical trials. A Phase 3 study called LAVENDER (NCT04181723) tested trofinetide against a placebo in girls with Rett ages 5 to 20, with results showing the therapy outperformed the placebo at improving measures of Rett symptom severity and overall health. Patients from this trial were then followed in two extension trials called LILAC-1 (NCT04279314) and LILAC-2 (NCT04776746), with data showing continued improvements in these measures.
A separate study, DAFFODIL (NCT04988867), tested trofinetide in girls as young as 2, with results showing that the therapy improved measures of overall health and quality of life. Across these clinical trials, the most common side effects of the therapy were vomiting and diarrhea.
Data from a real-world study of trofinetide’s use in the U.S. have shown results that are generally consistent with those of these clinical trials.
“We now have more than 1,000 patients on active treatment globally, from newly diagnosed 2-year-olds to adults who have lived with their disease for decades. Our ongoing real-world experience study in the U.S. continues to show outcomes that closely mirror the impact observed in rigorous randomized clinical trials conducted across a broad age range,” Adams said.
