European regulators are backing approval of Acadia Pharmaceuticals’ Daybue, following a re-examination of the data the drugmaker submitted after the agency initially voted against authorizing the medicine for a rare brain disorder.
The European Medicines Agency’s human drugs committee, called the CHMP, voted in February against a marketing authorization for Daybue in Rett syndrome, triggering Acadia’s formal request for a re-examination by a different set of reviewers. The new positive recommendation now goes to the European Commission for final approval, which is due within 67 days.
Known scientifically as trofinetide, the drug will be sold under the brand name “Daybu” in the 27 countries of the European Union. The Food and Drug Administration first approved the therapy for pediatric patients and adults with Rett syndrome, a genetic disorder that affects brain development and coordination, in 2023.
“The CHMP’s positive opinion for Daybu is an important milestone in our mission to bring this innovative therapy to the EU, where there are no therapies specifically approved for the neurobehavioral symptoms of this devastating condition," said Acadia CEO Catherine Owen Adams, in a statement.
In a client note, Mizuho analyst Uy Ear wrote that Acadia’s 2028 sales guidance of $700 million includes a “modest amount” of European revenue, suggesting the Wall Street consensus forecast of $595 million is too low. Acadia’s shares rose as much as 12% in morning trading.
The CHMP committee also voted to revoke its approval of Amgen’s Tavneos in two rare inflammatory conditions affecting the blood vessels due to the risk of serious side effects. Amgen’s commercial partner, Kissei, has reported that 20 people have died after receiving the treatment in Japan, most from a condition called “vanishing bile duct syndrome.”
In the U.S., the FDA has asked Amgen to withdraw Tavneos from the market, alleging the company that developed it, ChemoCentryx, manipulated trial data. Amgen, however, declined. Stepping further, the company earlier this month highlighted real-world data it said supports Tavneos’ use.
The committee additionally voted to expand use of Eli Lilly’s BTK inhibitor Jaypirca across all lines of chronic lymphocytic leukemia. The vote was based on data the company highlighted at the American Society of Hematology meeting in December.
It also voted to expand use of other drugs. Those included AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Datroway to first-line triple-negative breast cancer in people who don't qualify for immunotherapy; Johnson & Johnson’s bispecific antibody Tecvayli for second-line multiple myeloma use; and Incyte’s Opzelura for atopic dermatitis, a major expansion from its current use in vitiligo.
