Key Takeaways:
Acadia Pharmaceuticals Inc. said the European Medicines Agency's Committee for Medicinal Products for Human Use adopted a positive opinion recommending marketing authorization for DAYBU (trofinetide) to treat neurobehavioral symptoms of Rett syndrome in patients aged five years and older, reversing a prior negative trend vote.
"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," Catherine Owen Adams, Acadia's chief executive officer, said in a statement.
The recommendation follows a re-examination procedure and is based primarily on results from the Phase 3 LAVENDER study, which showed statistically significant and clinically meaningful improvements in core features of Rett syndrome as measured by the Rett Syndrome Behaviour Questionnaire and the Clinical Global Impression-Improvement scale. The CHMP had previously signaled a negative trend vote in February, sending shares lower before Friday's reversal.
If granted marketing authorization by the European Commission, which is expected to issue a final decision in the coming months, DAYBU would become the first therapy approved for Rett syndrome in the European Union, covering all 27 member states plus Iceland, Liechtenstein and Norway. Rett syndrome is a rare neurodevelopmental disorder that occurs in approximately one of every 10,000 to 15,000 female births worldwide, with no approved treatments in Europe for its neurobehavioral symptoms.
Shares of Acadia rose 12.2% to trade near their 52-week high, recovering from weakness suffered earlier this year after the initial negative CHMP signal. The company, which has a market capitalization of less than $5 billion, reported first-quarter Daybue revenue of $101 million and Nuplazid revenue of $167 million. The EU approval would open a new geographic market for Acadia's key drug asset, which already has approvals in the U.S., Canada and Israel. Investors will watch for the European Commission's final decision in the coming months as the next catalyst for the stock.
This article is for informational purposes only and does not constitute investment advice.
