Neuren Pharmaceuticals (ASX:NEU) shares plummeted as much as 22% on Feb. 3 morning, following a major regulatory hurdle for its flagship Rett syndrome treatment, trofinetide.
The company's North American licensee, Acadia Pharmaceuticals (NASDAQ:ACAD), announced it received a "negative trend vote" from the European Medicines Agency’s Committee for Medicinal Products for Human Use.
The preliminary setback, occurring during an oral explanation session in late January, casts doubt on the drug's immediate approval in the European Union.
Despite the unfavorable trend, Acadia CEO Catherine Owen Adams emphasised the therapy's established success elsewhere.
"The strong data that supported approval in the US, Canada, and Israel speak to the meaningful benefits trofinetide delivers," Adams stated, noting that over 1,000 patients are currently on active treatment globally.
Neuren CEO Jon Pilcher labeled the vote "frustrating" for the European Rett syndrome community, which currently lacks an approved therapeutic option.
Under EU protocols, Acadia intends to formally request a re-examination once the CHMP adopts its final opinion later this February.