Neuren Pharmaceuticals Ltd (ASX: NEU) shares are having a tough time on Tuesday.
In morning trade, the ASX 200 healthcare share was down as much as 22% to $12.66.
The pharmaceuticals company's shares have recovered a touch since then but remain down 12% at the time of writing.
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Why is this ASX 200 healthcare share crashing?
Investors have been hitting the sell button today after it was dealt a major blow in Europe.
This morning, Neuren Pharmaceuticals advised that its licensee, Acadia Pharmaceuticals Inc. (NASDAQ: ACAD), has provided an update on its marketing authorisation application (MAA) for trofinetide for the treatment of Rett syndrome in the European Union (EU).
According to the release, Acadia was informed by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) of a negative trend vote on its MAA for trofinetide, following its recent CHMP oral explanation.
Subject to the outcome of the CHMP vote in February, Acadia intends to request a re-examination of the opinion by the CHMP upon its formal adoption.
Acadia's CEO, Catherine Owen Adams, was disappointed with the news, but remains hopeful that approval will be achieved in time. She said:
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. 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. We look forward to working with the EMA and other stakeholders to advance trofinetide as an important potential treatment option in the EU. 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.
What's next?
Neuren notes that pursuant to EU legislation, an applicant has the right to request a re-examination of a CHMP opinion within 15 calendar days of receipt of the opinion. This is followed by submission of the grounds for the request for re-examination within 60 calendar days of receipt of the opinion.
The CHMP then has up to 60 days after receipt of these grounds to re-examine its opinion.
Neuren's CEO, Jon Pilcher, commented:
Given the totality of experience with trofinetide in clinical trials and real world use over many years, this negative trend vote is frustrating for us and the Rett syndrome community in the EU. We fully support Acadia's intention to seek re-examination of the CHMP opinion in February, if necessary.
