Regulatory Setback Threatens Trofinetide’s European Launch for Rett Syndrome
Acadia Pharmaceuticals has received a negative vote from the European Medicines Agency’s committee on its application to market trofinetide for Rett syndrome in the EU, prompting plans for a re-examination request.
- Negative trend vote from EMA’s CHMP on trofinetide EU approval
- Acadia plans to request re-examination of the opinion
- Trofinetide already approved in US, Canada, and Israel
- Over 1,000 patients currently treated globally
- Neuren Pharmaceuticals supports Acadia’s ongoing efforts
Regulatory Hurdle in Europe
Neuren Pharmaceuticals’ licensee, Acadia Pharmaceuticals, has encountered a significant regulatory challenge in its pursuit to bring trofinetide to the European market for the treatment of Rett syndrome. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative trend vote following an oral explanation session, casting uncertainty over the drug’s approval in the European Union.
While this development is a setback, it is not the final word. Acadia intends to request a re-examination of the CHMP’s opinion, a process that allows the company to present further arguments and data in hopes of reversing the decision. This procedural step underscores the company’s commitment to securing approval despite the initial disappointment.
Global Context and Clinical Confidence
Trofinetide has already secured approval in the United States, Canada, and Israel, where it is marketed under the brand name DAYBUE®. More than 1,000 patients worldwide are currently receiving treatment, ranging from young children recently diagnosed to adults who have lived with Rett syndrome for decades. Acadia’s CEO, Catherine Owen Adams, emphasised the robust clinical data supporting trofinetide’s efficacy and the real-world outcomes that mirror those seen in controlled trials.
Neuren’s CEO Jon Pilcher expressed frustration at the negative vote but reaffirmed support for Acadia’s strategy. The company remains dedicated to advancing treatments for serious neurological disorders, including Rett syndrome, which currently has limited therapeutic options.
Looking Ahead
The re-examination process provides a window for Acadia to address the CHMP’s concerns and potentially secure a positive opinion. However, the timeline and ultimate outcome remain uncertain. Meanwhile, Neuren continues to develop other drug candidates, such as NNZ-2591, targeting rare neurodevelopmental disorders with orphan drug designations in both the US and EU.
This regulatory episode highlights the complexities of drug approval in different jurisdictions and the challenges faced by companies developing treatments for rare diseases. The Rett syndrome community in Europe will be watching closely as the process unfolds, hopeful for a resolution that expands access to this promising therapy.
Bottom Line?
The upcoming CHMP re-examination will be pivotal for trofinetide’s European future and Neuren’s growth trajectory.
Questions in the middle?
- What specific concerns led to the CHMP’s negative trend vote on trofinetide?
- How might a prolonged approval process impact patient access and company revenues?
- What additional data or arguments will Acadia present during the re-examination?
