Neuren Moves to Challenge EU Setback for Rett Syndrome Drug
Neuren Pharmaceuticals Limited (ASX: NEU) has confirmed that its partner, Acadia Pharmaceuticals, will pursue a formal re-examination of the European regulator’s decision to reject marketing approval for trofinetide in Rett syndrome patients aged two years and above.
While trofinetide is already authorised in the United States, Canada and Israel as the only approved therapy for Rett syndrome, the European review committee concluded that the demonstrated treatment benefit after 12 weeks was modest. Additional concerns were raised regarding the range of symptoms assessed in the pivotal trial and the interpretation of longer-term results, which were affected by patient discontinuations.
Despite the negative recommendation, both companies continue to express confidence in the therapy’s clinical relevance. They highlight meaningful feedback from patients, caregivers and clinicians, reinforcing their view that trofinetide provides tangible benefits. Neuren emphasised that a significant unmet medical need persists in Europe and stated its commitment to working closely with regulators to seek reconsideration.
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Neuren Moves to Challenge EU Setback for Rett Syndrome Drug
Neuren Pharmaceuticals Limited (ASX: NEU) has confirmed that its partner, Acadia Pharmaceuticals, will pursue a formal re-examination of the European regulator’s decision to reject marketing approval for trofinetide in Rett syndrome patients aged two years and above.
While trofinetide is already authorised in the United States, Canada and Israel as the only approved therapy for Rett syndrome, the European review committee concluded that the demonstrated treatment benefit after 12 weeks was modest. Additional concerns were raised regarding the range of symptoms assessed in the pivotal trial and the interpretation of longer-term results, which were affected by patient discontinuations.
Despite the negative recommendation, both companies continue to express confidence in the therapy’s clinical relevance. They highlight meaningful feedback from patients, caregivers and clinicians, reinforcing their view that trofinetide provides tangible benefits. Neuren emphasised that a significant unmet medical need persists in Europe and stated its commitment to working closely with regulators to seek reconsideration.
Customer Notice:
Nextgen Global Services Pty Ltd trading as Kapitales Research (ABN 89 652 632 561) is a Corporate Authorised Representative (CAR No. 1293674) of Enva Australia Pty Ltd (AFSL 424494). The information contained in this website is general information only. Any advice is general advice only. No consideration has been given or will be given to the individual investment objectives, financial situation or needs of any particular person. The decision to invest or trade and the method selected is a personal decision and involves an inherent level of risk, and you must undertake your own investigations and obtain your own advice regarding the suitability of this product for your circumstances. Please be aware that all trading activity is subject to both profit & loss and may not be suitable for you. The past performance of this product is not and should not be taken as an indication of future performance.
Kapitales Research, Level 13, Suite 1A, 465 Victoria Ave, Chatswood, NSW 2067, Australia | 1800 005 780 | info@kapitales.com.au