EU Panel Recommends Approval of Neuren’s Daybue
Synopsis
Neuren Pharmaceuticals has moved closer to European approval for its Rett syndrome treatment Daybue after receiving a positive recommendation from the EU's medicines regulator, paving the way for potential milestone payments and broader market access.
Neuren Pharmaceuticals Inc is poised to gain European approval for its rare neurological disease drug for Rett syndrome following a positive opinion from European regulators.
The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) has recommended Daybue (trofinetide) for marketing authorisation in the EU. The treatment is approved to treat the genetic disorder in adults and children aged two years and older.
The application will now go to the European Commission, which gives marketing authorisation across 27 member countries, with a final decision usually expected about two months after a positive CHMP opinion.
Rare disease market broadens
If approved in Europe, Daybue will be the first European treatment option for Rett syndrome. In April the treatment gained marketing approval in Canada as its second region behind the US. It has already won approval for the treatment for Rett syndrome in the United States and has submissions under review in Japan by licensing partner Acadia Pharmaceuticals.
Broadening rare disease market Rett syndrome is one of a range of rare and debilitating neurological conditions caused predominantly by mutations in the MECP2 gene that primarily affect girls.
The syndrome, occurring at roughly one per 10,000-15,000 live female births, is defined by developmental regression.
Roughly between 9,000 and 12,000 people in Europe may live with Rett syndrome, compared with 6,000-9,000 in the US and 1,000-2,000 in Japan, according to the company’s annual report. Neuren sees Europe as its second largest market after the US. European approval of Daybue opens Neuren up beyond its North American market by creating another regulatory centre.
Under Neuren’s licence arrangement, Acadia Pharmaceuticals will sell Daybue across North America and oversee Europe and Japan approvals, paying Neuren milestone and royalty payments, plus royalties on its manufacturing activities.
Financial position and next steps
Financial situation and prospects Neuren reported $318.3 million (US) revenue and $179.3million net profit after tax in the 2025 fiscal year due to Daybue's royalty payments, manufacturing income and milestone payments. Daybue achieved $391 million in net sales in North America in 2025.
Neuren CEO Jon Pilcher said the positive opinion takes Daybue a significant step closer to being prescribed to eligible European patients.
He highlighted the “strong data that led to this favourable recommendation.” If Daybue receives marketing authorisation in Europe, the company will secure another regulated market alongside the US and Canada and could become eligible for further milestone payments.
Source: Capital Brief
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Pooja Malik is a business journalist with over six years of experience covering startups, entrepreneurship, and emerging trends. She has previously worked with leading media platforms such as YourStory Media and BW BusinessWorld, where she reported on business, policy, and market developments. Currently, she serves as Editor at The Inspirepreneur Magazine, where she writes and edits stories across business, lifestyle, and travel, with a focus on clarity, accuracy, and reader relevance.
