- The US Food and Drug Administration (FDA) approves Neuren Pharmaceuticals’ (NEU) North American partner’s trofinetide drug as a treatment for Rett syndrome
- Trofinetide, marketed under the brand name DAYBUE, is now the first drug approved for the treatment of the neurodevelopment disorder
- Neuren and Acadia Pharmaceuticals entered an exclusive license agreement in 2018 to develop and commercialise the drug in the US, Canada and Mexico
- Under the agreement, NEU received a milestone payment of US$10 million from Acadia in October last year, and a further $40 million will be paid upon the first commercial sale in the US
- Shares in Neuren are up 18.64 per cent to $9.10 at 10:48 am AEDT
Neuren Pharmaceuticals’ (NEU) North American partner, Acadia Pharmaceuticals, has had its treatment for Rett syndrome approved by the US Food and Drug Administration.
Trofinetide, to be sold under the brand name DAYBUE, is now the first drug to be approved for the treatment of the neurodevelopment disorder in adult and paediatric patients older than two.
Neuren CEO Jon Pilcher said many people had shown “great determination” over a long journey to reach this “historic” outcome.
“For Neuren, this is a transforming milestone that places us in a position to make the most of the opportunities ahead of us as we work with the communities to make a difference in four other neurodevelopmental disorders,” Mr Pilcher said.
Neuren and Acadia entered an exclusive license agreement in 2018 to develop and commercialise trofinetide for the treatment of Rett syndrome and other indications in the US, Canada and Mexico.
Neuren received a milestone payment of US$10 million (A$15 million) from Acadia in October 2022 after the new drug application was accepted for review by the FDA.
Now, NEU will receive a further US$40 million upon the first commercial sale of the drug in the US, which is anticipated by the end of April this year.
The company will also receive royalties on net sales, potential sales milestone payments and one-third of the market value of a Rare Pediatric Disease Priority Review Voucher awarded to Acadia.
Neuren retains all rights to trofinetide for all countries outside North America. The company said it would pursue registration and commercialisation of the drug through its partners and is currently advancing discussions with a number of third parties.
Shares in Neuren were up 18.64 per cent to $9.10 at 10:48 am AEDT.