A major win for Australian company Neuren Pharmaceuticals (ASX:NEU) with the US FDA approving its treatment for Rett syndrome.
The approval has been granted to Neuren's partner, Acadia Pharmaceuticals, which holds commercial rights to DAYBUE (trofinetide) in the US, Canada and Mexico.
The FDA has approved DAYBUE for the treatment of Rett syndrome in adult and paediatric patients two years of age and older. It is the first and only approved treatment for Rett syndrome.
Rett syndrome is a rare genetic neurological and developmental disorder that affects brain development. It primarily affects females and causes a progressive loss of motor skills and language.
In a statement, Acadia said it expects DAYBUE to be available by the end of April.
The US company gained North American rights to DAYBUE under an agreement with Neuren in 2018. Neuren retains all rights to DAYBUE outside North America and has a licence for all data generated by Acadia.
Neuren CEO Jon Pilcher said, “Many people have shown great determination over the long journey to reach this historic outcome. The greatest has been shown by the Rett syndrome community and I am delighted for them. 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.”
Neuren will receive a milestone payment of US$40 million following the first commercial sale of DAYBUE in the US. It is eligible to receive ongoing royalties, milestone payments of up to US$350 million and one-third of the market value of the Rare Pediatric Disease Priority Review Voucher that was awarded to Acadia by the FDA. The one-third share is estimated by Neuren to be US$33 million.