A major milestone for Australian company Neuren Pharmaceuticals with its US partner Acadia submitting trofinetide to the FDA for the treatment of Rett syndrome.
The submission is a step in a commercialisation process that could ultimately deliver milestone payments of over half a billion dollars.
Rett syndrome is a rare genetic disorder that impacts brain development and primarily affects females. It causes a progressive loss of motor skills and language. There are currently no approved treatments for the disorder.
Neuren retains all rights to trofinetide outside the US and says it is in discussions regarding additional commercial partnerships.
Late last year, the company announced positive results from a phase 3 trial of trofinetide in adult and paediatric patients with Rett Syndrome.
Neuren said that approval in the US would generate revenue in excess of $100 million during 2022 and 2023, including a combination of payments related to milestones and royalties.