Acadia closer to bringing first Rett syndrome drug to market

Acadia Pharma’s trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating neurological disorder Rett syndrome, although safety has dampened enthusiasm a little.

Top-line data from the LAVENDER study in 187 girls and young women with Rett have shown that trofinetide had a significant effect on two primary efficacy measures at 12 weeks – raising the hope that it may become the first-ever approved therapy for the disorder.