Acadia Pharmaceuticals awaits FDA decision for first-of-its-kind Rett syndrome drug

pharmafile | March 10, 2023 | News story | Research and Development  

Acadia Pharmaceuticals is aiming to follow in the footsteps of Reata Pharmaceuticals, who won approval for its novel drug to treat ultra-rare neurological diease Friedreich’s ataxia, with its own rare disease drug. The FDA has given a date of 12 March to announce its decision on trofinetide.


Trofinetide is a novel synthetic analogue of the amino-terminal tripeptide domain of the IGF-1 protein. It is used to treat Rett syndrome, a multisystem neurodevelopmental disorder which leads to developmental regression, loss of language and hand function and impaired gait. The syndrome mainly affects female children who develop normally until the age of two, when normal developmental milestones begin to be missed.


While Rett syndrome affects neurons throughout the brain, it is not neurodegenerative, meaning trofinetide could potentially restore connections at any age.


Trofinetide was assessed in the phase 3, 12-week Lavender study which compared the drug against placebo in 187 girls and young women aged between five and twenty. The co-primary endpoints were a change in Rett Syndrome Behavior Questionnaire scores from a baseline, and Clinical Global Impression-Improvement scale score. Both were significantly improved after treatment with trofinetide.


Kathie Bishop, PhD, SVP and head of rare disease and external innovation at Acadia, said: “What happens [with Rett syndrome] is that the connections between the brain cells are lost, so you still have the neurons there, but they can’t communicate with each other…On the clinical side, with the advances in genetic testing, patients are being more readily identified and studied. Then on the science side, I think [there are] a lot of great discoveries and advancements. I think this is just the beginning of a lot more to come in neurologic rare diseases, especially genetic diseases.”


James Spargo

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