Dear GP2C Community,

Encouraging news from the Rett Syndrome research world!

Many of our GP2C families have been connected to the Acadia Trofinetide trials over the last few years. The announcement below informs that the U.S. FDA has agreed to a “priority” review of Acadia’s New Drug Application (NDA)! This is fantastic news for all Rett families who have waited patiently (for many years) for a potential therapeutic treatment for their children. We at GP2C and Rett University are encouraged and hopeful that this review is quick and successful!

From the article (Acadia Pharmaceuticals Announces Trofinetide New Drug Application for the Treatment of Rett Syndrome has been Accepted for Filing and Review by U.S. FDA):

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) of trofinetide for the treatment of Rett syndrome. The FDA has granted a priority review and assigned a PDUFA (Prescription Drug User Fee Act) action date of March 12, 2023… “We’re pleased that the FDA has accepted our NDA filing and we will be working closely with them to facilitate completion of the review in a timely manner,” said Steve Davis, Acadia’s Chief Executive Officer. “If approved, trofinetide will be the first drug available for the treatment of Rett syndrome, a rare and devastating condition for patients and their families…”

See the full announcement here:

Regards, Kevin