ACADIA has shared the exciting news that the US Food and Drug Administration (FDA) has reviewed the documents received as part of the New Drug Application (NDA) for trofinetide, and they have determined that the application is complete and can be filed. They also determined that trofinetide qualifies for Priority Review. The Prescription Drug User Fee Act (PDUFA) dictates that from the date of the NDA filing, the FDA has 6 months to review the NDA and share the result with ACADIA. The date of the decision is called the PDUFA action date and the PDUFA date is March 12, 2023.
- Trofinetide is not currently approved by the FDA.
- The U.S. Food & Drug Administration (FDA) acknowledged receipt of a New Drug Application (NDA) for Trofinetide on Sept. 12, 2022.
- Now the FDA will review the NDA for Trofinetide.
- The FDA will make its decision on March 12, 2023, as to whether Trofinetide will be an approved drug for Rett syndrome.
- If Trofinetide receives FDA approval in 2023, additional work will be needed before it becomes commercially available by prescription (manufacturing, insurance coverage determinations).
We know that this sparks a lot of excitement within our community. Together we’re making history! No potential treatment for Rett syndrome has ever made it this far. We’re eager to hear the updates as well, but we know the FDA will not provide any information until the PDUFA date. The hardest part now is the waiting.
In the meantime, IRSF is relentlessly pursuing every possible solution for those affected by Rett while empowering families today. We will continue to bring you helpful information about this and all clinical trial news as it becomes available.