Neuren announces important progress in the US and Europe for trofinetide in Rett syndrome.

July 29, 2015

Neuren held a productive meeting with US Food and Drug Administration (FDA) about the drug trofinetide in treatment for Rett syndrome.  Neuren is committed to a pivotal trial intended to provide evidence for a drug marketing approval and to support a New Drug Application for Rett Syndrome.  Neuren will propose a trial design to the FDA for a single Phase 3 clinical trial that includes a primary efficacy endpoint identified by Neuren and the FDA at this past meeting.  Prior to the Phase 3 trial, there will be a brief tolerability clinical trial in children and adolescents to test higher doses of trofinetide in a younger population and confirm dose levels for the Phase 3 trial.

What does this mean for families?

1.  A small phase 2 trial in the pediatric population will be conducted to look for the best dose of trofinetide that is safe and tolerable.
2.  If the phase 2 is successful, then they will move to a “pivotal” Phase 3 trial that will be conducted by Neuren Pharmaceuticals.  A pivotal trial is one that is usually a larger and longer study, where the data will be used as evidence for drug marketing approval from the FDA.
3.  If the phase 3 is successful, this pivotal trial will form the basis for a new drug application by Neuren Pharmaceuticals for Rett syndrome.

In Europe, Orphan drug designation for trofinetide in both Rett syndrome and Fragile X Syndrome was recommended by the European Medicines Agency. is excited that Neuren is deeply committed to bringing trofinitide into a pivotal trial for Rett syndrome.

Read the full press release.