Share Price, page-2747

Dr Jeffrey Neul has spoken about trofinetide..... summing up the current situation nicely and how we sit now with the FDA.
September 20, 2022.

NeurologyLive®: If approved, what immediate impact might trofinetide have on the treatment paradigm in Rett?

Jeffrey L. Neul, MD, PhD:

There are currently no FDA approved treatments for Rett syndrome, so this will provide an approved treatment that clinicians can readily incorporate into their treatment paradigm.

In that same vein, are there any challenges in care the possible approval of this agent may aid?
The results from the Lavender trial indicated that trofinetide had impact across a variety of areas of clinical concern, suggesting that approval and utilization might address a number of challenges people with Rett syndrome face.

Are there any particular findings from LAVENDER that you feel are important to point out?
Importantly, not only is the first phase 3 study that has meet primary endpoints in Rett syndrome, but it is also one of the first times a successful phase 3 trial has been completed in any neurodevelopmental disorder. There has been significant concern about the potential to develop therapies in these disorders because of many previous failed phase 2 trials, and this demonstrates that successful phase 3 trials are possible in neurodevelopmental disorders and provides hope that future successful phase 3 trials can be completed in these challenging disorders.

What has trofinetide’s safety profile revealed? Is there anything of which physicians should be aware?
Broadly, trofinetide was safe. Physicians should be aware of the increased frequency of diarrhea in people on trofinetide compared with placebo, which is unusual as people with Rett syndrome typically have severe constipation and are on medical treatments such as laxatives to address this problem. If trofinetide is approved by the FDA for use in Rett syndrome, adjustments to ongoing constipation treatments and monitoring for clinical issues related to diarrhea will need to be considered by physicians who prescribe this drug to people with Rett syndrome.