TROFINETIDE BECOMES THE FIRST LICENSED DRUG FOR RETT SYNDROME IN THE US

Here are some key facts which have been published by the US-based Rett Syndrome Research Trust about Daybue.

The following information is based on RSRT’s interpretation of thepublicly available information includingresults of theLavenderTMtrial, based on the officialprescribing information for DAYBUE. Key Summary Facts of LavenderTM and LILAC-1 Trials:

Trofinetide has become the first drug treatment to be approved by the US Food and Drug Administration FDA for patients with Rett syndrome, age 2 and over. The drugs new license name is Daybue.

This is a significant moment for everyone in the Rett community because until now there has been no approved treatment anywhere in the world for Rett

FDA approval means that from April 2023, Daybue will be available for doctors to prescribe in the USA.

The company which developed Daybue is called Neuren. Neuren have not sold the marketing rights for Trofinetide within the EU or UK.

Whilst it is positive the FDA have recognised the burden of the lack of approved treatments for children and adults with Rett syndrome, it is important for patient organisations like Reverse Rett to be clear about our understanding of a given treatment and the potential for improvement for the patients who have access to it.

61% of patients taking DAYBUE did not improve.

13% of patients were rated as “much improved”.

No data is provided regarding which specific symptomsimprove

85% of patients treated with DAYBUE had diarrhea and 29% had vomiting.

In the study where everyone received DAYBUE, 46% of patients withdrew before completing the study

Concluding thoughts from RSRT

It’s important that every family considering DAYBUE as a treatment for their loved one make their ownrisk: benefit assessment with theirphysician DAYBUE is not a cure

The data from the trial show that there will likely be no change for the majority of patients. A subset of patients may improve. The specifics on what symptoms improvehave not beendisclosed

Read the full article on the RSRT website