This week’s Trofinetide announcement

Much excitement has been generated across the UK Rett community this week due to the announcement by the company, Acadia that they have submitted a New Drug Application to the American Food and Drug Administration (FDA), for their drug ‘Trofinetide’ for people with Rett Syndrome.  

 At Reverse Rett, we are receiving some questions about this announcement and wanted to share our perspectives.  

  Reverse Rett is a patient advocacy and research organisation and we work with companies and organisations internationally to further our mission to deliver treatment for Rett Syndrome to all affected. We are always delighted to see progress in the field.  

 We have children with Rett Syndrome too and we are all too aware that families are waiting and desperately hoping for treatment that will positively impact their loved ones’ lives.  

  We have a responsibility though, to make sure that when we share information about emerging treatments, we have all our questions answered and that we have confidence in the company sharing the information.  

 The most important part of that responsibility is to children, young people and adults with Rett Syndrome and their families. We never want to be raising the expectations of families without good reason. For us as a research organisation, good reason means data, or a positive approval or something else of substance.  

 As such, we want to share with you some key thoughts about this and other industry announcements. 

An announcement by a drug company is not in and of itself newsworthy. If the company is a public company, announcements are typically based on what works best for the financial interests of the company, not necessarily what is best for the patient community.  

This particular announcement by Acadia is the company announcing their submission to the FDA. It is not an approval by the FDA.  

The FDA is the American regulator.  

In the UK, our regulator is the Medicines and Healthcare products Regulatory Authority (MHRA). 

In Europe it is the European Medicines Agency (EMA). 

FDA submission or approval does not mean MHRA/EMA approval and vice versa. Each requires a separate application.  

The company which has marketing rights for Trofinetide in the USA is Acadia. At Reverse Rett, we have not received confirmation about which company has marketing rights in the UK/Europe, despite reaching out to various companies including Acadia and Neuren over the last 12 months.  

The following points add to our reservations about this application:  

• There have been some anecdotal reports from families in America sharing positive experiences of the Trofinetide Lavender study.  However, the data which have been shared by Acadia to date, only show very small differences between patients treated with Trofinetide over patients who received placebo. Also, a relatively high number of people (20%) left the trial; the most commonly cited side effect was diarrhoea. More evidence is needed before the community can arrive at a conclusion regarding Trofinetide and its potential for improving symptoms in people with Rett Syndrome.  

• On a broader level, we have spent the last 18 months learning as much as we can about the UK regulatory processes and Health Technology Assessments, so that we are prepared to work with regulators and payers when the time comes for new treatments for Rett to be assessed for funding. We know from this work, that it is going to be a complex and difficult task to get any treatment for Rett through in this economic climate, even though the patient population needs it so badly. Our early interactions with regulators have shown us that they much more strongly favour treatments which have clear cut positive effects, even if they are expensive. Especially here in the UK, where public funds are tight, and the NHS is under so much pressure. The regulators want to see they are getting value for money from new treatments.  

•  Any company seeking market authorisation in the UK for a drug for people with Rett Syndrome, will need the support of one or more patient organisations to bolster their application. At this stage, we are still unclear even about which company has the rights to market Trofinetide in the UK, despite reaching out for this information. 

  We think it is important, in the interests of transparency, of respecting patients and families and of managing our community’s expectations, that we share our reservations as freely as we share our hopes, and this is what we’ve done in this article.  

 Moving forward, Reverse Rett will work with Rett UK under the umbrella of the Rett Disorders Alliance, to present cohesive and consistent information to the UK Rett community about emerging treatments for Rett Syndrome. 

We hope for the opportunity to meet with the company with marketing rights to Trofinetide in the UK in the near future. We hope to see some convincing data. We hope the FDA finds substance enough in the application to approve the New Drug Application.  

We hope that the company then choose to apply in the UK and that there is substance enough in the application for Reverse Rett, Rett UK and the wider Rett Disorders Alliance to support. If there is enough evidence in the application that the drug can improve the symptoms of people with Rett Syndrome, we will work together to support and push for the payers (NICE, SMC etc.) to fund the treatment for all UK individuals with Rett Syndrome. 

Until then, on this and all other issues regarding emerging treatments for Rett Syndrome, we will always be clear about where we stand.