Newron announce Rare Disease Paediatric Designation from the FDA


Newron, the pharmaceutical company sponsoring the clinical trial of Sarizotan for breathing irregularities in Rett, recently announced that they have received Rare Disease Paediatric Designation from the FDA.

This is important because this designation gives the company incentives to advance the development of Sarizotan and if approved, the company may be eligible for a priority review for any further marketing applications for the drug, for example, in different age groups etc.

Reverse Rett have been instrumental in the success of the UK arm of the trial at King’s College Hospital. The UK trial site was the number one recruiting site worldwide, largely because of the unique collaboration between Reverse Rett, the trial site and our partners at Newron.

Since November 2016, Reverse Rett have provided ongoing funding for the team’s Clinical Trial Co-ordinator, pre-screening and referral of patients and management of the logistics of patient/family accommodation since April 2017 to the present day.

On Friday 22nd November, Dr Munaf Ali from Newron met with Professor Santosh and Rachael Stevenson to discuss about the extended work we are doing to support Professor Santosh’s work at KCH/KCL and further opportunities for collaboration. Emily Marsden who is mum to 5-year-old, Daisy was also present to give a parents perspective.

We very much look forward to working even closer with Newron in the future and strengthening our relationship.

From L to R – Mum to Daisy, Emily Marsden, Clinical Trial Lead at KCL, Professor Santosh, Reverse Rett ED, Rachael Stevenson, Newron Regulatory Advisor, Dr Munaf Ali