Soleno Theraputics the company behind DCCR, recently published positive results from their randomised withdrawal phase of an extended treatment of DCCR. In the study, 77 patients previously on DCCR treatment were randomised to receive either DCCR or a placebo for four months. The primary endpoint to evaluate hyperphagia-related behaviours significantly improved in the DCCR group. Secondary endpoints, including body composition and behaviours also favoured DCCR. No new safety concerns were identified during the study.
The data certainly looks encouraging and the recent FPWR presentation from Dr Miller confirmed this. As things stand, it is looking entirely possible that we will see the first treatment for PWS since the approval of Growth Hormone! This is obviously hugely exciting for our community. Our understanding is that Soleno Theraputics plan to make a submission to the American medical authorities, the Food and Drug Administration (FDA) in 2024 with a view to making the drug available in the USA from early 2025.
In terms of availability in the UK, our goal is to ensure that should the drug be licenced in the USA, we will have it here as soon as possible afterwards. FPWR UK are currently in discussions with PWSA with a view to establishing a joint approach for communicating with the authorities here in the UK on this matter.
It is hoped cooperation with PWSA will not only be useful for DCCR, but will also be important as we look towards other new treatments on the horizon which are currently in the trial phase. This includes the real potential for helping to facilitate drug trials in the UK.