Santhera has submitted a marketing application to the EMA – the drug regulator in Europe – for Puldysa. Puldysa, which used to be called Raxone, is a potential treatment for breathing problems in patients with Duchenne who are not using steroids.
The EMA did not approve a previous submission from Santhera. The company believes the new submission includes more information with data from several clinical studies. These include the recent Phase II and Phase III studies as well as the long-term extension studies. In addition, the company included long-term data from patients who took Puldysa for up to six years.
Santhera also plans to submit a new drug application to the FDA – the US drug regulator. This will be submitted when the company completes a study testing the effectiveness of Puldysa in people living with Duchenne who are taking steroids.
We will keep you updated with this application. It’s important to note that marketing approval is not the last step in drug approval. In the UK, the cost-effectiveness of treatments must be assessed before drugs are made available to patients.
Find out more
- Raxone is available in the UK on an Early Access to Medicine Scheme
- Contact Neil, our Director of Research on 02072508240
