A community update about vamorolone in the United States

The drug companies Santhera Pharmaceuticals and ReveraGen BioPharma have announced their new drug application (NDA) for vamorolone for the treatment of Duchenne muscular dystrophy (DMD) has been accepted by the US Food and Drug Administration (FDA) for review.

The FDA has set 26 October 2023 as the date they must approve the NDA or issue a response letter. Subject to approval and delays, Santhera plans to launch vamorolone in the US in the last three months of 2023.

Background to the development of vamorolone 

Vamorolone is a steroid which promises fewer side effects than the current standard treatments for DMD (prednisolone or deflazacort).

In 2015, Duchenne UK, Joining Jack and Duchenne Research Fund invested £750,000 towards a phase 1 clinical trial in boys with DMD, which then enabled ReveraGen to win a £6 million grant form Europe’s Horizon 2020. Duchenne UK’s DMD Hub enabled swift recruitment to a phase 2 clinical trial in the UK.

The UK and EU

In a recent update, Santhera announced they have applied to regulators in different countries for market authorisation, including the EU and the UK.  

At the same time NICE, the regulator responsible for assessing the clinical and cost effectiveness of drugs in England, announced a health technology assessment (HTA) for vamorolone. HTAs assess whether a new treatment is clinically effective and cost effective for NHS patients. You can find NICE’s information on vamorolone here.  

This is an exciting development, as NICE start the HTA process before marketing authorisation is granted, with an aim to have finished evaluating a drug soon after market authorisation is achieved. The MHRA don’t publish scientific opinions on treatments until they’re approved, expired or withdrawn, nor give public updates on treatments going through the pipeline. But this development with NICE suggests vamorolone is progressing through the MHRA’s processes. 

We continue to work with Santhera and the UK’s regulators so vamorolone can be rigorously evaluated and, if it’s deemed suitable, made available to people with DMD in the UK. 

CEO Of Duchenne UK Emily Reuben said:

“This is another step towards a new treatment for the symptoms of Duchenne muscular dystrophy becoming available for patients.

“It is very exciting to see a treatment funded by Duchenne UK at the very beginning progressing so well. But there is still some way to go.”

If you have any questions, please get in touch with [email protected]. You can find out more about vamorolone on Santhera’s website.