Access to treatments

A community update about vamorolone and timeframes for patient access

Earlier this month, the drug company Santhera Pharmaceuticals, announced that it had applied for marketing authorisation (approval) of its Duchenne muscular dystrophy drug vamorolone in the UK. Vamorolone acts like a steroid but appears to have fewer of the side effects associated with steroid use in clinical trials.  

It is being developed by a US-based company called ReveraGen BioPharma Inc, though in 2020 Santhera acquired the worldwide rights to the drug. Vamorolone is still an investigative medicine, and is currently not approved for use by any health authority. 

Background to the development of vamorolone 

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, and Santhera has been engaging with us ever since to help secure access for patients in the UK. You can find more about the VISION-DMD programme, which is undertaking the phase 2b study, here. 

Will vamorolone become part of the Early Access to Medicine Scheme? 

In late 2019, vamorolone was granted a Promising Innovative Medicine (PIM) designation by the MHRA, the first step on entering the UK’s Early Access to Medicines Scheme (EAMS) – a government scheme Duchenne UK successfully campaigned for in 2014.  

EAMS is a way for drugs to become accessible to patients with life-threatening or severely debilitating illnesses before the MHRA grants market authorisation. Normally, all drugs need market authorisation, and an evaluation from the National Institute of Heath and Care Excellence (NICE), before becoming available through the NHS.  

Vamorolone needs to be given a positive scientific opinion before it can become available through EAMS. A positive scientific opinion lasts for one year, and is an assessment on the benefits and risks of the medicine, based on the data available. During the year, the pharmaceutical company provides regular updates on the safety and efficacy of the product in EAMS. The scientific opinion expires after the year is up, market authorisation is granted, or if the MHRA withdraws the positive opinion based on new data. The positive opinion can be renewed.  

What’s happening now?  

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 the project information for NICE’s HTA 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 EAMS pipeline. But this development with NICE suggests vamorolone is progressing through the MHRA’s processes. 

NICE’s HTA is at a very early stage – they are currently consulting on a draft scope, with a workshop at the end of October.  

Duchenne UK, Muscular Dystrophy UK and Action Duchenne are collaborating on our responses to this consultation. We will get a better idea of timescales once the scope has been finalised. 

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. 

If you have any questions, please get in touch with [email protected] 

Published on 14 October 2022

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