“A monumental milestone” in fight to beat Duchenne

Published Date
Alexa Follen

Since the news broke on the Food and Drug Administration’s (FDA) approval for Exondys 51, MDUK and fellow charities have been working hard to take forward the campaign to secure approval here in the UK and the rest of Europe.

Families across the world have welcomed the news that Exondys 51 has been given the green light in the United States. Claire O’Hanlon, Chair of MDUK’s Northern Ireland Council and whose son, Luke has Duchenne, has described it as a ‘monumental milestone’.

The drug could help people with Duchenne whose mutation is amenable to exon 51 skipping, such as Seth Burke (pictured).

Here’s an update on what’s been happening – and some details on what happens next.

  • When the news broke on FDA approval, MDUK’s Chief Executive wrote to the Executive Director of the European Medicines Agency. You can read the response here.
  • MDUK and fellow Duchenne charities have since met a number of times to share plans and campaign activity. Keep an eye out for further details on how we’ll all be working together to take this important work forward – and how you can get involved.
  • MDUK, Cheryl Gillan MP and a family affected by Duchenne will be meeting with the Health Minister, Lord Prior, this Thursday 13 October, where Exondys 51 and emerging drugs for Duchenne will be on the agenda.
  • In November, the All Party Parliamentary Group for Muscular Dystrophy will be holding a special session on Exondys 51 and other exon-skipping drugs. We’ll share a date for this very soon and will be asking you invite your MP to come along.

What needs to happen next

 The crucial next step is to ensure that Sarepta submits what is known as a Marketing Authorisation Application (MAA) to the EMA. In a recent webcast, the company has said that they plan to apply by the end of 2016. MDUK and fellow charities will be engaging with Sarepta in the coming weeks and months, and urging a broad licence application to include both ambulant and non-ambulant patients.

Once the company has applied:

  • The EMA will consider approval for the drug. The group within the EMA responsible for these considerations is the Committee for Medicinal Products for Human Use (CHMP). As part of this process, patients and families will be invited to share their experiences of living with Duchenne, and the importance of access to treatments such as Exondys 51 designed to slow down the progression of the condition.
  • Sarepta might be asked for further details, for example for clarifications on the clinical trial data
  • Once all the information required has been gathered, a CHMP ‘opinion’ will be issued which will determine whether the EMA is giving approval and granting a licence for Exondys 51.
  • If the EMA approves the drug, it will be up to authorities in the UK whether to make it available in hospitals here.
  • In England, NICE is responsible for this decision. NICE will consider drugs either by a Health Technology Assessment or through its Highly Specialist Technologies programme (HST). Given that Duchenne drug Translarna was assessed via HST, we would push for a drug like Exondys 51 to be assessed in the same way.
  • In Scotland, it is the Scottish Medicines Consortium who takes decisions on new drugs. In Wales and Northern Ireland, a separate process is used but this often follows the decision made by NICE. The Isle of Man has a separate process.

It is hard to give a definitive time frame for this whole process. For Translarna, the process took over three years. However, this was partly due to a request to the EMA for a reconsideration (they initially rejected a licence for the drug).This was followed by significant delays at NHS England, and a lengthy appraisal by NICE. MDUK has been working with NICE and NHS England – and we will be pushing to ensure that future decisions are taken with greater speed.

It will also be important to look at other ways of making the drug available more quickly – for example through the Early Access to Medicines Scheme.

Watch this video for more information on how the EMA interacts with patients – which has been shared by Alex Johnson at Joining Jack.

Lisa Burke, Seth’s mum, said:

Exondys 51 is so important to us as a family as it offers us hope…. For the first time, the outcome for DMD isn’t certain. We so hope that Seth gets a chance at a different and better outcome. He’s just a little boy and he deserves the same chances in life as every other child.

Chair of MDUK’s Northern Ireland Council, Claire O’Hanlon, said:

For me personally the approval of Exondys 51 is a monumental milestone in the life that has descended over three generations of my family… it has created the possibility of more efficient drug development and approval processes for other potential therapies and has presented opportunities for greater collaboration among the multitude of pharma companies, who will now feel that the Duchenne drug development industry in worth investing in. Approval of Exondys51 has heightened the resolve of the Duchenne community to fight tooth and nail to stop Duchenne.

Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK, said:

 The approval in the United States is a major step forward in the fight to beat Duchenne. We now need Sarepta to submit their application to the EMA without delay – and apply for the broadest terms possible, to include both ambulant and non-ambulant patients. The EMA must then act as quickly as possible to grant a licence to Exondys 51, taking into account the experiences of families living with DMD. Should the EMA give the drug the green light, NICE and other UK bodies need to ensure that the drug can be approved in the UK without delay. The approval process for Translarna took well over three years: there must not be a repeat of these delays this time around. Every day counts for patients and their families. MDUK is committed to working with fellow charities and patient groups here in the UK and the rest of Europe to drive this forward.  

To share your story – or for more information – please contact Peter Sutton on p.sutton@musculardystrophyuk.org or call 020 7803 4838.

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