Dear MP Name
I am writing to you regarding the availability of eteplirsen (Exondys51) for the treatment of Duchenne muscular dystrophy caused by a mutation amenable to the skipping the exon 51.
Duchenne muscular dystrophy is a severe muscle-wasting condition caused by the lack of a muscle protein called dystrophin. Around 2,500 people in the UK are affected by Duchenne muscular dystrophy, with approximately 100 diagnosed every year. This extremely serious condition causes progressive muscle weakness leading to dramatic loss of muscle function. The natural history of Duchenne shows that patients typically lose ambulation in their early teens, will require respiratory support in their mid to late teens and most are likely to die of heart failure or respiratory compromise before the age of 30.
You may like to include extra information here on your own connection to Duchenne muscular dystrophy
Recently, families in the United States received the long awaited for news that the drugeteplirsen is to receive accelerated approval from the Food and Drug Administration. This is a landmark decision – eteplirsen is the first Duchenne muscular dystrophy exon-skipping drug to receive approval from a regulator.
It’s now important that we focus efforts on securing approval from regulators here in Europe at the European Medicines Agency (EMA). This will be dependent upon the submission of a Marketing Authorisation Application (MAA) from Sarepta Therapeutics. Patient groups such as Muscular Dystrophy UK are in close contact with the company to gain a full understanding of their plans in this area. They had originally hoped to apply by the end of 2016.
However, at this stage I am contacting to ask that you write to the Executive Director at the EMA – Professor Guido Rasi- to seek his assurances that any approval of eteplirsen would be carried out quickly. Duchenne is rapidly progressive – and every day counts for those affected. A long wait for an EMA approval could make all the difference between the levels of benefit that a patient would derive from the drug: as once lost, muscle mass and strength cannot be restored.
Professor Rasi can be contacted at:
European Medicines Agency
30 Churchill Place
Canary Wharf
London E14 5EU
United Kingdom
If you require any further information – or a suggested letter to send to Professor Rasi – please contact Peter Sutton on 020 7803 4838 or email .
Thank you for taking the time to consider the questions posed in this letter. I look forward to hearing from you shortly.
Yours sincerely,