Recommended aproval of Muscular Dystrophy drug in EU


The European Medicines Agency (EMA) has given the go ahead for a drug which treats a particular type of Duchenne muscular dystrophy. The drug may be available in the UK within six months.

The EMA made the decision not to clear the drug during January, but have since re-examined the available evidence.

A group of campaigners have asked that the drug reaches the children who need it without delay. There are currently no approved treatments for this life-threatening illness. The patients who will gain the most from this treatment are those who are aged five years and older, and who still have the ability to walk.

Duchenne muscular dystrophy is a genetic condition which causes gradual weakness and eventual loss of muscle function. Patients who suffer from this disease have a lack of a protein present in muscles, which aids in protecting the muscles from injury.

In the UK, there are around 2400 children affected by this condition. However, only those with a ‘nonsense mutation’, about 200 children, will benefit from the use of Translarna.

UK Need

The drug, ataluren, which was developed by PTC Therapeutics, will be named Translarna in the EU.

The next step in the process will be for the European Commission to approve the go ahead by the EMA within three months, and authorise the marketing of the drug in the European Union.

Once that has happened, the member states, including the UK, must make a decision on the method of funding.

The Muscular Dystrophy Campaign has called for urgent meetings with NHS England and the National Institute of Health & Clinical Excellence (NICE) to discuss the approval process and use of the drug in the UK. It stated that families within the UK may have access to this drug by spring 2015.

The Chief Executive of the campaign, Robert Meadowcroft, said the decision by the EMA should be regarded as fantastic news. He said that most patients diagnosed with Duchenne muscular dystrophy before the age of five, will be using a powered wheelchair by the time they reach the age of 12. They will not have adequate muscle strength to pick up a glass of water by the time they reach the age of 20, and will not live long enough to celebrate their 30th birthday.

He further added that this huge step offers many patients much needed hope for the future, and that by extending the numbers of patients who use Translarna, the development of the drug will be boosted.

The clinical trials on the drug are still in process and the results will need to be presented to the EMA.

Image Credit: worak


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