An influential committee was due to meet today to discuss whether or not to recommend approving the drug eteplirsen for people with Duchenne Muscular Dystrophy.
Members of the Food and Drug Administration (FDA) will sit down to consider the safety and effectiveness of the potential treatment before making their recommendation.
Claire O’ Hanlon, whose five-year-old son Luke suffers with the muscle-wasting disease, will be representing the charity Muscular Dystrophy UK at the all-important meeting.
“This is a really big day for Duchenne families and important we all come together to show our support for FDA approval of this drug,” said Claire, from Northern Ireland.
“We also need approval in Europe to get the drug to our children in the UK, so it’s crucial we persuade the drug company to apply to the European Medicines Agency.”
Ahead of the meeting, 36 researchers signed an open letter urging the FDA to recommend approval.
They cited evidence taken from clinical trials which suggested that eteplirsen had made a “significant difference” to the symptoms of those with the condition. After four years of treatment, they said that the tests had also confirmed the drug was safe.
Professor Matthew Wood, an expert from Oxford University, said: “Muscular Dystrophy UK has played a critical role over almost two decades supporting the development of exon skipping drugs [such as eterplirsen], which have real potential to bring significant clinical benefit to Duchenne patients.”
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