https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/brc-news/accelerated-approval-granted-drug-developed-gosh/
Accelerated approval granted for drug developed at GOSH
14 Dec 2016, 9:55 a.m.
The United States Food and Drug Administration (FDA) granted accelerated approval for a new medication to treat Duchenne muscular dystrophy (DMD). The drug, Eteplirsen, was developed by a UK consortium led by Novel Therapies Theme lead, Professor Francesco Muntoni.
DMD is a severe, muscle-wasting condition caused by a fault in a gene, resulting in a failure to produce functional dystrophin. Currently DMD has few treatment options.
Eteplirsen was filed by Sarepta Therapeutics for accelerated approval by the FDA and is planned for immediate clinical use in the US. The approval for use in the EU is still underway. The drug will be used to treat patients with a specific subset of mutations of the dystrophin gene that affects around 13% of boys with DMD.
The drug ‘skips’ a part of the gene that makes dystrophin, which results in a shortened form of the dystrophin protein being produced, alleviating some of the symptoms of DMD and potentially extending a patient's mobility for a longer period of time.
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