Title: Exon skipping therapy for Duchenne muscular dystrophy: the long road to accelerated drug approval
Presenter: Dr Karen Anthony PhD, AFHEA, Lecturer in Molecular Bioscience, University of Northampton
A new drug to treat Duchenne muscular dystrophy (DMD) was recently granted accelerated approval by the United States Food and Drug Administration (FDA). Dr Karen Anthony worked on the early development of this drug at University College London (UCL) and will tell the story of how this drug was successfully brought to market.
DMD is a rare, 100% fatal, childhood genetic disorder that overwhelmingly affects boys. It is characterised by muscle weakness and breakdown leading to death in early adulthood. The drug, branded Exondys 51, forms a ‘molecular patch’ over specific DNA mutations that cause Duchenne. It is hoped that the drug can restore some muscle function and improve the lives of those living with Duchenne.
Controversially, clinical trials have failed to demonstrate that taking the drug actually provides a meaningful clinical benefit. The FDA have ruled that, in this case, the consequences of not approving an effective drug would be far greater than approving an ineffective drug. Under the accelerated approval pathway Sarepta Therapeutics (the company that made the drug) must now perform a confirmatory clinical trial to prove that the drug can indeed achieve its intended outcomes.
Even if deemed a success, this drug is not a cure. Karen will talk about the future challenges of this field and her latest research stemming from this pivotal work.
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