PTC Therapeutics will need to carry out at least one further trial to demonstrate the effectiveness of its Duchenne Muscular Dystrophy therapy Translarna if it is to have a chance of bagging its approval in the US.
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Muscular Dystrophy UK is campaigning for faster approval and funding for therapies for children and adults with muscle-wasting treatments, and warns that the wait for a government response to the Accelerated Access Review “adds to delays to new drugs”.
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PTC Therapeutics has been denied its first appeal contesting US regulators’ refusal to accept its marketing application for Translarna as a treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
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Cost regulators for the NHS in England and Wales have now issued final guidance recommending the use of PTC Therapeutics’ Translarna for patients with Duchenne muscular dystrophy.
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Children in England suffering from a rare form of muscular dystrophy will soon be able to access the only drug licensed for their condition after a ground-breaking agreement was reached between NHS England and PTC Therapeutics.
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