Scots Hammer PTC's Translarna As EU Reviews Conditional Approval

In another blow for PTC Therapeutics' orphan drug for Duchenne muscular dystrophy, Translarna (ataluren), the Scottish Medicines Consortium has rejected the use of the product on the national health service, saying that the company had failed to justify its high cost in relation to its clinical benefits. The move comes in wake of the US Food and Drug Administration's February refusal to accept PTC's filing for Translarna.

The SMC, Scotland's health technology assessment body, said that despite the "powerful and moving testimonies" given by patient groups and...

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