This weekly column offers opinions on the latest pharmaceutical industry news.
An advisory panel to the Food and Drug Administration was supposed to have met last Friday to decide the fate of an experimental treatment from Sarepta Therapeutics for a rare disease called Duchenne muscular dystrophy. But with a blizzard engulfing the nation’s capital, the agency delayed the meeting — and, in so doing, left alive the last glimmer of hope for families afflicted by this fatal genetic disorder.
They may have a long wait. When the FDA panel finally meets, it appears likely it will recommend against approving the drug from the Cambridge, Mass.-based company. And under the circumstances, this might be a good thing.
Of course, no one wants to see children suffer, and it would be a huge boon to patients and society for a therapy to become available for a critical unmet need.
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