As a crucial deadline nears for a closely watched regulatory decision, two Republican senators are urging the Food and Drug Administration to approve a controversial drug to treat Duchenne muscular dystrophy.
In a letter sent last Friday, the senators expressed “disappointment” that an FDA advisory panel last month voted not to recommend eteplirsen to combat the disease, which is a rare and fatal genetic disorder that causes muscles to waste away. About 13,000 children, mostly boys, are afflicted and they typically die before turning 25 years old. The panel determined the drug was not effective.
Read Full Article »