Vertex may have misjudged the data that regulators will want to see before approving one of the company's most prized pipeline drugs. On Wednesday, the Food and Drug Administration held a workshop on developing medicines for alpha-1 antitrypsin deficiency, a genetic disorder where lack of a protein called AAT causes lung and liver damage. Vertex aims to treat the disorder with VX-814, an experimental therapy that should yield Phase 2 proof-of-concept results by next year.
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