FDA’s First Breakthrough Designations Continue Vertex Kalydeco’s Regulatory Successes
This article was originally published in Pharmaceutical Approvals Monthly
Label expansion efforts for Vertex’s cystic fibrosis therapy will be claims taken through FDA’s new review pathway; the initial product could reach more patients more quickly and serve as a guide to how the agency will handle drugs earmarked for streamlined development and approval.
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With FDA prohibited from disclosing information about products in development, sponsors must decide whether to announce if the agency has granted or denied a designation request. Communications are particularly challenging for small companies, for whom a denial might be a material event.
Trade organization may put its lobbying muscle behind ideas intended to lower trial costs and raise efficiency.
A few requests have gone to FDA for candidates to be named breakthrough therapies and gain access to shorter development programs, but the agency has not established all the program requirements yet.