FDA: 'Breakthrough' designation allows for drug approval on PhI data
One of the most tantalizing new programs now being rolled out at the FDA is its brand new category for "breakthrough" drugs, offering a select number of companies a chance at a shortcut to the market based on early-stage data for transformational new therapies. So when Janet Woodcock, the influential director of the FDA's Center for Drug Evaluation and Research, started outlining how the program will work--indicating that a company can move from an expanded Phase I directly to commercialization, Bloomberg reporters were paying close attention.
According to the business news wire, Woodcock says that companies which earn breakthrough status will have the ear of the agency. "We expect many of these would come available very quickly with Phase I data," she said.
So far, we know that Vertex ($VRTX) has won breakthrough status for two drugs for cystic fibrosis, the approved drug Kalydeco and the experimental VX-809, now being studied as a combination therapy. According to Woodcock, a third drug has been anointed with the special status. And developers have submitted 18 for review, most of which are for cancer.
Biotechs have been paying particularly close attention to this new, infinitely shorter path to the marketplace. A breakthrough designation would revolutionize their commercial prospects, upending some well known methods for building value through long-term partnerships with Big Pharma--which can easily take years to complete. Whether the FDA will allow smaller companies with less experience dealing with the FDA on the ultimate inside track, though, will be interesting to see.
- here's the story from Bloomberg
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