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.
"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."
Could Insmed be denied the "the ultimate inside track" because of its size?
But Woodcock also disclosed -
"We are having meetings for development of drugs for multi-resistant organisms with different companies".
Amikacin (the antibiotic delivered by Arikace liposomes) is already approved by the FDA for the treatment of serious pulmonary infections due to susceptible strains of all four Gram-negative pathogens named in the GAIN legislation as having multi-drug resistant strains with the potential to pose a serious threat to public health.
Amikacin is also routinely used off-label for the treatment of multi-drug resistant tuberculosis - also named in the GAIN legislation as having the potential to pose a serious threat to public health.
I've no doubt that Insmed is one of the companies with which the FDA has been having special meetings as part of its strategy to prevent the development of drug-resistant pathogens.
Many Longs on this MB recognize that Arikace for NTM will probably qualify under these criteria. There are virtually no NTM drugs that will eradicate (kill all) NTM bacteria among these patients. The science that will prevail will be the INSM liposome mimicing the naturally occuring lipid surfactant in the lungs and being phagocytized (engulfed) by the fixed macrophages in the lung which is where the NTM bacteria hide. There are no treatments that consistently eradicate NTM at this time.
The next big breakthrough drug for the liposome will probably be a long acting asthma drug in the INSM liposome which will modulate the acute reactive proteins in these same macrophages. These macrophages produce small molecules that signal changes the physiology of lung blood flow, white blood cell infiltration and other pulmonary functions. But first we have to get the antibiotic liposome approved.