"Infectious diseases are caused by pathogenic microorganisms, such as bacteria, viruses, parasites or fungi; the diseases can be spread, directly or indirectly, from one person to another."
The definition of a Qualified Infectious Disease Product under the Generating Antibiotic Incentives Now legislation would, strictly speaking, allow the designation of Arikace as a QIDP on the strength of its ability to combat NTM infection. But NTM clearly is not a pathogen targeted by the GAIN legislation. The intention of that legislation is to encourage the development of therapies with the potential to control pathogens which pose a threat to public health - i.e. pathogens with are transmitted person-to-person.
I don't believe for one moment that the ability of Arikace to kill NTM in itself would influence the FDA to designate Arikace as a QIDP. Nor do I believe that Lewis misunderstands the issue.
I see the confidence of Lewis in a QIDP designation as a clue to what he is really expecting, as opposed to what a CEO is allowed to communicate to investors. The crucial factor is that Lewis can only offer guidance on the basis of the current regulations. But he may privately be expecting the FDA to make decisions which are heavily influenced by the spirit of the new legislation, for which the relevant regulations are still to be written.
I make no apology for referring to the recent comments by Janet Woodcock, the head of the FDA, concerning the FDA's new resolve to "move drugs for serious diseases to market more quickly" -
... "We are having meetings for development of drugs for multi-resistant organisms with different companies".
... "That's a life-threatening situation, so we are treating it differently".
... "We expect many of these would come available very quickly with Phase 1 data".
That last comment is jaw-dropping for those of us who have invested in biotechs for any length of time. The FDA will now approve drugs primarily on the basis of safety data, together with preliminary evidence sufficient to support a reasonable expectation of efficacy.
Lewis twice refused to be drawn on how he expects Arikace to be positioned in the Cystic Fibrosis market, emphasising that it all depends upon the data due mid-year from the study in Europe and Canada. On that note it's worth pointing out the lack of relevance of the Technavio market analysis Rehdvm and Zake are banging on about. Technavio's projection of a inhaled antibiotic market worth $875 million in 2015 can make little allowance for -
peak sales of Arikace (not due to be launched until 2015)
the likely effect on the use of inhaled antibiotics of approval for patients with FEV-1 greater than 75%
the likely effect on the use of inhaled antibiotics of approval as part of a public health strategy.
I suspect Lewis is hoping to present the FDA with vastly superior efficacy over TOBI in the control of pseudomonas infection, together with compelling efficacy in the control of NTM infection both from the NTM study and from the Expanded Access program. And that the FDA, in the spirit of its new mandate, will issue a label which reflects a reasonable expectation (based upon the totality of the data) of the potential of Arikace to control pathogens which pose a threat to public health.
Or maybe I'm the only one thinking that :-)
But if he IS thinking along those lines he wouldn't tell us.
Btw - for me, a reasonable expectation based upon evidence that Arikace kills Non-tuberculosis mycobacteria (NTM) would be that Arikace will also kill Mycobacterium Tuberculosis.
One of the pathogens named in the new legislation is "multi-drug resistant tuberculosis". But if Lewis does consider it possible the FDA will approve Arikace as a therapy for susceptible strains of drug-resistant tuberculosis on the basis of data from the NTM study he couldn't let on. The FDA has regulations covering any guidance which could be construed as the promotion of a drug for a use not approved by the FDA.
Lewis is allowed to discuss the ongoing Cystic Fibrosis program, and the ongoing NTM program. Anything wider than that he may privately be expecting is off-limits for public discussion.
"No patient with latent or active TB will be inhaling Arikace or amikacin until a full blown clinical trial of that prescriptive use has been performed and reviewed by FDA, EMEA or whatever regulatory agency receive a master file."
1. You previously told us that TB is "not considered amikacin sensitive".
Why would anybody perform a full blown clinical trial of Arikace or amikacin for an infection which is not considered sensitive to amikacin?
2. According to you, the FDA will not approve a therapy for multi-drug resistant tuberculosis without a full blown clinical trial.
According to the head of the FDA, the FDA will approve a therapy for multi-drug resistant tuberculosis with a Phase 1 clinical trial.