to the presentation and am even more convinced that NTM and CF agendas will yield billions of dollars via direct revenues and valuation or acquisition by a major pharma. If anyone does not think that the big pharmas are watching, then I urge them to listen to yesterdays presentation. Imagine a new drug that could possibly cure NTM.........not just control it...........the big picture is far more critical than the current pps.
For me they key here is that if Arikace is effective against NTM it must also be effective against MDR-TB - a far bigger problem for the WHO and health authorities World-wide.
It killed around 150,000 people in 2011 despite the therapies currently available. Little wonder considering the length of time patients currently have to endure the nasty side-effects associated with the current antibiotic regimen.
A shorter regimen without those nasty side-effects would be a major game-changer for the WHO. Arikace looks all but certain to be granted an EU marketing authorisation, as efficacy has been established at Phase III and the safety issues have (unusually at this point) already been put to bed.
Imo widespread off-label use beckons as a result of the safety profile.
x10 - imo the likelihood of widespread off-label use can reasonably be inferred from the comments of a leading pulmonologist during the FDA-sponsored workshop on Non-CF Bronchiectasis in 2012 -
[ In the unit - in the hospital - we frequently double coverage people with pseudomonal pneumonia, for example, or gram-negative pneumonia, or just empirically before we know what anything is.
And a patient with a ventilator-associated pneumonia, or just a rip-roaring severe pseudomonal pneumonia, often gets two drugs of a different class ]
[ There's a lot more adverse events when you use two drugs versus one. I think in the end, probably you should use two drugs if someone's really sick, because it improves the chances that you're going to actually have one of them working ]
It's obvious Arikace can deliver an effective concentration of antibiotic to a pulmonary infection far more quickly than injections or tablets. When a patient is critically ill that could vastly increase his chances of survival.
We don't know how many of the 50,000 Americans who die from pneumonia each year are critically ill with an amikacin-susceptible infection, but amikacin most certainly is a top-ranked therapy for the 150,000 World-wide who die from pneumonia caused by MDR-TB each year.
And presumably the number of patients in both categories who are critically ill with pneumonia each year is far greater than the number who actually succumb.
I agree. IF approved, then why not use this therapy against other Amikacin susceptible pulmonary infections. Doctors should absolutely consider it if Arikace gains approval. Pulmonologists will know about the treatment through their CF or NTM patients, and it is a very small leap to start trying it against other infections.