From Form 10-K, Next Expected Milestones for NTM indication...
• We expect to report top-line clinical results from our phase 2 clinical trial in March 2014.
• We expect to enroll the first patient in our single-arm, open label, supportive study in the United States and Europe during the second quarter of 2014.
• We expect to have dialogue with the FDA and the European Medicines Agency ("EMA") in the second quarter of 2014 to discuss the regulatory pathway.
• If approved, we expect ARIKAYCE would be the first approved inhaled antibiotic treatment for NTM lung infections.
• We are developing plans to commercialize ARIKAYCE, if approved, initially in the United States, in certain countries in Europe, and Canada and eventually Japan.
" If the EMA allows a filing that includes both the CF and NTM indication"
The EMA will not allow a dual filing for 2 different indications unless NTM shows cures.
In the US the FDA will not allow arikayce to be sold unless they trial it here.
That's going to be a dialogue with the FDA. And frankly out of respect for them we will not be coming out with a comment on whether we're filing or not when we have the data release at the end of March. We're going to wait until we have had dialogue with them based on the data we have to inform where we go from here. ]
This explains the official company guidance on the US launch of Arikace - it had to allow for the possibility of a Phase III NTM study.
However - the primary objective of the legislation enacted last July was the "expedited development and review of innovative new medicines intended to address unmet medical needs".
There are no antibiotics currently available to treat pulmonary mycobacterial infections such as TB and NTM which are not associated with serious side-effects.
The side-effects have historically resulted in breaks in the antibiotic regimen - leading to the current situation where well over half a million people a year are infected with pathogens which have evolved resistance to first-line antibiotics.
The side-effects associated with the antibiotics (particularly aminoglycosides) currently used to treat drug-resistant mycobacterial infections are even worse. Injected aminoglycosides are associated with permanent loss of hearing.
If the NTM results due by the end of this month do indeed show that Arikace not only delivers an effective concentration of aminoglycoside to a pulmonary mycobacterial infection, but does so in a manner which is far safer and far more patient-friendly - Arikace will obviously have the capability to address a serious unmet medical need.
What reason could the FDA have to act in a manner inconsistent with the primary objective of the new legislation?