Number of subjects with treatment-emergent adverse events by seriousness -
Arikace: 26 out of 148
Tobi: 29 out of 146
2. Not serious
Arikace: 125 out of 148
Tobi: 113 out of 146
3. Life Threatening or Disabling
Arikace: 0 out of 148
Tobi: 1 out of 146
Arikace will not only be far safer than the antibiotics presently used by the Cystic Fibrosis population (the liposomal delivery prevents all but a negligible amount of the antibiotic from reaching the bloodstream) - it will be the only antibiotic capable of delivering a noticeable improvement in lung function with prolonged use - and the only inhaled antibiotic requiring administration just once daily.
Seattle Children's Hospital, Director, Cystic Fibrosis Therapeutics, Development Network Coordinating Center and Chair of the study Steering Committee
Right. So on Steering Committee, we have looked at the adverse event profile compared with the comparator, which is TOBI and none of them were unexpected. This is a patient population that tends to have respiratory symptoms of all kinds. And therefore, it's not unexpected to have a certain level of symptoms such as cough and so the distribution actually looked very comparable between the two.
There was one patient, who had two episodes of severe hemoptysis and that patient actually was in the TOBI arm. So there was no patient in the ARIKACE arm with severe hemoptysis, which might have been a concern.
Thanks. That actually links directly to my follow up, which is any of the severe side effects that you have seen on either ototox or kidney tox?
I can take that question. There is no evidence of renal toxicity and there is no evidence of ototoxicity attributed to either study.
As regards efficacy - the FDA decided to approve Tobramycin Inhalation Powder despite the fact that it failed in one of its Phase III studies to demonstrate superior efficacy to a PLACEBO.
27% of the TIP arm dropped out during the study, and agency staff publicly voiced concerns over both safety and efficacy.
But even though TIP needs to be administered twice-daily it still takes around one-quarter of the time of the inhalation solution. The FDA clearly has a high priority of countering the threat of antimicrobial resistance by promoting better compliance. Once-daily Arikace imo will be a no-brainer for the FDA.