Here is the rub. CF patients have Only 3 pharmacological options...

Here is the rub. CF patients have Only 3 pharmacological options to help mucus clearance from the lungs: 1) rhDNASE / pumozyme/ dornase alfa 2) hypertonic saline 3) Bronchitol . rhdnase only works in 30% of patients and it works by chopping up DNA in the infected mucus. In the remaing 70% it doesnt do anything. Ergo these patients only other option is hypertonic saline (very salty water) which is very unpleasant to taste and bronchitol which is slightly sweet to taste. bronchitol and HS work in a similar manner , but not by chopping up DNA. The Cf foundation in the USA was and is very enthusiastic about Bronchitol as this is giving the 70% of patients who dont respond to DNase a treatment that may very well work. Physicians would be negligent by not giving their patients a trial on bronchitol. If it happens to work for a particular patient, fantastic. Note that bronchitol demonstrated improvements in FEV1 in patients who were also taking DNASE. concurrently. The CF physicians in Europe objected strongly when the CHMP voted negatively last June, and that was probably one of the most instrumental acts causing the reversal of the CHMP decision to approve bronchitol in the EU. The FDA , approaches new drug applications by using a panel of CF experts in the decision making process (unlike the CHMP), so approval should be straighforwardish. I dont think it will be a slam dunk, but pressure from the CF community will guarantee that it will happen.