In my research into patients and my emails to clinicians who worked with Bronchitol in Europe, the drug works in some but not all patients and some better than others. You can argue that is the nature of CF and that also happens when you have trials with people aged 6 to 56. There is no miracle cure yet and Bronchitol is a mucus clearing agent. The target market is the 50% who don't get a benefit from Pulmozyme.
One issue in the trials is that the placebo was acually 50mg of Bronchitol twice a day (this is what the regulator wanted). The main group had 400mg twice a day. In both trials the placebo group showed some improvement and more so in the second trial as it has approx 50% under 17 years of age compared to only 35% in trial one. It seems even the low dose helped Children with smaller lungs. The regulator likes to net the 2 figures off (Control minus Placebo) so a placebo should not work. Obviously 50mg does a bit.
If the EMEA is willing to get the drug to those it works for then they have to find a label / clinical guidelines approval format that does that. Obviously the current one size fits all application won't be approved.
The way forward around adolescence and clinical meaningfulness is to work with the EMEA on a time frame in the current data that predicts when they know it will work or not. In the trials PXS took data after 6 weeks when I look at their reports. If they can show that in a clinical setting the outcome after 6 weeks follows the same pattern then this is one option. The risk/benefit becomes - If you don't respond you have only lost 6 weeks treatment time - If you do respond you continue on the drug. There were no safety issues.
No more trials would be required if this approach was successful.
I guess next week we will find out their plans.
Cheers
PXS Price at posting:
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