aHUS Trials Watch 6

As aHUS Awareness Day approaches it is timely to be aware of developments in the next version of eculizumab , the version now known as ALXN 1210.
There are 7 trials taking place , 5 for PNH patients and 2 for aHUS patients.
The two trials for aHUS patients are split between adults and children. The  trials will take another 2 years to complete.
The adult trials are about both the amount of the drug which will be needed according to the weight of the patient and the length of time between doses. The delivered doses will be higher than with original eculizumab but not as much would be needed as previously because the interval between doses will be 8 weeks.  56 trialists have been recruited from 41 locations.
Recruitment to the children’s trial is still taking place and 16 candidates are being sought. As there will be 4 weight bands to determine dosage it is planned to have 4 patients for each band . The same dose interval of 8 weeks is being used.
These are the sites currently recruiting

Click on the following links to find out more about what the trial is about , including the cities in which the adult trialists have been recruited.
Children ‘ s Trial
Adults’ Trial 
 
It is hopeful that this version will be as clinically effective as the original eculizumab which has transformed the lives of so many that have benefitted from it.
The treatment burden will be reduced too , just 6 infusion per year instead of 26 something to be welcomed.
But perhaps even more good may come , all things being equal , from the roughly 33% or more  reduction in the amount drug used reducing the cost of treatment and making it more affordable and sustainable for those in counties which deny or cap access because of affordability. Wouldn’t it be good if at least 50% more patients could access it when needed for as long as it is needed?
On the other hand the manufacturer may just increase the vial price by 50%.
That will be an acid test too.