Are there alternative aHUS drugs and ways to deliver them?

A top topic if the number of questions relating to this issue in the RDD2022 Video are to go by. Drug developments are very important, most importantly to provide more options for patients. It is important they work, also important is the how easily the drug can administered.

When eculizumb came along most aHUS patients were facing the burden of lifetime kidney dialysis, with or without plasma therapy, and all that it does to a patients. Two week infusions that took an hour of so , represented a considerable lifting of treatment burden. Falling three to six or more treatment sessions per week at four to six hours per session, to fortnightly infusions . No wonder when eculizumab was compared with the alternative it meant a considerable improvement to patients’ quality of life. Very considerable.

Of course many aHUS patients treated by eculizumab may never have experienced chronic dialysis, and so would see things differently, particularly children. Two week infusions procedures taking more than an hour or so to do when someone has not had to do that before is perceived as much more of a treatment burden. Infusions need entry points, usually with needling of veins or fistulas and sometimes with fixed ports. Those on treatment know the variation in ”needling” skills among health care professionals, Isabella.


When ravulizumab was offered with 4 or 8 week intervals between treatment it was viewed as lifting some of that burden. For ex dialysis patients it was a godsend. Though there was a quality of life improvement it was fraction of what was attained when eculizumab first became available. Health policy makers rarely witnessed such high quality of life year increases when comparing treatments as they did with eculizumab and PEX / Dialysis.


There are alternative drugs on their way, Landon and Darcey. And they will involve different ways in which they are delivered . The two most advanced developments are Crovalimab and Iptacopan,

The first is a monoclonal antibody which can be self or carer administered at home. At four week intervals. The drug is injected sub-cutaneously, into the skin. It takes a few seconds to do. More frequent treatments than ravulizumab but less frequency than eculizumab. And much quicker to do. An injection versus a line infusion. Fewer limitations for traveling. No more ports, Isabella and Darcey.

Ipcatopan is a tablet taken orally twice a day, Carine and Landon. Daily treatment but with little time needed to do it. Just swallow. Much more flexible when traveling from home to anywhere.

So the search is on, Brooke. There are more in the pipeline too, about nine possible drugs by Global Action’s reckoning. They could make the 2020s, a decade of greater treatment opportunities for those with Complement disorders, including aHUS. Some may only be used in niche markets which suit their competitiveness as earlier rivals to eculizumab have done. Some may not get that far.


So, daily, weekly, fortnightly, monthly , bimonthly by infusion , injection or by mouth. What would be your preference, Phoenixx

It is going to take a few years for Crovalimab and Iptacopan to be available as they need to be trialed first. Those trials are at phase 3 level i.e. (being used to treat aHUS patients) and have begun recruiting patients. If successful there are the further hurdles of getting a market licence and then insurance companies, hospitals and health policy makers deciding to use and fund them.

The funding decision will depend on whether they are cost effective and affordable . Will they be? Well that is another question?

Article No. 496

Previous articles about Crovalimab and Iptacopan and extended use of Ravulizumab for other Complement TMAs on the website.

aHUS Trial Watch 15 (Iptacopan)

Iptacopan is a complement inhibitor that it is taken orally, twice daily. It works by inhibiting Complement Factor B (CFB ). Without CFB , C5 cannot be activated to create…

aHUS Trial Watch 14

A trial has been designed by Alexion to examine the efficacy of Ravulizumab to treat those with a Thrombotic Microngiopathy , TMA, caused by a trigger. This somewhat vague scoping…CONTINUE READING

aHUS Trial Watch 14

aHUS Trials Watch 12a (crovalimab)

Article No. 445 13 July 2021 Hoffmann Roche has announced its pediatric trial of crovalimab for aHUS. It is featured on the ClinicalTrials.Gov website and can be found at this…CONTINUE READINGaHUS Trials Watch 12a (crovalimab)

aHUS Trial Watch 12 (crovalimab update)

aHUS Trial Watch 12 (crovalimab update)

Article No. 432 7 May 2021 In the past week or so, the possible trial of a new technology for treating aHUS, that we reported on in website  article  No…CONTINUE READINGaHUS Trial Watch 12 (crovalimab update)

aHUS Trials Watch 11 ( Crovalimab)

aHUS Trials Watch 11 ( Crovalimab)

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