aHUS Trial Watch 18 – subcutaneous Ravulizumab

To the best of Global Action’s knowledge there has not been a trial of subcutaneous Ravulizumab for use in the treatment of aHUS.

There has been a comparison study between infusion and jab methods on PNH patients. That trial was completed and the results reported in 2022. The trial conclusion reported was

“Patients reported more satisfaction with subcutaneous ravulizumab than intravenous eculizumab, as assessed by the Treatment Administration Satisfaction Questionnaire. This study showed that patients with PNH can switch from intravenous eculizumab or ravulizumab to subcutaneous ravulizumab without loss of efficacy. Subcutaneous ravulizumab provides an additional treatment choice for patients with PNH.”

FDA had approved the once a week Ravulizumab SC technology for PNH patients and also for aHUS use even though there had NOT been a visible clinical study of its use by aHUS patients.

And that is how it stayed with very little further news. Although the health policy committee in the UK decided that the product would not be included in topic selection for appraisal saying the following :

Decision makers have concluded that this technology will not progress as a potential technology appraisal or a highly specialised technology. This decision follows consideration of the product against the eligibility and selection criteria for technology appraisal and highly specialised technologies. Section 6.2.1 of the Topic Selection Manual states that: Medicines that meet the eligibility criteria will be selected, except when there is a clear rationale not to do so. For example, when: changes to the dose, formulation or administration will not significantly affect the clinical and cost effectiveness of the medicine.”

SC Ravulizumab was the same active ingredient as infused Ravulizumab nothing is known about whether it is cost effective.

Recently however the.manufacture has announced* that the product has been discontinued explaining:

“Ultimiris discontinued plans to deliver subcutaneous administration for adults with aHUS or PNH. This decision follows persistent efforts to reliably secure the availability of the on-body delivery system”

*Q3 20023 Results- Astra Zeneca. (Page 5)

That leaves Crovalimab as the only known potential subcutaneous treatment for aHUS at the moment and that product is in mid trial. More information at Trial Watch 12 and Trial Watch 12a


December 4, 2023

Dear aHUS Community,
As many of you may have heard, after thorough evaluation, Alexion has made the difficult decision to
discontinue plans to the subcutaneous administration of Ultomiris (ravulizumab-cwvz) for adults with
atypical hemolytic uremic syndrome (aHUS) or paroxysmal nocturnal haemoglobinuria (PNH). The
decision follows persistent efforts to secure the reliability and availability of the on-body medical device
delivery system.

We recognize this news is disappointing to the aHUS and PNH patient communities. It has been a shared
journey to research and study the delivery system for subcutaneous administration and we are deeply
grateful to the patients and clinicians who participated in the clinical trials. Additionally, we appreciate
the time and commitment that the aHUS and PNH patient communities have shown – the insights you
have shared with us are invaluable. We will continue to look for ways to leverage these learnings to
improve the lives of those living with rare diseases.

We want to assure you that each of us at Alexion Astra Zeneca remains steadfast in our commitment to
the aHUS community.


VP, Global Patient Experience: Patient Advocacy, Engagement and Insights

Article No. 621

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