Ultomiris's reimb standards eased for aHUS

Expected to improve access to aHUS treatment

Treatment eligibility expanded this month to include transplant patients

“Timing of administration determines prognosis...need to establish a rapid treatment environment”

Prof.

 

Myung-Gyu Kim of Korea University Anam Hospital (Nephrology)

The reimbursement criteria for C5 complement inhibitors in atypical hemolytic uremic syndrome (aHUS) have been eased this month.

 

With this improvement, as the previous criteria had stringent reimbursement conditions, such as the requirement for prior review, patient access is expected to improve.

 

On the 24th, AstraZeneca Korea held a briefing at the Westin Seoul Parnas Hotel to commemorate the relaxation of reimbursement criteria for ‘Ultomiris (ravulizumab)’ in aHUS.

 

Ultomiris is a next-generation C5 complement inhibitor with a half-life approximately four times longer than that of the existing Soliris (eculizumab).

 

While Soliris requires administration every 2 weeks, Ultomiris extends the dosing interval to 8 weeks, improving treatment convenience.

 

aHUS is an acute rare disease where the immune system's complement system becomes overactivated due to a genetic defect, causing thrombotic microangiopathy.

 

This can lead to severe damage to multiple organs, particularly the kidneys.

 

aHUS refers to cases of hemolytic uremic syndrome that occur independently of E.

 

coli infection.

 

When complement component C5 is activated on bacterial surfaces, it generates a membrane attack complex that perforates cell membranes.

 

If this normal immune system process of complement activation continues unchecked, it causes problems in vascular endothelial cells, leading to related diseases.

 

Ultomiris has a mechanism that inhibits this process.

 

Expanded diagnostic and administration criteria enable timely treatment Ultomiris became reimbursable by national health insurance in January for patients with aHUS accompanied by thrombotic microangiopathy (TMA) and kidney damage.

 

Previously, treatment was restricted to patients who had received prior approval through a pre-review process.

 

As this can cause delays leading to disease progression, clinicians have called for a switch to post-approval review to allow faster initiation of therapy.

 

In fact, since the implementation of the aHUS pre-review system, the average reimbursement approval rate has been only 18% (based on cases reviewed from July 2018 to August 2025), often resulting in missed treatment windows.

 

The revised regulations, effective from the 1st of this month, minimize treatment delays by broadening the diagnostic and administration requirements for aHUS patients.

 

Experts evaluate that clarifying the evaluation standards for treatment effectiveness may help maintain treatment continuity.

 

Improvements in Korea

The revised criteria specify that TMA activation can now be confirmed if three or more of five hematologic criteria, including thrombocytopenia, are met.

 

Furthermore, reimbursement is also granted if the patient meets the administration criteria, including ADAMTS-13 activity of 10% or higher.

 

Even before test results are confirmed, if the platelet count is 150×10⁹/L or higher, immediate administration is possible after submitting a pre-application form, and the administered dose is covered until the review result is notified.

 

Furthermore, a new treatment pathway has been created as it explicitly states that when a kidney transplant is performed due to end-stage renal failure caused by aHUS, coverage is granted on a case-by-case basis.

 

The criteria for coverage of switching from Soliris to Ultomiris have also been clearly defined.

 

Prof.

 

Myung-Gyu Kim of Korea University Anam Hospital (Nephrology) said, “aHUS can rapidly progress to end-stage renal failure or multi-organ damage within 48 hours, making treatment timing critical to prognosis.

 

The revised criteria reflect global best practices and strengthen evidence for continued therapy in high-risk patients, establishing an environment enabling the rapid design of treatment strategies.”