Drug similar to Soliris safe, shows long-term benefits for treating aHUS
Therapy approved in 2 countries, but not US, helped keep disease stable
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One year of treatment with Elizaria — a drug similar to the U.S.-approved therapy Soliris (eculizumab) — for atypical hemolytic uremic syndrome (aHUS) was found to be safe and to help stabilize kidney function among people with the genetic disease taking part in a study in Russia.
The results of the long-term study (NCT04749810) showed overall disease stabilization in aHUS patients who had not been treated previously, according to the researchers. Additionally, a stable course of aHUS was seen in those switching to the medication from Soliris. Safety results were positive, the scientists also noted.
According to the team, use of Elizaria — which is not approved in the U.S. — showed long-term benefits for those enrolled in the observational study, including normalized blood parameters. Elizaria, a Soliris biosimilar, has won regulatory approval in Russia and Turkey for treating aHUS.
The study, “Long-Term Therapy with Eculizumab Biosimilar in Patients with Atypical Haemolytic Uraemic Syndrome: Outcomes of a Prospective Observational Study,” was published in the journal Nephron. The work was funded by Generium, which developed Elizaria.
aHUS is caused by abnormal activity of the complement cascade, a part of the immune system, which results in blood clotting in small blood vessels. This is known as thrombotic microangiopathy, or TMA. The disease is marked by red blood cell destruction, known as hemolysis, low levels of platelets, which are small cell fragments that help the blood to clot, and kidney damage.
Drug similar to Soliris tracked over 1 year in Russia
Soliris is an antibody-based therapy approved for more than a decade in the U.S. that prevents the activation of the complement pathway. Elizaria is a Soliris biosimilar, meaning it has been designed to have similar biological properties, safety, and efficacy.
Here, a Russian research team reported on the long-term safety and effectiveness of Elizaria in treating people with aHUS at eight study centers in the country. Their study involved 50 aHUS patients, slightly more than half of whom (58%) were adults. The mean age of the enrolled children was 6.6 years; the average age of the adults was 33.6. More than one-third (38%) had previously received Soliris.
At screening, 72% of patients had chronic kidney disease, including 16% with end-stage renal disease, while 28% had acute kidney injury. Almost half of the patients tested had disease-causing variants in complement system genes, most commonly in CFH and C3.
During the study, each participant received 22 to 32 Elizaria infusions. Platelet counts improved in nearly all individuals after one year, the researchers noted, alongside a decline in lactate dehydrogenase (LDH) levels into the normal range. LDH is a marker of hemolysis.
Normalization of platelet and LDH levels was seen for 93% of the patients after 21 weeks, or about five months, and 95% after 52 weeks, or at the one-year mark. All adults who had not been treated with Soliris, and each of the children, irrespective of prior Soliris use, experienced a normalization of these parameters, the data showed.
The estimated glomerular filtration rate, an indicator of kidney function, remained stable during treatment, with 12% of patients improving above a predefined threshold after one year, the researchers noted.
The multicentre, prospective, observational study showed disease stabilisation in treatment-naive patients and a stable course of aHUS in treatment-experienced patients.
There was also a gradual shift toward milder disease categories, including more patients classified as having minimal chronic kidney disease activity and fewer with more advanced disease. These improvements appeared to occur more often in treatment-naïve patients, per the researchers.
TMA-related events were absent in 80% of patients, and plasma exchange therapy, which aims to replace a patient’s plasma (the liquid part of blood) to remove disease-causing components, was not used. At the end of the study, 8% of patients had a complete TMA response, defined as no abnormalities in LDH activity and platelet counts, as well as kidney function improvement, the data showed.
“The multicentre, prospective, observational study showed disease stabilisation in treatment-naive patients and a stable course of aHUS in treatment-experienced patients,” the researchers wrote, adding that this “may indicate similar efficacy of the biosimilar.”
No adverse events were deemed related to Elizaria
Elizaria was well tolerated, according to the study data.
While 63 adverse events were reported in 21 patients (42%), none were considered treatment-related. Most of these adverse events were mild to moderate in severity, though there were four serious adverse events occurring in two patients (4%). By the end of the study, most adverse events (88.9%) had resolved, the researchers noted.
Anti-drug antibodies were detected in 28% of patients during the study, which the data showing this was usually temporary and resolved during follow-up, according to the study.
The researchers noted that “the data obtained in this study may be valid for assessing the results of complement blocking therapy in other patients with aHUS.”
Still, per the team, “additional studies in a larger number of patients will provide more information on the efficacy and safety of the [Soliris] biosimilar [Elizaria] in the treatment of aHUS patients.”
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