The world's first long-acting C5 inhibitor! Ultomiris has been approved in Japan for its new adaptation: the treatment of atypical hemolytic uremic synthesis (aHUS)!

!--, 2020 // -- Alexion is a biopharmaceutical company dedicated to the development of new drugs for rare diseases.
recently, the company announced that Japan's Ministry of Health, Labour (MHLW) has approved Ultomiris (ravulizumab) for the treatment of atypical hemolytic uremic syndrome (aHUS) in children and adult patients.
aHUS is an extremely rare disease that can cause progressive damage to the kidneys and other organs.
Ultomiris is the first and only long-acting C5 inhibitor approved for the treatment of aHUS, which can be given monthly or every 2 months, depending on weight: (1) children and adults weighing more than 20 kg, once every February;
Ultomiris reduces the treatment burden on children and adults of aHUS and has the potential to become a new standard of care for aHUS clinical treatment.
U.S., Ultomiris was approved by the FDA in October 2019 for the treatment of aHUS adult and pediatric patients (-1 month) to suppress supplement-mediated thrombosis microvascular disease (TMA).
In the European Union, Ultomiris was approved in June this year for children and adults who had not previously received supplement inhibitor therapy (primary treatment) or had been treated with soliris (eculizumab) for at least 3 months and had evidence of a HUS in response to Soliris treatment, weighing more than 10 kg.
Ultomiris is the first and only long-acting C5 supplement inhibitor to receive regulatory approval.
In the U.S. and Japan, Ultomiris has previously been approved for the treatment of adult patients with cystic nocturnal hemoglobinuria (PNH), and in the European Union, Ultomiris has also been approved for the treatment of adult PNH patients with clinical symptoms indicating high activity and clinical stability after at least 6 months of treatment with Soliris.
is an extremely rare disease that can cause progressive damage to vital organs, mainly the kidneys, through damage to the walls of blood vessels and blood clots.
affects adults and children, many of them in critical condition in hospitals and often require supportive care in intensive care units, including dialysis.
can lead to sudden organ failure or slow loss of function over time, which can lead to transplants and, in some cases, death.
of ahuS is poor, with 56 per cent of adults and 29 per cent of children developing terminal kidney disease or death within one year of diagnosis.
, in addition to treatment, timely and accurate diagnosis is essential to improve patient prognosis.
consequences of aHUS are severe and can be life-threatening, facing significant challenges and uncertainties for patients and their families.
goal of aHUS therapy is to prevent the body from attacking itself by inhibiting uncontrolled C5 supplement activation, part of the body's immune system.
clinical studies showed that adult and child aHUS patients received immediate, complete C5 inhibition for up to eight weeks after Ultomiris was first given the drug.
Ultomiris provides greater freedom in addition to its clinically significant benefits for patients with aHUS, with a significant reduction in the number of infusions per year.
aHUS (Photo source: benthamopen.com) this time aHUS new adaptation approval, based on data from two global, one-arm, open-label studies (one for adult aHUS and the other for children aHUS).
currently, two studies are under way.
18 of the 21 cases of supplement inhibitors in primary treatment and 56 of the 58 cases of primary treatment of supplement inhibitors were included in the interim analysis.
The efficacy assessment of complete TMA remission was evaluated using hematological normalization parameters (platelet count and lactic acid dehydrogenase (LDH)) and improved renal function (measured by a 25% increase in serum creatinine from baseline levels).
showed that 54 percent of adults and 77.8 percent of children (medium-term data) showed complete TMA relief during the first 26 weeks of treatment.
Ultomiris therapy normalizes plateplate counts in 84 per cent of adults and 94 per cent of children, normalizes LDH (hemolysis markers) in 77 per cent of adults and 90 per cent of children, and improves renal function in 59 per cent of adults and 83 per cent of children (medium-term data) (for patients on dialysis at the time of entry into the group, the baseline is determined after they have been removed from dialysis).
During the 52-week follow-up period, four adult patients and three children confirmed full TMA remission after the initial assessment period of 26 weeks, resulting in overall full TMA remission in adults and children of 61% and 94% (medium-term data), respectively, at 8 Normalization of LDH (hemolysis markers) in 6 per cent of adults and 94 per cent of children (medium-term data) in 6 and 94 per cent of children (medium-term data) (baselines were established after they were removed from dialysis) in 6 per cent of adults and 94 per cent of children.)
pediatrics study also included a second group of 10 pediatric patients who had previously been treated with Soliris, which showed that a shift from Soliris to Ultomiris could maintain stable disease control, hematology and kidney parameters with no significant effect on safety.
these studies, the most common adverse reactions were upper respiratory tract infections, diarrhea, nausea, vomiting, headache, high blood pressure and fever.
meningococcal infection in patients treated with Ultomiris.
specific risk mitigation plans, including risk management plans (RPPs), have been developed for Ultomiris to minimize patient risk.
Ultomiris is a long-acting C5 supplement inhibitor that suppresses C5 proteins in the body's immune system's cascading response.
the drug has been positioned as an upgraded version of Alexion's heavyweight drug Soliris, which was first approved for sale in 2007 and has been approved for treatment of four ultra-rare diseases: PN H, atypical hemolytic uremia syndrome (aHUS), anti-acetylcholine-positive systemic severe muscle weakness (gMG), anti-AQP4 antibody-positive optic neurospinal myelitis spectrum disorder (NMOSD).
!--/ewebeditor:page--!--ewebeditor:page title"--Currently, Alexion relies heavily on Soliris, which accounts for more than 80 percent of its sales from the product.
Soliris is one of the world's most expensive drugs, priced at $500,000 a year, and since its launch, Soliris has accumulated more than $15 billion in sales for Alexion, one of the world's best-selling orphan drugs, with global sales of $3,946 million in 2019.
addition to expanding Soliris adaptations, Alexion is also developing an upgraded version of Ultomiris.
Ultomiris is the first and only long-acting C5 supplement inhibitor that can be given once every eight weeks.
based on strong clinical data and differentiated characteristics, the industry predicts that Ultomiris will become the new standard for clinical treatment in PNH and aHUS.
pharmaceutical market research agency, had forecast Ultomiris sales of $3.43 billion in 2024.
, Alexion is actively promoting market penetration at Ultomiris, as well as other adaptations to Ultomiris, including gMG, and Ultomiris, the subsc skin injection (SC) dosage form.
In June of this year, the phase III clinical study of the SC dosage form (once a week) was successful, with the new dosage form taking about 10 minutes to administer by subdern injection, while the Ultomiris intravenous preparation (IV) took 1.3-3.8 hours per dose (dose adjusted for weight).
() Original source: ULTOMIRIS (ravulizumab) Receives approval in Japan for Atypical Hemolytic Uremic Syndrome (aHUS) in Adults and Children !--/ewebeditor:page--.