The world's first long-lasting C5 inhibitor! Ultomiris New Indications has been approved by the European Union: Treatment of atypical hemolytic uremia synthesis (aHUS)!

, June 29, 2020 /PRNewswire/ --BiovalleyBIOON/ Alexion is a biopharmaceutical company dedicated to the development of new drugs for rare diseasesRecently, the company announced that the European Commission (EC) has approved ultomiris (ravulizumab) a new indication for the treatment of patients with atypical hemolytic uremic syndrome (aHUS) patients, specifically: children and adults who have not previously been treated with complementary inhibitors (initial treatment) or have been treated with Soliris (eculizumab) for at least 3 months and have evidence of a reaction to Soliris treatment, weighing up to 10 kgmedication, ultomiris maintenance doses can be infusions every 8 weeks or every 4 weeks (depending on weight) 2 weeks after the load dose (load dose)Previously, Ultomiris has been approved for the treatment of pontoonaryain (PNH) adult patientsit is worth mentioning that Ultomiris is the first and only approved long-acting C5 inhibitor for the treatment of aHUS, which can be administered monthly or every 2 months (depending on weight), reducing the therapeutic burden on aHUS children and adults, and has the potential to become the new standard of care for aHUS clinical treatment in EuropeIn the United States, Ultomiris was approved by theFDAin October 2019 for the treatment of patients in adult samps and pediatrics (-1 month) to suppress complementary-mediated thrombotic microvascular disease (TMA)Ultomiris' new application for aHUS for treatment is currently under review by Japanese regulatorsUltomiris is the first and only long-acting C5 supplement inhibitor to receive regulatory approvalIn the United States and Japan, Ultomiris has previously been approved for the treatment of pontoonnonoproteinin (PNH) adult patients;aHUS 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 clotsaHUS affects adults and children, and many patients are in critical condition in hospitals and often require supportive care, including dialysis, in the intensive care unitaHUS can lead to sudden organ failure or slow loss of function over time, which can lead to the need for a transplant and, in some cases, deathThe prognosis of aHUS is poor, with 56 per cent of adults and 29 per cent of children developing end-stage kidney disease or death within one year of diagnosis Therefore, in addition to treatment, timely and accurate diagnosis is essential to improve the patient's prognosis Professor Hermann Haller, a nephrology clinic at the University of Hannover in Germany, , said: "The consequences of aHUS are very serious and can be life-threatening, posing significant challenges and uncertainties for patients and their families The goal of aHUS therapy is to prevent the body from attacking itself by inhibiting uncontrolled Activation of the C5 supplement, part of the body's immune system Clinical studies showed that adults and children with aHUS received immediate, complete C5 inhibition for up to 8 weeks after Ultomiris was given the first administration In addition to its clinically significant benefits to aHUS patients, Ultomiris provides greater freedom, with a significant reduction in the number of infusions per year "At Alexion, our goal is to continue to improve the lives of patients and their families affected by aHUS and other severe rare diseases," said Dr John Orloff, Executive Vice President and Head of Research and Development at Alexion Ultomiris treatment provides a convenient 8-week dose, which we consider to be the first choice for patients because it provides patients with greater flexibility and quality of life, while reducing the burden on the healthcare systems that are currently under significant pressure in many countries Today's EU approval marks an important step in our efforts to establish Ultomiris as a new standard of care for the aHUS patient population "
aHUS (picturesource: benthamopen.com) this aHUS new indications approval, based on data from two global, one-arm, open-label studies (one for adults aHUS and one for children aHUS) Currently, two studies are under way In 18 of the 21 cases of complementary inhibitors in primary treatment in children and 56 in 58 adults with supplement inhibitors were included in the mid-term analysis The efficacy assessment of complete TMA mitigation was evaluated using hematologic normalization parameters (platelet counting and lactoacid dehydrogenase (LDH) and improved renal function (measured by serum creatininine by a baseline level of 25%) results 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 treatment normalized platelet counts in 84% of adults and 94% of children, normalized LDH (hemolytic marker) in 77% of adults and 90% of children, and improved renal function in 59% of adults and 83% of children (medium-term data) (for patients on dialysis at the time of admission, the baseline was determined after they were released from dialysis) during the 52-week follow-up period, 4 additional adult patients and 3 children confirmed full TMA remission after the initial assessment period of 26 weeks, resulting in total total Total TMA remission in adults and children of 61% and 94% (medium-term data), normalization of LDH (hemolytic markers) in 86% of adults and 94% of children, and improvement of renal function in 63% of adults and 94% of children (medium-term data) a second queue was included in the pediatric study, which showed that the switch from Soliris to Ultomiris to Ultomiris could maintain stable disease control, hematology and kidney parameters with no significant effect on safety in these studies, the most common adverse were upper respiratory tract infections, diarrhea, nausea, vomiting, headaches, high blood pressure
and fever Severe meningococcal infection occurred in patients treated with Ultomiris To minimize patient risk, specific risk mitigation plans, including risk management plans (RMP), have been developed for Ultomiris Ultomiris is a long-acting C5 supplement inhibitor that suppresses the C5 protein in the body's immune system complement cascade reaction The drug is positioned as an upgraded version of Alexion's heavy-duty drug Soliris (intravenous infusion every 2 weeks), which was first approved for market in 2007 and has been approved for treatment of four super-rare diseases: PNH, atypical hemolytic uremia syndrome (aHUS), anti-acetylcholine receptor-positive systemic severe muscle weakness (gMG), anti-AQP4 antibody-positive neurospinal malaria malaria neurostinitis.) , Alexion relies heavily on Soliris, which generates more than 80 percent of its sales Soliris is one of the world's most expensive drugs, priced at $500,000 a year, and since its launch, Soliris has generated more than $15 billion in sales for Alexion, one of the world's best-selling orphan drugs, with global sales of $3.946 billion in 2019 The industry predicts that NMOSD Indications, approved in August 2019, will generate about $700 million in additional sales for Soliris In addition to expanding Soliris indications, Alexion is also developing an upgraded version of Ultomiris, which has been approved for PNH indications in the United States, Japan, Europe and the United States, and aHUS in the United States and the European Union Ultomiris is the first and only long-acting C5 supplement inhibitor that can be administered once in 8 weeks, and in Phase III clinical studies for the treatment of PNH, Ultomiris infusions every 2 months (8 weeks) and Soliris infusions every 2 weeks have achieved non-deefficienance in all 11 endpoints industry predicts that Ultomiris will become the new standard for PNH and aHUS clinical treatment based on strong clinical data and differentiation characteristics Evaluate Pharma, a pharmaceutical market research firm, had forecast ultomiris sales of $3.43 billion in 2024 Alexion is actively promoting ultomiris market penetration, while also actively developing other ultomiris adaptations, including gMG, and the development of subcutaneous injection (SC) dosage form Ultomiris just recently, A Phase III clinical study of The SC Dosage (once a week) was successful, with the new dosage product taking about 10 minutes to be administered by subcutaneous injection, while Ultomiris intravenous formulations (IV) were administered for 1.3-3.8 hours each time (dose adjusted according to weight) , Alexion is also evaluating the hyperactive inflammatory response in patients with the treatment of the new coronavirus pneumonia (COVID-19) in Ultomiris and Soliris (BioValleyBioon.com) original origin: ULTOMIRIS ® (ravulyzumab) Receives Marketing European From European For Adults and Children with Atypical Hemolytic Uremic Syndrome (aHUS) .