The world's first long-lasting C5 inhibitor! Alexion's weekly Ultomiris subcutaneous preparation Phase III clinical success!

, June 25, 2020 /
PRNewswire/ -- Rare Disease Pharmaceuticals, a pharmaceutical giant, has released the results of a weekly clinical study of the weekly self-subcutaneous injection (SC) dose form Ultomiris (ravulizumab-cwvz) to treat patients with bispentasleep hemoglobininoine (PAH)ultomirisis the first and only long-acting C5 supplement inhibitor and is currently on the market as an intravenous (IV) formulationUltomiris has been approved for treatment in adult PNH patients in the United States, the European Union and JapanIn the United States, Ultomiris has also been approved for the treatment of atypical hemolytic uremic syndrome (aHUS) in adult and pediatric patients (age of 1 month) to inhibit complementary-mediated thrombotic microvascular disease (TMA)In adult patients, intravenous administration is administered every 8 weeks, and in pediatric patients weighing 20 kg, intravenous administration is administered every 4 weeksUltomiris doses are adjusted for weight, and the total infusion time for each intravenousis 1.3-3.8 hours
once a weekUltomiris SC preparations are administered with two specially designed, patient-friendly devices attached to the body and self-injected by pressing a buttonThese devices can be used simultaneously or sequentially, and can be used in about 10 minutesprovide sufficient amounts of Ultomiris SCs without hand- The SmartDose ® device, which has previously been approved by the U.SFDAfor another treatment, is a single-use device that includes a pre-filled cartridge and was developed in collaboration with West Pharmaceutical Services to provide patients with a more flexible Ultomiris treatmentis an ongoing global, random, open-label, parallel group, multicenter III study that included 136 clinically stable cases and received C5 supplement inhibitor Soliris (eculizumab) every 2 weeks prior to the start of the studyInfusion once) treatment of pNH adult patients for at least 3 months, the main purpose of which was to evaluate the non-toxicity (PK) of Ultomiris SC preparations and Ultomiris IV preparations, using the 71st day Ultomiris serum valley concentrationSecondary endpoints include: safety, immunogenicity, PK/PD, quality of life, equipment performance, efficacy evaluation measures, etcstudy, the patients were grouped by weight (from 40 kg to 60 kg, from 60 kg to 100 kg) and then randomly assigned to Ultomiris SC or Ultoares IV at a 2:1 ratioAll patients receive an initial IV load dose on day 1On the 15th day, patients in the Ultomiris SC group began receiving a fixed-dose self-administered dose of Ultomiris SC once a week, and patients in the Ultomiris IV group received a single infusion of approved weight-based IV dosesresults showed that the study reached the main end point: on the 71st day of treatment, the Ultomiris SC preparation was not inferior in terms of pharmacokinetics (PK) to Ultomiris IV (serum Ultomiris Valley concentration-Crough non-disadvantaged p 0.0001)The serum free C5 concentration in all patients remained below the target threshold, and the average lactic acid dehydrogenase (LDH) level remained below the normal upper limitInitial safety data for the 71-day randomized treatment period are consistent with ultomiris' known safety data and there are no unexpected safety findingsThere were no adverse events in either group that led to the discontinuation of the drugNo seriousadverse reactionsor meningococcal cases were reported, and anti-drug antibodies were not foundof 135 patients who completed the 71-day randomized controlled treatment period of the study, all but one chose to enter the Extended Ultomiris SC period and receive weekly Ultomiris SC treatment for a maximum of 182 additional weeksThe extension period will provide 12 months of security data for the submission of regulatory documents to regulators, which is currently expected to be submitted in the third quarter of 2021 to meet all regulatory requirements for the filing of the portfoliois currently under studyFollowing the completion of the study, including the collection of 12 months of safety data in accordance with the agreement with the U.S FDA , Alexion expects to submit a listing application for Ultomiris SC preparations and equipment combination products for PNH and atypical hemolytic uremia syndrome (aHUS) in the U.S and the European Union in the third quarter of 2021 "These data suggest that the subcutaneous injection agent Ultomiris provides the same immediate, complete and continuous supplement suppression benefits as intravenous formulations, while also providing an additional treatment option for patients who prefer to administer it themselves," said Dr John Orloff, executive vice president and head of research and development at Alexion Subcutaneous injection Ultomiris delivers drugs through a fast, patient-friendly dosing device, an example of Alexion's ongoing commitment to in-doing for patients This combination of drugs and devices has the potential to be the first undercut treatment option for PNH and AHU treatments, as well as the potential to improve the quality of life of patients "
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, which was first approved for sale in 2007 and has been approved for treatment of four super-rare diseases: PNH, atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor-positive systemic severe muscle weakness (gMG), anti-AQP4 antibody-positive spinal spinal spectrum disorder (NMOSDD) currently relying heavily on Soliris, where more than 80 per cent of its sales come 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 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 In 2019, Ultomiris had sales of $339 million Ultomiris is the first and only long-acting C5 supplement inhibitor administered every 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 were non-effective at all 11 endpoints industry predicts that Ultomiris will become the new standard for PNH clinical treatment Based on strong clinical data and differentiation characteristics, Ultomiris will account for the majority of the PNH market when it is marketed, including PNH patients with primary treatment and treated PNH patients from Soliris to Ultomiris, both of which are expected to generate combined sales of $5 billion by 2022 Evaluate Pharma, a pharmaceutical market research firm, predicts Ultomiris will have sales of $3.43 billion in 2024 Alexion is actively promoting the market penetration of Ultomiris, while also actively developing other ultomiris adaptations, including gMG, and the development of subcutaneous injection forms Ultomiris , 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 source: Alexion Announce Phases Study 3 of The Weekly Subcutance ULTOMIRIS ® (ravulyzumab-cwvz) Met Primary Endpoint .