Danicopan

Following achievements in Asia, Roche's PNH treatment drug PIASKY (crovalimab) received its first FDA approval. This approval has spiked the competition between Roche and current market leader AstraZeneca. Additionally, the other pharma companies also geared up to launch their respective lead assets. LAS VEGAS, Sept. 2, 2024 /PRNewswire/ -- Paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder, shows a triad of features: intravascular hemolysis, thromboembolic events, and cytopenia. Manifestations vary among patients, making classification based on typical presentations challenging due to the disease's unpredictable nature. In 2023, the Paroxysmal Nocturnal Hemoglobinuria diagnosed prevalent cases were ~12,000 cases in the 7MM, which might reach ~13,000 cases by 2034. In the 7MM, the highest number of paroxysmal nocturnal hemoglobinuria diagnosed prevalent cases were observed in the US. Until 2007, PNH was a devastating disease without treatment for hemolysis and thrombosis, the leading cause of death in patients. However, the last decade saw a revolutionary shift with the advent of the anti-C5 agent eculizumab. It significantly reduced hemolysis, decreased transfusion dependency, and, crucially, lowered the thrombosis rate. The disease-modifying therapeutic strategy for paroxysmal nocturnal hemoglobinuria includes complement inhibition therapy, with drugs like SOLIRIS, ULTOMIRIS, and EMPAVELI approved by the FDA, and considered the gold standard. SOLIRIS, the pioneering therapy for PNH, was succeeded by ULTOMIRIS, both developed by Alexion Pharmaceuticals, offering C5 inhibitors as essential therapeutic options. Recent approvals of factor B and D inhibitors like FABHALTA (iptacopan) in the US and VOYDEYA (danicopan) in Japan aim to offer improved treatment with fewer side effects. Learn more about the FDA-approved paroxysmal nocturnal hemoglobinuria drugs @ Drugs for Paroxysmal Nocturnal Hemoglobinuria Treatment The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended crovalimab for treating PNH in adults and adolescents aged 12 and above in June 2024, based on findings from the global Phase III COMMODORE 2 clinical trial. The FDA accepted a biologics license application for crovalimab in September 2023 and subsequently approved it in June 2024 for treating PNH in adults and adolescents aged 13 years and older. Genentech, a subsidiary of Roche, has made the drug available in the US. In Japan, Chugai Pharmaceutical submitted a new drug application for PiaSky to the Japan Ministry of Health, Labour, and Welfare (MHLW) in June 2023. The drug was approved in March 2024 and launched in May 2024 for patients with PNH aged 12 years and above. In February 2024, China's National Medical Products Administration (NMPA) approved crovalimab for treating PNH in adults and adolescents aged 12 years and older who have not previously received complement inhibitor treatment, following a priority review that began in August 2022. PiaSky is also undergoing review by other regulatory authorities, including those in Europe and Taiwan. To know more about paroxysmal nocturnal hemoglobinuria treatment options, visit @ New Treatment for Paroxysmal Nocturnal Hemoglobinuria The paroxysmal nocturnal hemoglobinuria market is crowded with so many companies working in the domain. Emerging PNH therapies include Pozelimab (REGN3918) + Cemdisiran, OMS906, NM8074 (ruxoprubart), and others, reflecting a dynamic evolution in treatment strategies. Discover which therapies are expected to grab major paroxysmal nocturnal hemoglobinuria market share @ Paroxysmal Nocturnal Hemoglobinuria Market Report Pozelimab is a novel, fully human monoclonal antibody currently under investigation. It aims to inhibit complement factor C5, thereby preventing the destruction of red blood cells (hemolysis) responsible for symptoms in conditions like Paroxysmal Nocturnal Haemoglobinuria (PNH) and other diseases influenced by complement pathway activity. This IgG4 antibody binds tightly to both wild-type and variant forms of human C5, effectively blocking its function. In its ongoing development, pozelimab is also being studied in combination with Alnylam's cemdisiran, a siRNA C5 inhibitor. This investigational combination therapy is aimed at treating various complement-mediated disorders, including PNH and myasthenia gravis. OMS-906 is currently being developed to treat paroxysmal nocturnal hemoglobinuria (PNH), complement 3 glomerulopathy (C3G), idiopathic immune complex-mediated glomerulonephritis (ICGN), and arthritis. It can be administered either subcutaneously or intravenously. This drug candidate is a monoclonal antibody designed to target mannan-binding lectin serine protease 3 (MASP-3) to exert its therapeutic effects. Discover more about drugs for paroxysmal nocturnal hemoglobinuria in development @ Paroxysmal Nocturnal Hemoglobinuria Clinical Trials The anticipated launch of these emerging therapies for paroxysmal nocturnal hemoglobinuria are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the paroxysmal nocturnal hemoglobinuria market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that in 2023, the total paroxysmal nocturnal hemoglobinuria market size in the 7MM was USD ~1.4 billion, which is expected to reach USD ~2.5 billion by 2034. This growth can be attributed to the advances in disease mechanisms that drive new diagnostics and therapeutics, fueling drug development. Additionally, the PNH market foresees positive growth, propelled by increasing cases and upcoming therapies in the forecast period. DelveInsight's latest published market report titled as Paroxysmal Nocturnal Hemoglobinuria Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the paroxysmal nocturnal hemoglobinuria country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The paroxysmal nocturnal hemoglobinuria market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of PNH Gender-specific Cases of PNH The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM paroxysmal nocturnal hemoglobinuria market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this paroxysmal nocturnal hemoglobinuria market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the paroxysmal nocturnal hemoglobinuria market. Also, stay abreast of the mitigating factors to improve your market position in the paroxysmal nocturnal hemoglobinuria therapeutic space. Related Reports Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast Paroxysmal Nocturnal Hemoglobinuria Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted paroxysmal nocturnal hemoglobinuria epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Paroxysmal Nocturnal Hemoglobinuria Pipeline Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key paroxysmal nocturnal hemoglobinuria companies, including Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, among others. Aplastic Anemia Market Aplastic Anemia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key aplastic anemia companies, including Pfizer, BioLineRx Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, among others. Aplastic Anemia Pipeline Aplastic Anemia Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key aplastic anemia companies, including Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

The U.S. FDA approved Amgen's Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to AstraZeneca's Soliris (eculizumab) for treating two rare diseases. Bkemv is now approved for reducing hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH) and inhibiting complement-mediated thrombotic microangiopathy in patients with atypical hemolytic uremic syndrome (aHUS). The approvals align with the indications currently approved for Soliris, which was first approved in 2007 . PNH and aHUS are characterized by the breakdown of red blood cells. PNH results in anemia, blood clots, low blood cell counts, and dark urine, while aHUS results in anemia, low platelets, and kidney failure. Bkemv, a monoclonal antibody, binds to the complement C5 protein, inhibiting the complement system and preventing the breakdown of red blood cells in PNH and aHUS patients. Bkemv is the 53rd approved biosimilar in the U.S., with 13 approved as interchangeable biosimilars. An interchangeable biosimilar can be substituted for the reference product without consulting the prescriber, subject to state pharmacy laws, similar to generic drug substitution for brand-name drugs. Recently, the FDA also approved AstraZeneca's voydeya as an add-on to the standard-of-care — Ultomiris or Soliris — to address the needs of the approximately 10-20% of patients with PNH who experienced clinically significant extravascular hemolysis (EVH) while treated with a C5 inhibitor. For some patients, EVH, which is the destruction of red blood cells outside of the blood vessels, resulted in continued symptoms of anemia and may require blood transfusions.