paroxysmal nocturnal hemoglobinuria treatment market

#Paroxysmal nocturnal hemoglobinuria treatment market trial#

Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study. Kulasekararaj AG, Hill A, Rottinghaus ST, Langemeijer S, Wells R, Gonzalez-Fernandez FA, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Lee JW, de Sicre FF, Wong Lee Lee L, Pessoa V, Gualandro S, Füreder W, et al. Ravulizumab: a novel C5 inhibitor for the treatment of paroxysmal nocturnal hemoglobinuria. Ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Design and preclinical characterization of ALXN1210: a novel anti-C5 antibody with extended duration of action. Sheridan D, Yu ZX, Zhang Y, Patel R, Sun F, Lasaro MA, et al. First in human single-ascending dose study: safety, biomarker, pharmacokinetics and exposure-response relationships of ALXN1210, a humanized monoclonal antibody to C5, with marked half-life extension and potential for significantly longer dosing intervals. Sahelijo L, Mujeebuddin A, Mitchell D, Larouche R, Yu Z-X, Zhang Y, et al. Ravulizumab (ALXN1210) in patients with paroxysmal nocturnal hemoglobinuria: results of 2 phase 1b/2 studies. Röth A, Rottinghaus ST, Hill A, Bachman ES, Kim JS, Schrezenmeier H, et al. Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria. Peipert JD, Kulasekararaj AG, Gaya A, Langemeijer SMC, Yount S, Gonzalez-Fernandez FA, et al. Results from multinational phase 3 studies of ravulizumab (ALXN1210) versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria: subgroup analysis of Japanese patients. Ishiyama K, Nakao S, Usuki K, Yonemura Y, Ikezoe T, Uchiyama M, et al.

Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS clinical trial. Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, et al. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. Impact of eculizumab treatment on paroxysmal nocturnal hemoglobinuria: a treatment versus no-treatment study. Loschi M, Porcher R, Barraco F, Terriou L, Mohty M, de Guibert S, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. Hillmen P, Young NS, Schubert J, Brodsky RA, Socié G, Muus P, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Hillmen P, Muus P, Dührsen U, Risitano AM, Schubert J, Luzzatto L, et al. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. 2007 137:181–92.īrodsky RA, Young NS, Antonioli E, Risitano AM, Schrezenmeier H, Schubert J, et al. Recent developments in the understanding and management of paroxysmal nocturnal haemoglobinuria. The clinical sequelae of intravascular hemolysis and extracellular plasma hemoglobin: a novel mechanism of human disease. Rother RP, Bell L, Hillmen P, Gladwin MT. Hill A, DeZern AE, Kinoshita T, Brodsky RA. Further follow-up data from this PMS will help to elucidate the long-term clinical safety and effectiveness of ravulizumab for treating PNH.īrodsky RA. However, transfusion independence was less likely in patients with bone marrow failure.

#Paroxysmal nocturnal hemoglobinuria treatment market trial#

These data for Japanese patients with PNH who were naïve to complement inhibitors and patients who switched from eculizumab show that the safety and effectiveness of ravulizumab are consistent with the published clinical trial data. In complement inhibitor-naïve patients, LDH decreased significantly and Hb increased significantly over 26 weeks of ravulizumab treatment. In patients who switched from eculizumab, lactate dehydrogenase (LDH) and hemoglobin (Hb) levels were maintained over 26 weeks of ravulizumab treatment. The incidence of serious AEs was 36.93/100 patient-years. The two most frequent AEs were anemia and pyrexia (each 3.01/100 patient-years). Over a median follow-up of 74.4 weeks, 193 adverse events (AEs) were reported in 66/218 patients (30.3% incidence 72.73/100 patient-years). We report safety data obtained through to December 2021 for 218 patients and effectiveness data for 194 patients (182 switched from eculizumab and 12 complement inhibitor-naïve). Post-marketing surveillance (PMS) was implemented following its approval in September 2019 in Japan. Ravulizumab is a long-acting C5 inhibitor available for treating paroxysmal nocturnal hemoglobinuria (PNH).