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Myositis
Published in Jason Liebowitz, Philip Seo, David Hellmann, Michael Zeide, Clinical Innovation in Rheumatology, 2023
Other treatment options being investigated in clinical trials are ustekinumab (an anti-IL-12/IL-23 monoclonal antibody), complement inhibitors (ravulizumab), subcutaneous Ig, and intravenous infusion of allogeneic umbilical cord–lining stem cells (ULSC).
Ravulizumab for the treatment of myasthenia gravis
Published in Expert Opinion on Biological Therapy, 2023
Fiammetta Vanoli, Renato Mantegazza
Considering the great limitations of the current therapeutic algorithm of MG, in terms of unpredictable response and troublesome side effects, it is critical to shift toward a more targeted approach, where drugs are engineered based on the specific pathogenic mechanisms that underlie the disease. In this sense, ravulizumab, as well as other novel drugs currently under study as previously discussed, was conceived to act on a very specific and pivotal pathogenic system, the complement cascade. It is likely that the current therapeutic approach, characterized mainly by nonselective immunosuppression, will be replaced by newer drugs with a more precise mechanism of action, that should decrease side effects while guaranteeing a satisfactory clinical benefit. Hence, a possible new therapeutic approach could be to start ravulizumab either before or in association to conventional immunosuppressants. Ravulizumab also demonstrated to have a rapid clinical effect, as improvements in the MG-ADL and QMG scores were observed already within 1 week of treatment.
Quantifying the economic effects of ravulizumab versus eculizumab treatment in patients with atypical hemolytic uremic syndrome
Published in Journal of Medical Economics, 2022
Adrian R. Levy, Peter Chen, Karissa Johnston, Yan Wang, Evan Popoff, Ioannis Tomazos
Treatment costs associated with being on-treatment (drugs, administration, and meningococcal vaccination), discontinuation (monitoring), and relapses (acute hospital events and management) were included in the CMM (Supplementary Table S3), in addition to the lost productivity costs associated with eculizumab or ravulizumab treatment multiplied by the number of treatment administration cycles per calendar year. Post-relapse costs included the cost of restarting treatment with eculizumab or ravulizumab following relapse and the treatment costs from the point of this reinitiation. Based on eculizumab clinical trial outcomes with long-term follow-up, adult and pediatric patients were assumed to first discontinue treatment after 2.3 and 1.3 years, respectively. Patients who receive treatment with eculizumab or ravulizumab are required to be vaccinated against meningococcal infection (as per product prescribing information) and the rate of meningococcal infections in patients with aHUS treated with eculizumab or ravlizumab have been reported to be low23–26. It was therefore assumed that the risk of meningococcal infection would be low and hence the cost of complications of meningococcal infections were not included in the model.
Role of complement, anti-complement therapeutics, and other targeted immunotherapies in myasthenia gravis
Published in Expert Review of Clinical Immunology, 2022
Like eculizumab, Ravulizumab (Ultomiris) also binds with high affinity to C5 preventing the generation of complement activation products C5a and C5b-9 [23]. In contrast to eculizumab however, Ravulizumab has increased half-life causing a more sustained complement inhibition requiring less frequent dosing (once every 8 weeks, instead of once every 2 weeks as for eculizumab) [38]. Ravulizumab has gained FDA approval for Paroxysmal Nocturnal Hemoglobinuria (PNH). Released data from a phase III FDA-approved study in 175 MG patients showed treatment efficacy with significant change in MG-ADL score from baseline to 26 weeks with meaningful and statistically significant improvements in QMG score (https://clinicaltrials.gov/ct2/show/NCT03920293). On this basis, the drug was just approved by the FDA (April 2022) for the treatment of g MG as the first long acting C5 inhibitor. Ultomiris, following a loading dose, is given intravenously every 8 weeks and is expected to benefit a broader range of patients offering another option to MG patients potentially even those with milder symptoms.