Ravulizumab

Generic Name
Ravulizumab
Brand Names
Ultomiris
Drug Type
Biotech
Chemical Formula
-
CAS Number
1803171-55-2
Unique Ingredient Identifier
C3VX249T6L
Background

Ravulizumab is a potent and selective complement 5 (C5) inhibitor. It is a humanized monoclonal IgG2/4 kappa antibody produced in Chinese hamster ovary (CHO) cells. Ravulizumab was engineered from eculizumab, another complement inhibitor, to increase the duration of action and reduce the frequency of drug administration. It works by blocking terminal complem...

Indication

Ravulizumab is indicated for the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH).
...

Associated Conditions
Atypical Hemolytic Uremic Syndrome (aHUS), Generalized Myasthenia Gravis, Paroxysmal Nocturnal Haemoglobinuria (PNH), Thrombotic Microangiopathies
Associated Therapies
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nyulangone.org
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Gene-Edited Pig Kidney Gives Living Donor New Lease on Life

An Alabama woman, Towana Looney, received a gene-edited pig kidney transplant at NYU Langone Health, marking a significant breakthrough in xenotransplantation. Looney, who had been on dialysis for years due to kidney failure, is now the healthiest she's been in eight years. The procedure involved 10 gene edits to the pig kidney, aiming to reduce rejection and improve compatibility. This is the third time a gene-edited pig kidney has been transplanted into a living human, and Looney is the first to receive a kidney with 10 gene edits.
ahusnews.com
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Platelet counts may predict long-term Ultomiris response in aHUS: Study

Early platelet count changes on Ultomiris predict long-term kidney response in aHUS patients, with 85% of those achieving renal recovery showing normal platelet counts within 15 days.

Ultomiris helped most gMG patients in Phase 3 trial over half year

More than half of adults with generalized myasthenia gravis (gMG) show a clinically meaningful response to Ultomiris (ravulizumab-cwvz) within about two weeks of the first dose, with over 80% experiencing meaningful symptom reduction after six months of regular treatment every eight weeks. The study suggests that a longer 'trial of therapy' may be required before considering Ultomiris ineffective.
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