There are currently 319 ongoing clinical trials involving Paroxysmal Nocturnal Hemoglobinuria
Of the 319 trials,219 trials are in Phase III
Furthermore, 61 trials are in Phase II
The global pharmaceutical industry is steadily developing new drugs for Paroxysmal Nocturnal Hemoglobinuria, a Hematological Disorder. The largest number of ongoing clinical trials for Paroxysmal Nocturnal Hemoglobinuria are conducted in North America and Europe. Asia-Pacific and South and Central America are among some of the other prominent regions engaged in Paroxysmal Nocturnal Hemoglobinuria-related drug trials.
Alexion Pharmaceuticals Inc : The leading ongoing Paroxysmal Nocturnal Hemoglobinuria related clinical trial sponsor
Alexion Pharmaceuticals Inc is the top sponsor for Paroxysmal Nocturnal Hemoglobinuria-related ongoing clinical trials.
Novartis AG, Regeneron Pharmaceuticals Inc, F. Hoffmann-La Roche Ltd and BioCryst Pharmaceuticals Inc are among other notable clinical trial sponsors involved in Paroxysmal Nocturnal Hemoglobinuria. A clinical trial sponsor can be a Company, Government, Individual, or Institution.
Marketed Drugs involving Paroxysmal Nocturnal Hemoglobinuria
Ravulizumab (Ultomiris), Pegcetacoplan (Empaveli, Aspaveli) and Eculizumab (Soliris) are among the key marketed drugs involving Paroxysmal Nocturnal Hemoglobinuria.
Ravulizumab (Ultomiris) is a humanized monoclonal antibody. It functions via Complement C5 (C3 And PZP Like Alpha 2 Macroglobulin Domain Containing Protein 4 or C5) Inhibitor mechanism of action. Ravulizumab is formulated as solution and concentrate solution (long acting) for intravenous route of administration. Ravulizumab was first approved in 2018 and is marketed globally in the US, UK, Australia, Japan, Germany, and France by Alexion Pharmaceuticals Inc and its subsidiaries.
Pegcetacoplan (Empaveli, Aspaveli) is a synthetic peptide acts as a complement inhibitor. It functions via Complement C3 (C3 And PZP Like Alpha 2 Macroglobulin Domain Containing Protein 1 or C3) Inhibitor mechanism of action. Pegcetacoplan is formulated as injectable solution for subcutaneous route of administration. Pegcetacoplan was first approved in 2021 and is marketed globally in the US, Belgium and EU by Apellis Pharmaceuticals Inc and Swedish Orphan Biovitrum AB.
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