Soliris is approved for the treatment of two indications – paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Both aHUS and PNH sections of the EU label have been updated.
The AHUS section of Soliris’ EU label now includes new efficacy data that specify that longer-term treatment with Soliris is associated with a greater proportion of patients achieving clinically significant benefits. The label update also includes new information on the risks associated with treatment discontinuation.
The PNH section of Soliris’ EU label now indicates that patients with PNH without a history of transfusions will be able to benefit from treatment with Soliris. The updated label also states evidence of clinical benefit in patients with high disease activity as defined by elevated hemolysis and the presence of related clinical symptoms including fatigue and hemoglobinuria.
The EC approval for Soliris label updates did not come as a surprise considering the European Medicines Agency’s Committee for Medicinal Products for Human Use had given a positive opinion regarding the update of the therapeutic indication for Soliris earlier this year.
We believe that EU label updates for Soliris could increase demand for Soliris in both PNH and aHUS patients. Meanwhile, Alexion is working to expand the Soliris label. The company is evaluating Soliris for several additional indications including delayed kidney transplant graft function (phase III), neuromyelitis optica (phase III) and myasthenia gravis (phase III).