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Alexion : EU Approves New Formulation Of Ultomiris For Two Rare Diseases Treatment

Alexion Pharmaceuticals Inc. (ALXN) said Friday that the European Commission has approved the new 100 mg/mL intravenous formulation of Ultomiris or ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria or PNH and atypical hemolytic uremic syndrome or aHUS.

The approval was based on a comprehensive chemistry, manufacturing and control submission and a supplementary clinical data set showing that the safety, pharmacokinetics and immunogenicity following administration of Ultomiris 10 mg/mL and Ultomiris 100 mg/mL were comparable.

Ultomiris 100 mg/mL is an advancement in the treatment experience for patients with aHUS and PNH by reducing average annual infusion times by about 60 percent compared to Ultomiris 10 mg/mL, while delivering comparable safety and efficacy.

The company said it plans to submit regulatory filings in the U.S. and EU in the third quarter of 2021 for an Ultomiris subcutaneous formulation and device combination for PNH and aHUS that can be self-administered at home. It is pending completion of the ongoing Phase 3 study and collection of 12-month safety data.

In addition, the Ultomiris clinical development programs present an opportunity to expand treatment for rare diseases across hematology, nephrology, neurology, and for the treatment of severe COVID-19, with seven Phase 3 programs that are ongoing or have planned clinical trial initiations in 2020.

PNH is a serious ultra-rare blood disorder with devastating consequences. It is characterized by the destruction of red blood cells, which is also referred to as hemolysis.

aHUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots.

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