Benzinga | Sep 21, 2020 05:31AM EDT

Alexion Announced CHMP Positive Opinion for New Advanced Formulation of ULTOMIRIS with Significantly Reduced Infusion Time

Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending marketing authorization in the European Union for a new 100 mg/mL intravenous (IV) advanced formulation of ULTOMIRIS(r) (ravulizumab). ULTOMIRIS is the first and only long-acting C5 inhibitor administered every eight weeks for the treatment of two ultra-rare diseasesparoxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). ULTOMIRIS 100 mg/mL would constitute an advancement in the treatment experience for patients with aHUS and PNH by reducing average annual infusion times by approximately 60 percent compared to ULTOMIRIS 10 mg/mL while delivering comparable safety and efficacy. With ULTOMIRIS 100 mg/mL, most patients will spend six hours or less a year receiving treatment.

"ULTOMIRIS is anticipated to be the new standard of care for people with PNH and aHUS and the advanced formulation would reduce the time they spend receiving their infusion. Lessening the overall burden on healthcare systems, especially at a time when many are already under strain due to the pandemic, is an important advance," said Professor Rth, Department of Hematology and Stem Cell Transplantation, University Hospital Essen, Essen, Germany.

PNH is a blood disorder characterized by complement-mediated destruction of the red blood cells that can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death. Atypical HUS can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. Affecting both adults and children, aHUS patients can present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of both aHUS and PNH can be poor in many cases, so a timely and accurate diagnosisin addition to appropriate treatmentis critical to improving patient outcomes.

"The advanced formulation of ULTOMIRIS marks continued progress in Alexion's ongoing efforts to innovate for patients, and we expect it will represent yet another step in refining the treatment schedule for patients," said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. "Importantly, it would mean that most patients would spend only six hours or less a year receiving treatment, allowing them to focus on other aspects of their lives."

The CHMP positive opinion is based on a comprehensive chemistry, manufacturing and control (CMC) submission and a supplementary clinical data set showing that the safety, pharmacokinetics and immunogenicity following administration of ULTOMIRIS 10 mg/mL and ULTOMIRIS 100mg/mL were comparable. Similarly, the data set showed no notable effect in the efficacy measure of mean lactate dehydrogenase (LDH) levels across the two formulations. The new proposed formulation requires an infusion time of 0.4 to 1.3 hours (25 to 75 minutes) depending on body weight, reducing the infusion time by more than half compared with the currently available 10mg/mL IV formulation, which ranges from 1.3 to 3.3 hours (77 to 194 minutes) depending on body weight.

The European Commission typically reaches a decision on approval in approximately two months of the CHMP recommendation.

Alexion continues to innovate with ULTOMIRIS, with the goal of improving the patient experience. We plan 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, pending completion of the ongoing Phase 3 study and collection of 12-month safety data. In addition, the collective ULTOMIRIS clinical development programs present an opportunity to expand the treated patient populations 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.