– New 100 mg/mL formulation will reduce infusion time by approximately 60 percent, lessening the burden on patients –
– ULTOMIRIS is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) –
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved ULTOMIRIS® (ravulizumab-cwvz) 100 mg/mL formulation for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) and for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy for adult and pediatric (one month of age and older) patients. ULTOMIRIS 100 mg/mL is an advancement in the treatment experience for patients with aHUS and PNH, as it reduces 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.
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Image of ULTOMIRIS® (ravulizumab-cwvz) 100 mg/mL vials (3 mL and 11mL) (Photo: Business Wire)
"We have seen the significant clinical benefits ULTOMIRIS has demonstrated for patients, through its complete C5 inhibition and sustained efficacy," said Jamile Shammo, M.D., FASCP, FACP, Professor of Medicine and Pathology, Department of Internal Medicine, Rush University Medical Center. "Based on the scientific evidence presented, the advanced formulation demonstrated comparable safety and efficacy to the original formulation, with the additional benefit of significantly shorter infusion times. This reduced treatment burden is important to consider for patients, as it has the potential to make a meaningful difference in their lives."
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, atypical HUS 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 diagnosis—in addition to appropriate treatment—is critical to improving patient outcomes.
"ULTOMIRIS is the market leader for the treatment of PNH and is emerging as the standard of care for the treatment of aHUS," said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. "The approval of ULTOMIRIS 100 mg/mL represents the next step in advancing patient care for the PNH and aHUS communities, as it reduces infusion times for patients and decreases the number of vials that need to be stored and prepared for the majority of patients' infusions, allowing healthcare providers more time to focus on the patient. With ULTOMIRIS 100 mg/mL, most patients will spend six hours or less each year receiving treatment, giving them more time to do what they enjoy, which we believe will provide a meaningful benefit to their quality of life."
Alexion plans to make ULTOMIRIS 100 mg/mL available within a few days and has comprehensive training plans to educate all stakeholders about the new formulation. ULTOMIRIS 10 mg/mL will continue to be available until mid-2021, at which time Alexion will remove ULTOMIRIS 10 mg/mL from the market, as communicated to the FDA. The transition period is intended to provide a seamless conversion to the new formulation without interruption to patients' infusion schedules.
Regulatory filings for marketing authorizations of the 100 mg/mL formulation of ULTOMIRIS are under review with regulators in the European Union (EU) and Japan. The Committee for Medicinal Products for Human Use (CHMP) has recently adopted a positive opinion, recommending marketing authorization of ULTOMIRIS 100 mg/mL in the European Union, and we anticipate receiving a decision from the European Commission in November.
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.