The world's first long-acting C5 inhibitor! EU approves Ultomiris 100mg/mL high concentration preparation: 60% reduction in infusion time!

November 21, 2020 // -- Alexion is a biopharmaceutical company dedicated to the development of new drugs for rare diseases.
recently, the company announced that the European Commission (EC) has approved Ultomiris (ravulizumab-cwvz) 100mg/mL intravenous (IV) preparations for the treatment of two super-rare diseases: bursts of phemoglobinuria (PNH) and atypical hemolytic uremia syndrome (aHUS).
the United States, Ultomiris 100mg/mL IV preparations were approved in October this year: (1) for adult ≥ patients with PNH;
Ultomiris is the first and only long-acting C5 inhibitor: it can be given every 8 weeks in children and adults weighing ≥20 kg, and once every 4 weeks in children weighing <20 kg.
Ultomiris 100mg/mL preparation represents an advance in clinical treatment experience for patients with aHUS and PNH, as Ultomiris 100mg/mL preparations reduce average annual infusion time by approximately 60% compared to Ultomiris 10mg/mL preparations, while having a comparable safety and ability.
Ultomiris 100mg/mL preparation, most patients receive treatment for no more than 6 hours per year.
this approval is based on a comprehensive chemical, manufacturing and control submission and a complementary clinical data set that shows that the safety, pharmacogenics and immunogenicity of Ultomiris 10mg/mL and Ultomiris 100mg/mL administration therapy are comparable.
, the data set showed no correlation between the effectiveness measurements of the average lactic acid dehydrogenase (LDH) levels of the two formulations.
Depending on the weight, the new proposed formula requires 0.4 to 1.3 hours (25 to 75 minutes) of infusion time, which is approximately 60 per cent shorter than the current 10 mg/ml intravenous infusion formula (depending on weight, infusion time is 1.3 to 3.3 hours (77 to 194 minutes).
Ultomiris has provided patients with greater flexibility, and this new formula is another step forward in reducing the overall burden of treatment," said Professor Alexander Röth, Department of Hematology and Stem Cell Transplantation, Essen Hospital, University of Essen, Germany.
the new formula, patients receive significantly less time each year while their safety and efficacy will be comparable to that of the original formula, which has the potential to have a significant impact on their lives.
" Ultomiris is the first and only long-acting C5 supplement inhibitor to receive regulatory approval, based on strong efficacy and differentiation characteristics, and pharmaceutical market research agency Evaluate Pharma had forecast sales of $3.43 billion in 2024.
, Alexion is continuing its Ultomiris innovation with the goal of improving the patient experience.
, after completing its ongoing Phase 3 clinical study and collecting 12 months of safety data, plans to submit regulatory filings for Ultomiris' combination of Ultomiris preparations and devices for treatment of PNH and aHUS to U.S. and European Union regulators in the third quarter of 2021, an innovative product that allows patients to self-administration at home and significantly reduces the time it takes to do so, reducing Ultomiris treatment to about 10 minutes.
PNH is a blood disease characterized by complement-mediated red blood cell destruction that can cause a wide range of debilitating symptoms and complications, including thrombosis that can occur throughout the body and lead to organ damage and premature death.
aHUS can cause progressive damage to vital organs (mainly the kidneys) by damaging blood vessel walls and blood clots.
affect both adults and children and are in critical condition, often requiring supportive care in intensive care, including dialysis.
in many cases, aHUS and PNH have poor prognosis, so timely and accurate diagnosis and appropriate treatment are key to improving patient prognosis.
the original source: New development of Alexion's Ultomiris Ok'd in Europe for rare blood disease and kidney and disorder