April 14, 2024

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FDA approved Ultomiris for paroxysmal nocturnal hemoglobinuria (PNH)

New drugs for rare diseases! The U.S. FDA approved Ultomiris, a long-acting C5 complement inhibitor: treatment of paroxysmal nocturnal hemoglobinuria (PNH) in children and adolescents!



FDA approved Ultomiris for paroxysmal nocturnal hemoglobinuria (PNH).  Alexion Pharma recently announced that the U.S. Food and Drug Administration (FDA) has approved the expansion of the use of Ultomiris (ravulizumab-cwvz) to include paroxysmal nocturnal hemoglobinuria (PNH) children (one month and older) and Adolescent patients.

Ultomiris is a long-acting C5 complement inhibitor that can inhibit the C5 protein in the complement cascade of the human immune system, and can provide immediate, complete and sustained complement inhibition. Now, Ultomiris is the first and only drug approved by the FDA for children and adolescents with PNH.

Since it was first approved in 2018, Ultomiris has quickly become the standard treatment for adult patients with PNH in the United States. PNH is a blood disease characterized by complement-mediated destruction of red blood cells. It 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. The symptoms and complications of PNH are caused by the lack of regulation or control of the complement system, which is an important part of the human immune system.

Ultomiris is designed to inhibit the C5 protein in the terminal part of the complement cascade, while retaining the functions of other parts of the immune system to resist common pathogens and infections. Ultomiris reduces the risk of intravascular red blood cell destruction (intravascular hemolysis and thrombosis) by providing immediate, complete, and continuous terminal complement inhibition.

This approval is based on the results of a phase 3 clinical study. Data show that in children and adolescents under 18 years of age, Ultomiris effectively achieved complete C5 complement suppression during 26 weeks of treatment. In addition, Ultomiris did not report serious adverse events related to treatment, and no patients discontinued treatment or experienced breakthrough hemolysis during the initial evaluation period, which could lead to disabling or potentially fatal blood clots.

The efficacy and safety of Ultomiris in children and adolescents with PNH are consistent with the established characteristics of Ultomirs in clinical studies of adult PNH patients, and represent a wide range of PNH patients in a real clinical environment.

In December 2020, AstraZeneca announced the acquisition of Alexion in the form of $39 billion in cash and stock. Currently, the transaction is undergoing antitrust review in many countries and regions.

Alexion’s main products are two C5 complement inhibitors Soliris and Ultomiris, the latter is a long-acting version of the former. Both drugs work by inhibiting the C5 protein in the terminal part of the complement cascade. The complement cascade is part of the immune system, and its uncontrolled activation plays an important role in a variety of serious rare diseases and super rare diseases.

Soliris was first approved for marketing in 2007, and a variety of super rare diseases have been approved, including: paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), systemic myasthenia gravis (gMG), Neuromyelitis optica spectrum disorder (NMOSD). Ultomiris is an upgraded version of Soliris. It is a second-generation, long-acting C5 complement inhibitor. It was first approved for marketing at the end of 2018. The approved indications include: PNH and aHUS.

Soliris is one of the world’s best-selling rare disease drugs, with sales in 2020 reaching US$4.065 billion. Ultomiris’s sales in 2020 have reached 1.077 billion US dollars. The total sales of the two C5 inhibitors amounted to US$5.142 billion.


(source:internet, reference only)

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