AstraZeneca bringing rare disease medicine to patients in China
Soliris (eculizumab) is used for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) in adults and children.
PNH is a rare, chronic, progressive, and potentially life-threatening blood disorder. aHUS is a rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots
Soliris can reduce disease symptoms and prevent the dysregulated complement system from causing further damage, said the company. The drug is designed to inhibit the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the terminal complement cascade over-responds, leading the body to attack its own healthy cells. Soliris is administered intravenously every two weeks, following an introductory dosing period.
The drug is approved in the US, the EU, and Japan for the treatment of PNH, aHUS, certain adults with gMG and certain adults with neuromyelitis optica spectrum disorder (NMOSD).
In addition, AstraZeneca reported that the National Medicine Products Administration of China (NMPA) has accepted its supplementary application for Soliris for the treatment of adults with refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AchR) antibody positive.
Clinical trials
The NMPA has also granted AstraZeneca approval to start ongoing, global clinical trials in that country for investigational therapies under evaluation for the treatment of lupus nephritis (LN), immunoglobulin A nephropathy (IgAN) and light chain (AL) amyloidosis.
Patients and families living with rare diseases in China currently have limited treatment options or no available treatment at all, noted Leon Wang, executive VP, international and China president of AstraZeneca.
In the future, he said, the company aims to introduce more innovative medicines in China, targeting the complement system and beyond, for the treatment of rare diseases including PNH, aHUS, gMG, NMOSD, hypophosphatasia, IgAN, LN and amyloidosis.
AstraZeneca established a rare disease business unit in China in September 2021.
