November 11, 2022: “AstraZeneca announced the availability of the first rare disease therapy from Alexion, AstraZeneca’s Rare Disease group, in China.
The availability of Soliris (eculizumab) for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) in adults and children in China represents a significant expansion of the company’s global footprint in rare disease, following the acquisition of Alexion Pharmaceuticals, Inc. in 2021.
In addition to announcing the availability of Soliris, AstraZeneca announced that the National Medicine Products Administration of China (NMPA) has accepted its supplementary application for Soliris for the treatment of adults with refractory generalised myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AchR) antibody positive.
The NMPA has also granted AstraZeneca approval to start ongoing, global clinical trials in China for investigational therapies being evaluated for the treatment of lupus nephritis (LN), immunoglobulin A nephropathy (IgAN) and light chain (AL) amyloidosis.
Marc Dunoyer, Chief Executive Officer, Alexion, said: “These milestones represent significant progress against our commitment to expand access to our rare disease medicines globally, including the introduction of innovative rare disease therapies in China.
Expanding access to our medicines is possible because of the combined strength of Alexion’s rare disease expertise and AstraZeneca’s vast global footprint, and we look forward to continue bringing our therapies to people living with rare diseases around the world in the future.”
Leon Wang, Executive Vice President, International and China President of AstraZeneca, said: “This milestone reflects our ambition to bring transformative, rare disease medicines to the significant number of patients and families living with rare diseases in China who currently have limited treatment options or no available treatment at all.
We look forward to advancing our commitment in China and aim to bring additional, innovative therapies to even more rare disease patients across the country.”
As the first C5 complement inhibitor, Soliris works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. Soliris is approved for multiple indications in many countries around the world.
The availability of Soliris in China provides people living with PNH or aHUS with a new treatment option that can reduce disease symptoms and prevent the dysregulated complement system from causing further damage.
AstraZeneca established a rare disease business unit in China in September 2021.
In the future, 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, neuromyelitis optica spectrum disorder (NMOSD), hypophosphatasia, IgAN, LN and amyloidosis.”
