AstraZeneca today 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 ³Ô¹ÏÍøÕ¾ 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.
Notes
PNH
PNH is a rare, chronic, progressive and potentially life-threatening blood disorder. It is characterised by red blood cell destruction within blood vessels (also known as intravascular haemolysis) and white blood cell and platelet activation, which can result in thrombosis (blood clots).1-3
PNH is caused by an acquired genetic mutation that may happen any time after birth and results in the production of abnormal blood cells that are missing important protective blood cell surface proteins. These missing proteins enable the complement system, which is part of the immune system and is essential to the body’s defence against infection, to ‘attack’ and destroy or activate these abnormal blood cells.1 Living with PNH can be debilitating, and signs and symptoms may include blood clots, abdominal pain, difficulty swallowing, erectile dysfunction, shortness of breath, excessive fatigue, anaemia and dark-coloured urine.1,4,5
aHUS
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. aHUS occurs when the complement system-a part of the body’s immune system-over-responds, leading the body to attack its own healthy cells.6 aHUS can cause sudden organ failure or a slow loss of function over time-potentially resulting in the need for a transplant, and in some cases, death. aHUS affects both adults and children, and many patients present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit.7 The prognosis of aHUS can be poor in many cases, so a timely and accurate diagnosis-in addition to treatment-is critical to improving patient outcomes. Available tests can help distinguish aHUS from other haemolytic diseases with similar symptoms.6
Soliris
Soliris (eculizumab) is a first-in-class C5 complement inhibitor. The medication works by inhibiting 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.
Soliris is approved in the US, EU and Japan for the treatment of PNH, aHUS, certain adults with gMG and certain adults with NMOSD.
Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related haemolytic uraemic syndrome.
Alexion
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit and follow the Company on Twitter .
