KUALA LUMPUR, March 19 — AstraZeneca has made its rare disease treatment Soliris available in Malaysia, marking a significant advancement in treatment options for patients with rare neurological autoimmune diseases and severe blood disorders.
In a statement, the global biopharmaceutical company said Soliris has contributed to improved clinical outcomes in treating conditions including generalised myasthenia gravis (gMG), neuromyelitis optica spectrum disorder (NMOSD), atypical haemolytic uraemic syndrome (aHUS) and paroxysmal nocturnal haemoglobinuria (PNH).
The therapy targets the complement system, a key component of the immune response, by binding and blocking the C5 protein within the terminal complement pathway to help control excessive immune activity associated with complement-mediated diseases.
AstraZeneca Malaysia Country President Svetlana Yanchuk said the availability of Soliris reflects the company’s continued commitment to supporting patients living with rare diseases.
“With Soliris we can advance our efforts in Malaysia to transform the lives of persons living with rare diseases as well as their families in the country and help them fully live their meaningful lives.
“Research and development into rare diseases require continuous innovation to deliver meaningful medicines for patients. AstraZeneca, through our subsidiary Alexion, acquired in 2021 to harness insights into the immune system’s complement pathway to develop novel, life-changing treatments for patients with complement-mediated diseases.
“Moving forward, we remain committed to advancing our legacy and leadership in this area to advocate for the rare disease community. In fact, efforts are in place to introduce next-generation treatments to enhance the care of those living with rare diseases,” she said.
The company noted that rare diseases are often difficult to recognise and diagnose, with patients frequently experiencing delays, repeated testing, and misdiagnosis before receiving accurate treatment.
Globally, about 300 million people are living with rare diseases, many of which are serious, long-lasting and may worsen over time. In Malaysia, 491 rare diseases had been recorded as of 2020, although many cases remain underdiagnosed.
Consultant haematologist at Hospital Umum Sarawak Dr Chew Lee Ping said aHUS and PNH are linked to immune system abnormalities affecting blood.
“aHUS is a rare disease due to immune dysregulation of the blood which can cause damage to the kidneys while PNH is a rare, chronic, progressive and potentially life-threatening blood disorder that causes red blood cells to break down prematurely, resulting in blood clots,” she said.
Consultant neurologist at Sunway Medical Centre Dr Hiew Fu Liong said gMG and NMOSD affect the nervous system and can lead to serious complications.
“gMG is a chronic autoimmune neuromuscular disease which begins with muscle weakness around the eyeballs and eyelids and leads to weakness in the head, neck, limb and respiratory muscles,” he said.
He added that NMOSD is a rare, debilitating disorder that affects the central nervous system, particularly the optic nerves and spinal cord, and can eventually cause vision loss, paralysis and other neurological complications.
