Alexion, AstraZeneca Rare Disease reaches an agreement with the pan-Canadian Pharmaceutical Alliance (pCPA) for Ultomiris for the treatment of adults with neuromyelitis optica spectrum disorder (NMOSD) and adults with generalized myasthenia gravis (gMG)

The agreement is a critical step in ensuring adult patients living with NMOSD and gMG have public access to Ultomiris

MISSISSAUGA, ON, Aug. 7, 2025 /CNW/ - Alexion Pharma Canada Corp., AstraZeneca's Rare Disease group, has entered into a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for Ultomiris (ravulizumab) for the treatment of adult patients with anti-aquaporin 4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD) and adult patients with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) generalized myasthenia gravis (gMG). With the agreement in place with the pCPA, individual provinces and territories may now initiate the process to list Ultomiris on their formularies, the timing of which will vary by province and territory. 

NMOSD is a rare and debilitating autoimmune disease that affects the central nervous system (CNS), including the spine and optic nerves.^1-4 Most people living with NMOSD experience unpredictable relapses, characterised by a new onset of neurologic symptoms or worsening of existing neurologic symptoms, which tend to be severe and recurrent and may result in permanent disability.^4,5

gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.⁶ Those living with gMG may initially experience slurred speech, double vision, droopy eyelids and weakness, with symptoms becoming more severe as the disease progresses, including extreme fatigue, difficulty swallowing, choking and respiratory failure.^7-8

"NMOSD is a rare neurological disease characterized by severe relapses that have significant impact on patients' lives. In the pivotal trial for Ultomiris (ravulizumab), all Ultomiris-treated patients remained adjudicated relapse free at Week 72. This was achieved with a much-improved delivery regimen every 2 months compared to Soliris (eculizumab) that is dosed every 2 weeks. This agreement with the pCPA is a huge step forward to providing patients access to this treatment and improving treatment of NMOSD in Canada," said Mark S. Freedman, HBSc, MSc, MD, CSPQ, FANA, FAAN, FRCPC, Professor of Medicine (Neurology), The Ottawa Hospital.

"Ensuring NMOSD patients have access to therapies proven highly effective is a top priority for The Sumaira Foundation globally. Since establishing TSF Canada in 2021, we have been supporting efforts to make these therapies available to Canadian patients as well. We are therefore very pleased to hear the news that ravulizumab is now becoming accessible to AQP4+ NMOSD patients in Canada. Congratulations to all stakeholders for their tireless efforts to make this possible, including the pan-Canadian Pharmaceutical Alliance (pCPA), Alexion Canada and of course, the patients and advocates who supported this important initiative," said Sumaira Ahmed, Executive Director and Founder, The Sumaira Foundation.

"The recent pCPA decision marks a key milestone in access to ravulizumab, offering patients with generalized myasthenia gravis a targeted, evidence-based biologic therapy. This agreement advances the treatment landscape and reflects a commitment to improving outcomes and quality of life for those affected by this chronic neuromuscular disorder," said Hans Katzberg, MD, MSc, FRCPC, FAAN, Professor of Medicine, University of Toronto.

"Reaching this agreement is an important milestone in addressing the treatment gap for individuals with generalized myasthenia gravis. We look forward to seeing public drug programs list Ultomiris quickly and equitably across the country. It's critical that our health systems reflect fairness and urgency in responding to the needs of those with rare diseases like MG," said Dr. Homira Osman, Vice President of Research & Public Policy, Muscular Dystrophy Canada.

"We would like to thank the pCPA for its partnership in recognizing the need for this important innovation for Canadians living with NMOSD and gMG. We look forward to our ongoing collaboration with provincial and territorial jurisdictions to finalize listing agreements and secure public reimbursement for patients and their caregivers," said Karen Heim, Vice President and General Manager of Alexion Canada.

Notes

NMOSD
NMOSD is a rare disease in which the immune system is inappropriately activated to target healthy tissues and cells in the CNS.¹ Approximately three-quarters of people with NMOSD are anti-AQP4 Ab+, meaning they produce antibodies that bind to a specific protein, aquaporin-4 (AQP4).^2-4 This binding can inappropriately activate the complement system, which is part of the immune system and is essential to the body's defence against infection, to destroy cells in the optic nerve, spinal cord and brain.^2-4

It most commonly affects women and begins in the mid-30s. Men and children may also develop NMOSD, but it is even more rare.^9-12 People with NMOSD may experience vision problems, intense pain, loss of bladder/bowel function, abnormal skin sensations (e.g., tingling, prickling or sensitivity to heat/cold) and impact on coordination and/or movement.⁵ Most people living with NMOSD experience unpredictable relapses, also known as attacks. Each relapse can result in cumulative disability including vision loss, paralysis and sometimes premature death.^4,5NMOSD is a distinct disease from other CNS diseases, including multiple sclerosis.¹³ The journey to diagnosis can be long, with the disease sometimes misdiagnosed.¹

gMG
gMG is a rare autoimmune disorder characterised by loss of muscle function and severe muscle weakness.⁶

Eighty-five percent of people with gMG are AChR antibody-positive meaning they produce specific antibodies (anti-AChR) that bind to signal receptors at the neuromuscular junction (NMJ), the connection point between nerve cells and the muscles they control.¹⁴ This binding activates the complement system, causing the immune system to attack the NMJ leading to inflammation and a breakdown in communication between the brain and the muscles.¹⁵

gMG can occur at any age, but it most commonly begins for women before the age of 40 and for men after the age of 60.¹⁶ Initial symptoms may include slurred speech, double vision, droopy eyelids and lack of balance; these can often lead to more severe symptoms as the disease progresses such as impaired swallowing, choking, extreme fatigue and respiratory failure.^7,8

Ultomiris
Ultomiris (ravulizumab), the longest-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. 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 complement cascade over-responds, leading the body to attack its own healthy cells. Following a loading dose, Ultomiris is administered intravenously every eight weeks in adults, or every four or eight weeks in paediatric patients (based on body weight).

Ultomiris is approved in the US, EU, Japan and other countries for the treatment of certain adults with paroxysmal nocturnal haemoglobinuria (PNH) and for certain children with PNH in the US and EU.

Ultomiris is also approved in the US, EU, Japan and other countries for the treatment of certain adults and children with atypical haemolytic uraemic syndrome (aHUS).

Additionally, Ultomiris is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with generalised myasthenia gravis (gMG).

Further, Ultomiris is approved in the US, EU, Japan and other countries for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD).

Ultomiris is being assessed as a treatment for additional indications as part of a broad development programme. 

Alexion
Alexion, AstraZeneca Rare Disease is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. For more information, please visit https://alexion.com/worldwide/canada.

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's innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit www.astrazeneca.ca and follow the Company on social media @AstraZeneca.

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SOURCE AstraZeneca

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