Alexion Pharma Canada Corp., AstraZeneca's Rare Disease group, has secured a critical access agreement with the pan-Canadian Pharmaceutical Alliance (pCPA) for Ultomiris (ravulizumab), bringing hope to Canadian patients with two rare and debilitating autoimmune diseases. The Letter of Intent covers treatment for adult patients with anti-aquaporin 4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD) and anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG).
Breakthrough Clinical Outcomes Drive Access Decision
The agreement follows compelling clinical evidence demonstrating Ultomiris's efficacy in preventing disease relapses. According to Mark S. Freedman, Professor of Medicine (Neurology) at The Ottawa Hospital, "In the pivotal trial for Ultomiris (ravulizumab), all Ultomiris-treated patients remained adjudicated relapse free at Week 72." This complete prevention of relapses represents a significant therapeutic advancement for NMOSD patients, who typically experience unpredictable and severe neurological attacks that can result in permanent disability.
The treatment also offers practical advantages over existing therapies. Ultomiris requires dosing every two months, a substantial improvement compared to Soliris (eculizumab), which requires administration every two weeks. This extended dosing interval could significantly reduce treatment burden for patients managing these chronic conditions.
Addressing Critical Unmet Medical Needs
NMOSD affects the central nervous system, including the spine and optic nerves, with approximately three-quarters of patients being anti-AQP4 antibody-positive. The disease predominantly affects women beginning in their mid-30s, though men and children can also develop the condition. Patients experience vision problems, intense pain, loss of bladder and bowel function, and coordination difficulties. Each relapse can result in cumulative disability including vision loss, paralysis, and sometimes premature death.
Generalized myasthenia gravis presents as a chronic autoimmune neuromuscular disease leading to severe muscle weakness. Eighty-five percent of gMG patients are AChR antibody-positive, producing antibodies that disrupt communication between nerves and muscles. Initial symptoms include slurred speech, double vision, and droopy eyelids, potentially progressing to extreme fatigue, difficulty swallowing, choking, and respiratory failure.
Mechanism of Action and Therapeutic Impact
Ultomiris functions as the longest-acting C5 complement inhibitor, providing immediate, complete, and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, preventing the immune system from attacking healthy cells when the complement system becomes inappropriately activated.
Hans Katzberg, Professor of Medicine at the University of Toronto, emphasized the therapeutic significance: "The recent pCPA decision marks a key milestone in access to ravulizumab, offering patients with generalized myasthenia gravis a targeted, evidence-based biologic therapy."
Pathway to Patient Access
With the pCPA agreement in place, individual provinces and territories can now initiate processes to list Ultomiris on their formularies, though timing will vary by jurisdiction. Karen Heim, Vice President and General Manager of Alexion Canada, expressed gratitude for the partnership and commitment to ongoing collaboration with provincial and territorial jurisdictions to finalize listing agreements.
Dr. Homira Osman from Muscular Dystrophy Canada stressed the importance of equitable access: "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."
Broader Treatment Landscape
Ultomiris has established a comprehensive global approval profile across multiple rare disease indications. The drug is approved in the US, EU, Japan, and other countries for treating adults with paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), generalized myasthenia gravis, and neuromyelitis optica spectrum disorder. The medication is also approved for certain pediatric populations in specific indications.
The Canadian access agreement represents a significant milestone for rare disease patients, as noted by Sumaira Ahmed, Executive Director and Founder of The Sumaira Foundation: "We are therefore very pleased to hear the news that ravulizumab is now becoming accessible to AQP4+ NMOSD patients in Canada."
