Health Canada Authorizes PiaSky® (crovalimab for injection) As the First Monthly (Every Four Weeks) Subcutaneous Treatment for People With Paroxysmal Nocturnal Hemoglobinuria (PNH)

• PiaSky® is the first monthly (every four weeks) subcutaneous treatment for PNH in Canada with the option to self-administer following adequate training⁽¹⁾
• PiaSky® (crovalimab for injection) has the potential to help reduce treatment burden for people with PNH in Canada and their caregivers ^{(2, 3)}

MISSISSAUGA, ON, June 11, 2025 /CNW/ - Hoffmann-La Roche Limited (Roche Canada) is pleased to announce that Health Canada has authorized PiaSky® (crovalimab for injection) as the first monthly (every four weeks) subcutaneous therapy indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults and adolescents 13 years of age and older with a body weight of at least 40 kg.⁽¹⁾

"We are pleased to offer PiaSky as a new option to the treatment landscape for Canadians living with PNH," said Brigitte Nolet, President and CEO, Roche Pharma Canada. "With a  self-administration option available for appropriate patients, as determined by their doctor, individuals can choose to treat their disease at home without requiring IV access, and feel empowered to take an active role in their own care. This flexibility of administration may help reduce treatment burden for patients, their caregivers, and healthcare systems, and is part of Roche's ongoing commitment to bringing Canadians new therapies that have a broader value to society."

PNH is a rare, genetically acquired, and life-threatening disease of the bone marrow where red blood cells deficient in certain protective markers are destroyed by the complement system, which helps defend against infection.⁽⁴⁾ The destruction of these abnormal blood cells lead to symptoms such as anemia, fatigue, hemoglobinuria (blood in the urine), and blood clots.^{(5, 6)} Globally, the estimated prevalence of PNH is estimated at 15.9 cases per million, affecting fewer than 1,000 individuals in Canada.⁽⁷⁾ Complement component 5 (C5) inhibitors – treatments that block part of the complement system cascade – have been shown to be effective in treating PNH.^{(4, 5, 6, 8)}

"The approval of crovalimab, a novel C5 inhibitor, provides further freedom of choice for patients with PNH receiving terminal complement blockade," said Dr. Christopher Patriquin, Chair of the Canadian PNH Network and Assistant Professor of Medicine and Clinician Investigator at the University of Toronto. "Blocking C5 directly was the first approved strategy for treating such patients, and this new drug will allow them another option. They can now choose to get the disease-controlling treatment in small-volume, subcutaneous doses every 4 weeks, and even, in appropriate cases, self-administer them, instead of the other available C5 inhibitors which are given intravenously."

"Monthly (every four weeks) self-injection of crovalimab has the potential to improve the quality of life for people living with PNH," said Barry Katsoff, President, Canadian Association of PNH Patients. "The potential for a more manageable, home-administered solution is especially meaningful for PNH patients whose daily lives are often disrupted by the demands of ongoing therapy. Canadian patients welcome the introduction of crovalimab to our basket of options."

Health Canada's authorization of PiaSky is based on the pivotal COMMODORE 2 Phase III study in people with PNH who have not previously been treated with C5 inhibitors.^{(1, 9, 10)} The study demonstrated that PiaSky, administered as subcutaneous (SC) injections every four weeks, achieved disease control (as measured by hemolysis control and transfusion avoidance) and was well-tolerated. ⁽¹⁾

Roche Canada is committed to working with the provincial and territorial jurisdictions to make PiaSky available as soon as possible through public and private drug plans for the patients who need it.

About paroxysmal nocturnal hemoglobinuria (PNH)

PNH is a rare hematological disease that may develop on its own or in the context of other bone marrow disorders such as aplastic anemia. Somatic mutations to the PIGA gene lead to clonal expansion of hematopoietic stem cells in the bone marrow which lack certain protein cell surface markers. The absence of these markers renders blood cells susceptible to lysis by the complement system, part of our innate immune defense against common pathogens. These processes cause anemia, thrombosis (blood clots), fatigue, and other debilitating symptoms that significantly impact quality of life and mental health. Individuals can be diagnosed at any stage of life, but are most commonly diagnosed in their 30's or 40's.^{(5, 6, 11, 12)}

About PiaSky® [crovalimab for injection]

PiaSky is a novel recycling monoclonal antibody that inhibits the complement protein C5 and is designed to block the complement system.^{(1, 10)} It is recycled within the bloodstream, allowing the medicine to bind and inhibit the C5 protein multiple times and to act longer in the body with a small volume of medicine.^{(1, 13)} Individuals will have the option to self-administer PiaSky following an initial intravenous (IV) infusion and weekly subcutaneous (SC) loading doses in the first month of treatment, in appropriate cases following adequate training.⁽¹⁾ PiaSky's flexibility of administration may help to reduce treatment burden and disruption to the lives of people with PNH and their caregivers.^{(2, 3)}

About the COMMODORE 2 study

The COMMODORE 2 study is a Phase III, randomised, open-label study evaluating the efficacy and safety of PiaSky® (crovalimab for injection) versus eculizumab in people with paroxysmal nocturnal hemoglobinuria who have not been treated previously with C5 inhibitors. The study's co-primary efficacy endpoints measure transfusion avoidance and control of hemolysis (the ongoing destruction of red blood cells measured by lactate dehydrogenase levels). The adults enrolled in the study were randomised in a 2:1 ratio to be treated with either subcutaneous (SC) PiaSky every four weeks or intravenous eculizumab every two weeks. The participants who were less than 18 years old were included in a non-randomised treatment arm and were treated with SC PiaSky every four weeks.^{(9, 10)}

About Roche Canada
At Roche Canada, patients and science are at the heart of everything we do. Our passion for science and our commitment to relentlessly pursuing the impossible for patients have made us one of the world's leading pharmaceutical, in-vitro diagnostics, and diabetes care management companies.

With our combined strength in diagnostics and pharmaceuticals, we're driving healthcare forward, while ensuring we deliver meaningful benefits for patients and a sustainable healthcare system. Because we're committed to making quality healthcare accessible to everyone.

And we're adding our expertise in new areas, such as artificial intelligence, real world data collection and analysis and collaborating with many different sectors and industries.

Having the courage to reinvent ourselves and question the status quo is what patients and the healthcare system expect from Roche - and our commitment is as strong today as it was on the first day of our Canadian journey in 1931. Today, Roche Canada employs more than 1,800 people across the country through its Pharmaceuticals division in Mississauga, Ontario as well as its Diagnostics and Diabetes Care divisions in Laval, Quebec.

For more information, please visit www.RocheCanada.com or follow Roche Canada on LinkedIn.

References

1. PiaSky Product Monograph, June 4, 2025
2. Roth A, Nishimura JI, Nagy Z, Gaal-Weisinger J, Panse J, Yoon SS, et al. The complement C5 inhibitor crovalimab in paroxysmal nocturnal hemoglobinuria. Blood. 2020;135(12):912–20.
3. Sampei Z, Haraya K, Gan SW, Muraoka M, Hayasaka A, Fukuzawa T, et al. Beyond Recycling Antibodies: Crovalimab's Molecular Design Enables Four-Weekly Subcutaneous Injections for PNH Treatment. Int J Mol Sci. 2024;25(21).
4. Shah N, Bhatt H. Paroxysmal nocturnal hemoglobinuria. StatPearls. Treasure Island (FL) 2022.
5. Schrezenmeier H, Roth A, Araten DJ, Kanakura Y, Larratt L, Shammo JM, et al. Baseline clinical characteristics and disease burden in patients with paroxysmal nocturnal hemoglobinuria (PNH): updated analysis from the International PNH Registry. Ann Hematol. 2020;99(7):1505–14.
6. Patriquin CJ, Kiss T, Caplan S, Chin-Yee I, Grewal K, Grossman J, et al. How we treat paroxysmal nocturnal hemoglobinuria: a consensus statement of the Canadian PNH Network and review of the national registry. Eur J Haematol. 2019;102(1):36–52.
7. Hill A, Platts PJ, Smith A, Richards SJ, Cullen MJ, Hill QA, et al. The incidence and prevalence of paroxysmal nocturnal hemoglobinuria (PNH) and survival of patients in Yorkshire. Blood. 2006;108:985.
8. Hillmen P, Young NS, Schubert J, Brodsky RA, Socie G, Muus P, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355(12):1233–43.
9. COMMODORE 2 (NCT04434092). [Internet; cited June 2025] Available at: https://www.clinicaltrials.gov/ct2/show/NCT04434092.
10. Roth A, He G, Tong H, Lin Z, Wang X, Chai-Adisaksopha C, et al. Phase 3 randomized COMMODORE 2 trial: Crovalimab versus eculizumab in patients with paroxysmal nocturnal hemoglobinuria naive to complement inhibition. Am J Hematol. 2024;99(9):1768-77.
11. Oliver M, Patriquin, C. Paroxysmal Nocturnal Hemoglobinuria: Current Management, Unmet Needs, and Recommendations. J Blood Med. 2023:613-28.
12. Parker C, Omine M, Richards S, Nishimura J, Bessler M, Ware R, et al. Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood. 2005;106(12):3699–709.
13. Fukuzawa, T, Sampei, Z, Haraya, K, Ruike, Y, Shida-Kawazoe, M, Shimizu, Y, et al. Long lasting neutralisation of C5 by SKY59, a novel recycling antibody, is a potential therapy for complement-mediated diseases. Sci Rep. 2017;7(1):1080.

SOURCE Hoffmann-La Roche Limited (Roche Canada)

Media Contact: Amanda Rutyna, Strategic Communications, Hoffmann-La Roche Limited, [phone], [email protected]