Roche’s PiaSky Approved in EU as First Monthly Self-Administered Treatment for PNH

(IN BRIEF) Roche has received approval from the European Commission for PiaSky® (crovalimab), a novel treatment for paroxysmal nocturnal haemoglobinuria (PNH), making it the first monthly subcutaneous (SC) treatment available in the European Union. PiaSky, which can be self-administered by patients, offers a less burdensome alternative to the traditional bi-weekly intravenous infusions, potentially improving the quality of life for those with PNH. The approval is based on the results of the Phase III COMMODORE 2 study, which demonstrated that PiaSky is as effective and safe as existing treatments while offering the convenience of monthly administration.

(PRESS RELEASE) BASEL, 28-Aug-2024 — /EuropaWire/ — The European Commission has granted approval for PiaSky® (crovalimab), a groundbreaking treatment for paroxysmal nocturnal haemoglobinuria (PNH), developed by Roche. This approval marks PiaSky as the first subcutaneous (SC) treatment for PNH that can be administered on a monthly basis, offering patients the option to self-administer the treatment at home after proper training. This development is set to significantly reduce the treatment burden for PNH patients and their caregivers by providing a more flexible and less intrusive alternative to the existing intravenous therapies that require frequent clinic visits.

PNH is a rare and potentially life-threatening blood disorder where red blood cells are attacked by the body’s immune system, leading to severe symptoms such as anaemia, fatigue, and blood clots, which can result in complications like kidney disease. PiaSky works by inhibiting the complement protein C5, a critical component in the disease’s progression. The innovative recycling technology used in PiaSky allows the medicine to be effective with monthly SC administration, a significant improvement over current treatments that often require bi-weekly intravenous infusions.

The approval is based on positive outcomes from the Phase III COMMODORE 2 study, where PiaSky demonstrated comparable safety and efficacy to eculizumab, a standard C5 inhibitor, while offering the convenience of less frequent dosing. With its introduction, PiaSky is poised to offer PNH patients in Europe a new level of independence and control over their treatment, helping to improve their quality of life.

About PiaSky® (crovalimab)
PiaSky® (crovalimab) is a novel recycling monoclonal antibody that inhibits the complement protein C5 and is designed to block the complement system – a vital part of the innate immune system that acts as the body’s first line of defence against infection. PiaSky has been engineered by Chugai Pharmaceutical Co., Ltd, to address the needs of people living with complement-mediated diseases. It provides patients with a potential for subcutaneous (SC) self-administration following adequate training, an initial intravenous infusion and weekly SC loading doses in the first month of treatment.1,3

PiaSky works by binding to C5, blocking the last step of the complement cascade and delivering rapid and sustained complement inhibition. It is also recycled within the bloodstream, enabling small volume SC administration every four weeks. In addition, PiaSky binds to a different C5 binding site from current treatments, which has the potential to provide a treatment option for people with specific C5 gene mutations who do not respond to current therapies.1,3

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) versus eculizumab in people with paroxysmal nocturnal haemoglobinuria who have not been treated previously with C5 inhibitors. The study’s co-primary efficacy endpoints measure transfusion avoidance and control of haemolysis (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.1,2

About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.

In recognising our endeavour to pursue a long-term perspective in all we do, Roche has been named one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for the fifteenth consecutive year. This distinction also reflects our efforts to improve access to healthcare together with local partners in every country we work.

Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.

For more information, please visit www.roche.com.

All trademarks used or mentioned in this release are protected by law.

References
[1] Roth A, et al. The Phase III, Randomised COMMODORE 2 Trial: Results from a Multicentre Study of Crovalimab vs Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Naïve to Complement Inhibitors. Presentation at European Hematology Association (EHA) Annual Congress; 2023 June 08-13. Abstract #S181.
[2] COMMODORE 2 (NCT04434092). [Internet; cited August 2024] Available at: https://www.clinicaltrials.gov/ct2/show/NCT04434092.
[3] Fukuzawa T, et al. Long lasting neutralisation of C5 by SKY59, a novel recycling antibody, is a potential therapy for complement-mediated diseases. 2017; Sci Rep 7, 1080.
[4] National Organization for Rare Diseases. Paroxysmal nocturnal hemoglobinuria. [Internet; cited August 2024]. Available at: https://rarediseases.org/rare-diseases/paroxysmal-nocturnal-hemoglobinuria/.
[5] Peffault de Latour R, et al. Hemolytic paroxysmal nocturnal hemoglobinuria: 20 years of medical progress. Seminars in Hematology. 2022;59(1):38-46
[6] Scheinberg P, et al. Phase III Randomised, Multicentre, Open-Label COMMODORE 1 Trial: Comparison of Crovalimab Vs Eculizumab in Complement Inhibitor-Experienced Patients with Paroxysmal Nocturnal Hemogobinuria (PNH). Presentation at European Hepatology Association (EHA) Annual Congress; 2023 June 08-13. Abstract #S183.
[7] COMMODORE 1 (NCT04432584). [Internet; cited August 2024] Available at: https://www.clinicaltrials.gov/ct2/show/NCT04432584.
[8] Liu H, et al. Six-month Crovalimab Extension in the Phase III COMMODORE 3 Study: Updated Efficacy and Safety Results in Complement Inhibitor-Naive Patients with Paroxysmal Nocturnal Hemoglobinuria. Poster presentation at European Hematology Association (EHA) Annual Congress; 2023 June 08-13. Abstract #P785.
[9] COMMUTE-p (NCT04958265). [Internet; cited August 2024] Available at: https://www.clinicaltrials.gov/ct2/show/NCT04958265.
[10] COMMUTE-a (NCT04861259). [Internet; cited August 2024] Available at: https://www.clinicaltrials.gov/ct2/show/NCT04861259.
[11] Nishimura J, et al. Crovalimab for Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria And Complement C5 Polymorphism – Experience From The Composer Phase I/II Study. EHA Library. 2020; Abstract #PB1992.
[12] CROSSWALK-a (NCT04912869). [Internet; cited August 2024]. Available at: https://clinicaltrials.gov/ct2/show/NCT04912869.
[13] CROSSWALK-c (NCT05075824). [Internet; cited August 2024]. Available at: https://clinicaltrials.gov/ct2/show/NCT05075824.