Roche Treatment for Paroxysmal Nocturnal Hemoglobinuria Approved in EU

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The European Commission (EC) has approved Roche’s PiaSky (crovalimab) for certain patients with paroxysmal nocturnal hemoglobinuria (PNH), Roche announced in a press release on Aug. 27, 2024 (1). PiaSky is a recycling monoclonal antibody that inhibits the complement protein C5; the EC approval was granted for adults, and adolescents aged 12 and older with a body weight of at least 40 kg (approximately 88 pounds), who are either new to or have been previously treated with C5 inhibitors.

PNH is a rare, life-threatening blood condition typified by the complement system, part of the innate immune system, working to destroy red blood cells. This can result in symptoms such as anemia, fatigue, and blood clots and can lead to kidney disease (1).

Roche said that the recycling technology used in the development of PiaSky allows for monthly, subcutaneous administration in which a smaller volume of medicine is able to bind and inhibit the C5 protein multiple times and act longer in the body (1). It is the first monthly subcutaneous treatment for PNH approved in the European Union (giving patients the option to self-administer, following adequate training) and has been approved for similar use in the United States and Japan.

A Phase III study (COMMODORE 2) in people with PNH who had not been treated with C5 inhibitors showed that the rate of adverse events in patients treated with crovalimab (78%) was similar to that of an existing standard of care C5 inhibitor, eculizumab (80%) (2–3).

“People with PNH are often burdened with lifelong, frequent intravenous infusions with time-consuming clinic visits, meaning that their lives, as well as their caregivers’ and families’ lives, may revolve around the demands of their treatment,” Alexander Röth, MD, head of classical hematology and hemostasis at the West German Cancer Centre, University Hospital Essen, Germany, and lead investigator of the study, said in the press release. “More flexible treatment options such as PiaSky, which are just as effective but less frequent and can be given more quickly at home, are essential to give people with PNH greater control over their treatment and more independence.”

“The PiaSky approval brings a new option to the PNH treatment landscape, combining the disease control achievable through C5 inhibition with a cutting-edge recycling technology that enables monthly subcutaneous administration,” said Levi Garraway, MD, PhD, Roche chief medical officer and head of global product development. “We are pleased to bring this new treatment to people with PNH in Europe with the hope it may lessen the treatment burden faced by many living with this condition.”

References

1. Roche. Roche’s PiaSky Approved in the EU as the First Monthly Subcutaneous Treatment for People with PNH. Press Release. Aug. 27, 2024.
2. Röth, A.; He, G.; Brodsky, A.; et al. The Phase III, Randomized COMMODORE 2 Trial: Results from a Multicenter Study of Crovalimab vs. Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Naïve to Complement Inhibitors. HemaSphere 2023, 7 (S3) e72750f1. DOI: 10.1097/01.HS9.0000967636.72750.f1
3. National Library of Medicine. A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. (COMMODORE 2). ClinicalTrials.gov, updated Aug. 13, 2024 (accessed Aug. 27, 2024).