Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission has approved PiaSky® (crovalimab), a novel monoclonal antibody designed to inhibit the complement protein C5, for the treatment of adults and adolescents (12 years and older, weighing at least 40 kg) with paroxysmal nocturnal haemoglobinuria (PNH). PiaSky is intended for patients who are either new to C5 inhibitor therapy or have previously been treated with C5 inhibitors. PNH is a rare, life-threatening blood disorder where red blood cells are destroyed by the complement system, leading to symptoms like anaemia, fatigue, blood clots, and potentially kidney disease.
“People with PNH often endure life-long, frequent intravenous infusions that require time-consuming clinic visits, significantly impacting their lives and the lives of their caregivers and families,” said Prof. Alexander Röth, M.D., Head of Classical Haematology and Haemostasis at the West German Cancer Centre, University Hospital Essen, Germany. “More flexible treatment options like PiaSky, which are equally effective but less frequent and can be administered at home, are crucial for providing greater control and independence to those with PNH.”
PiaSky is the first monthly subcutaneous (SC) treatment for PNH approved in the European Union, offering patients the option of self-administration after proper training. This alternative to regular intravenous C5 inhibitors aims to reduce the treatment burden and the impact on the lives of patients and their caregivers.
“The approval of PiaSky marks a significant advancement in PNH treatment, combining effective disease control through C5 inhibition with cutting-edge recycling technology that allows for monthly subcutaneous administration,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “We are proud to introduce this new treatment in Europe, with the hope that it will alleviate the treatment burden for many living with PNH.”
C5 inhibitors, which block a key part of the complement system, have proven effective in treating PNH. PiaSky was developed to address the needs of PNH patients by offering an innovative recycling technology that allows for monthly SC administration. This technology enables the medicine to bind to and inhibit the C5 protein multiple times, extending its action in the body with a small volume of medication.
The European Commission’s approval is based on data from the Phase III COMMODORE 2 study, which involved patients with PNH who had not previously received C5 inhibitors. The study demonstrated that PiaSky, administered as SC injections every four weeks, effectively controlled the disease and was well-tolerated. PiaSky was found to be non-inferior to eculizumab, an existing standard C5 inhibitor administered intravenously every two weeks, with a comparable safety profile. Additional supportive data from the Phase III COMMODORE 1 and COMMODORE 3 studies, which included patients switching from other C5 inhibitors and those new to C5 inhibitor treatment in China, also contributed to this approval.
PiaSky is now the first monthly SC treatment for PNH to be approved in multiple regions worldwide, including the US and Japan, based on the results of the COMMODORE studies.
