Roche’s PiaSky approved in the EU as the first monthly subcutaneous treatment for people with PNH

Roche announced that the European Commission has approved PiaSky (crovalimab), a novel recycling monoclonal antibody that inhibits the complement protein C5, for adults and adolescents (12 years of age or older with a weight of 40 kg and above) with paroxysmal nocturnal haemoglobinuria (PNH) who are either new to, or have been previously treated with C5 inhibitors. PNH is a rare and life-threatening blood condition where red blood cells are destroyed by the complement system – part of the innate immune system – causing symptoms such as anaemia, fatigue and blood clots, and potentially leading to kidney disease.

“People with PNH are often burdened with life-long, 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,” 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 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.”

PiaSky is the first monthly subcutaneous (SC) treatment for PNH in the European Union, with the option to self-administer following adequate training. It provides an alternative option to current C5 inhibitors that require regular intravenous infusions, which could help to reduce treatment burden and disruption to the lives of people with PNH and their caregivers.

“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, M.D., Ph.D., Roche’s 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.”

C5 inhibitors – treatments that block part of the complement system cascade – have been shown to be effective in treating PNH. PiaSky has been developed to address the needs of people living with PNH and some of the challenges that accompany these existing treatment options. It advances complement inhibition through its innovative recycling technology, which enables monthly SC administration by 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.

This approval is based on the results from the Phase III COMMODORE 2 study in people with PNH who have not been previously treated with C5 inhibitors. The study demonstrated that PiaSky, administered as SC injections every four weeks, achieved disease control and was well-tolerated. PiaSky was non-inferior with comparable safety to eculizumab, an existing standard of care C5 inhibitor, given intravenously every two weeks. The rate of adverse events in people treated with PiaSky was similar to treatment with eculizumab. The application included supportive data from two additional Phase III studies, the COMMODORE 1 study, in people with PNH switching from currently approved C5 inhibitors, and the COMMODORE 3 study in people new to C5 inhibitor treatment in China.

PiaSky is the first monthly SC treatment for PNH, approved in multiple territories around the world, including the US and Japan, based on results of the COMMODORE studies. It is being investigated in a broad clinical development programme, including five Phase III studies and three earlier phase studies in complement-mediated diseases, including PNH, atypical haemolytic uremic syndrome and sickle cell disease.