Swiss drugmaker Roche has received the European Commission (EC) approval for PiaSky (crovalimab) to treat a type of paroxysmal nocturnal haemoglobinuria (PNH).
PiaSky is a novel recycling monoclonal antibody that inhibits the complement protein C5, blocking part of the complement system cascade and has shown effectiveness in treating PNH.
The drug is indicated for adults and adolescents with PNH, aged 12 years and older, weighing 40kg and above, who are either new to, or have been previously treated with C5 inhibitors.
PNH is a rare, severe blood condition where red blood cells are destroyed by the complement system, causing anaemia, fatigue, and blood clots, and may lead to kidney disease.
Roche said that PiaSky has been developed to meet the needs of people living with PNH and some of the challenges related to the existing treatment options.
Also, PiaSky is the first monthly subcutaneous (SC) treatment for PNH in the European Union, (EU) with a self-administration option after adequate training, said the Swiss drugmaker.
Roche chief medical officer and global product development head Levi Garraway said: “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.
“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.”
The EC approval is based on the results from the Phase 3 COMMODORE 2 clinical study in people with PNH who have not been previously treated with C5 inhibitors.
The Phase 3 study showed that PiaSky, administered as SC injections every four weeks, achieved disease control and was well-tolerated.
In the study, PiaSky was non-inferior with comparable safety to eculizumab, an existing standard of care C5 inhibitor, administered intravenously every two weeks.
According to Roche, PiaSky is the first monthly SC treatment for PNH, approved in multiple territories, including the US and Japan, based on the results from the COMMODORE studies.
The drug is being studied in a broad clinical development programme, including five Phase 3 studies and three earlier phase studies in complement-mediated diseases, including PNH.
University Hospital Essen West German Cancer Centre classical haematology and haemostasis head Alexander Röth said: “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.
“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.”
