Roche’s PiaSky (crovalimab) has been approved by the European Commission (EC) as the first monthly subcutaneous (SC) treatment for paroxysmal nocturnal haemoglobinuria (PNH), a rare and life threatening blood disorder.
The monoclonal antibody, which can be self-administered by patients following adequate training, has been authorised for use in adults and adolescents aged 12 years and older and weighing at least 40kg, who are either new to or have been previously treated with C5 inhibitors.
Affecting up to 1.5 per million people, PNH occurs when the red blood cells are destroyed by the complement system, resulting in symptoms such as anaemia, fatigue, blood clots, and potentially leading to kidney disease.
C5 inhibitors, including AstraZeneca’s Soliris (eculizumab), can be effective in treating the condition but require regular intravenous infusions.
PiaSky is designed to advance complement inhibition through its recycling technology, which enables monthly SC administration by allowing the drug to bind and inhibit the C5 protein multiple times.
The EC’s decision on the drug follows a recent recommendation from the European Medicines Agency’s human medicines committee and was supported by positive results from the late-stage COMMODORE 2 trial.
The study demonstrated that PiaSky, administered as SC injections every four weeks, achieved disease control and was well tolerated in C5 inhibitor-naive PNH patients. The drug was shown to be non-inferior to and had comparable safety with standard-of-care Soliris, which is given intravenously every two weeks.
Data from the phase 3 COMMODORE 1 trial of PNH patients switching from approved C5 inhibitors, and the late-stage COMMODORE 3 of those who were new to C5 inhibitor treatment also supported the company’s application.
Levi Garraway, Roche’s chief medical officer and head of global product development, 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 SC administration.”
PiaSky, which was engineered by Chugai Pharmaceutical Co, already holds approvals in major markets such as the US and Japan.
“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,” Garraway said.
