Roche has received approval for PiaSky (crovalimab) for use in patients with paroxysmal nocturnal haemoglobinuria (PNH) from the European Commission (EC).
The treatment regimen is suitable for those 12 years or older, but patients must weigh 40kg or over.
PiaSky is a monoclonal antibody that works to inhibit complement protein C5.
This protein system is intimately involved in the disease pathogenesis of PNH, as its aberrant form results in the breakdown of red blood cells.
This can lead to fatigue, blood clots, anaemia and kidney disease if left untreated.
PiaSky is the first subcutaneous treatment for PNH to receive approval from the EU, and it can be self-administered when the appropriate training is given.
Roche designed the therapeutic as a patient-centric option to currently available C5 inhibitors, which need to be administered intravenously.
“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.”
The EC’s decision to approve PiaSky was based on the results of the Phase III COMMODORE 2 study, which found that the monthly administration of PiaSky could control disease and was well tolerated in patients.
The therapeutic has now been approved in the US, Japan and Europe.