REGULATORY APPROVAL | PiaSky Becomes the First Monthly Treatment for PNH in the EU
The European Commission has approved PiaSky® (crovalimab), a new type of recycling monoclonal antibody that blocks the complement protein C5. This approval is for adults and teenagers (12 years or older and weighing at least 40 kg) with paroxysmal nocturnal haemoglobinuria (PNH), regardless of whether they are receiving C5 inhibitors for the first time or have used them before.
Paroxysmal Nocturnal Haemoglobinuria (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.
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.
“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,” affirmed 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.”
In June 2024, the FDA also approved crovalimab for treating adults and children aged 13 and older with PNH, who weigh at least 40 kg, based on data from the COMMODORE 2 study.
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.
According to Roche, 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.
Source: Roche Media Release
