Roche Holding AG announced that the European Medicines Agency?s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for PiaSky® (crovalimab) for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). The CHMP has recommended PiaSky, a novel recycling monoclonal antibody that inhibits the complement protein C5, for use in adults and adolescents (12 years of age or older with a weight of 40 kg) who are either new to, or have been previously treated with C5 inhibitors. If approved, PiaSky will be the first monthly subcutaneous (SC) treatment for PNH in the European Union, with the option to self-administer following adequate training.

This may provide an alternative option to current C5 inhibitors that require regular intravenous (IV) infusions, potentially helping to reduce treatment burden and disruption to the lives of people with PNH and their caregivers. A final decision regarding the approval of PiaSky is expected from the European Commission in the near future. PNH is a rare and life-threatening blood condition, which affects approximately 20,000 people worldwide.

In PNH, red blood cells are destroyed by the complement system ? part of the innate immune system. This causes symptoms such as anaemia, fatigue and blood clots, and can lead to kidney disease.

C5 inhibitors ? treatments that block part of the complement system cascade ? have been shown to be effective in treating PNH.

PiaSky is a novel C5 inhibitor that is recycled within the bloodstream, 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 enables SC administration every four weeks, following an initial IV infusion and weekly SC loading doses in the first month of treatment. The CHMP recommendation 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.

Results from 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 (78% versus 80%, respectively).

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.