EC Approves Roche's PiaSky for Rare Blood Disorder PNH

Roche’s PiaSky (crovalimab) has received approval from the European Commission (EC) as the first monthly subcutaneous (SC) treatment for paroxysmal nocturnal haemoglobinuria (PNH), a rare and life-threatening blood disorder. This monoclonal antibody, which patients can self-administer following proper training, is approved for use in adults and adolescents aged 12 and older, who weigh at least 40 kg. It is suitable for patients both new to or previously treated with C5 inhibitors.

PNH is a condition affecting up to 1.5 per million people where red blood cells are destroyed by the complement system. This leads to symptoms such as anemia, fatigue, blood clots, and potentially kidney disease. Traditionally, C5 inhibitors like AstraZeneca’s Soliris (eculizumab) have been used to treat PNH, but they require regular intravenous infusions.

PiaSky aims to advance complement inhibition using its recycling technology, which allows monthly SC administration by enabling the drug to bind and inhibit the C5 protein multiple times. The EC's decision follows a recommendation from the European Medicines Agency’s human medicines committee, bolstered by positive outcomes from the late-stage COMMODORE 2 trial. This trial demonstrated that PiaSky, administered as SC injections every four weeks, controlled the disease effectively and was well tolerated in C5 inhibitor-naive PNH patients. The results showed that PiaSky was non-inferior to, and had comparable safety with, the standard-of-care Soliris, which requires intravenous administration 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 trial of patients new to C5 inhibitor treatment also supported Roche’s application. Levi Garraway, Roche’s chief medical officer and head of global product development, remarked that the approval of PiaSky provides a new option in the PNH treatment landscape. It combines the disease control achievable through C5 inhibition with advanced recycling technology, enabling monthly SC administration.

PiaSky, engineered by Chugai Pharmaceutical Co, already has approvals in major markets including the US and Japan. Garraway expressed satisfaction in bringing this new treatment to Europe, hoping it would reduce the treatment burden for many living with PNH.

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