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Paroxysmal Nocturnal Hemoglobinuria Approval Recommended for Roche’s PiaSky® by EMA

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The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Roche’s PiaSky® (crovalimab) for treating paroxysmal nocturnal hemoglobinuria (PNH). PiaSky, a novel recycling monoclonal antibody that inhibits the complement protein C5, is recommended for use in adults and adolescents (12 years or older with a weight of 40 kg) new to, or previously treated with C5 inhibitors. If approved, PiaSky will be the first monthly subcutaneous (SC) treatment for PNH in the EU, offering an option to self-administer after training, potentially reducing the treatment burden compared to regular intravenous (IV) infusions.

The CHMP’s recommendation is based on the Phase III COMMODORE 2 study results, where SC PiaSky given every month demonstrated equivalent disease control and comparable safety to IV eculizumab given every two weeks. The study showed that PiaSky achieved disease control and was well-tolerated, with adverse event rates similar to eculizumab. The application included supportive data from two additional Phase III studies: COMMODORE 1 in PNH patients switching from current C5 inhibitors and COMMODORE 3 in paroxysmal nocturnal hemoglobinuria patients new to C5 inhibitor treatment in China.

Paroxysmal Nocturnal Hemoglobinuria Treatment Advances with Roche’s PiaSky® Offering Enhanced Patient Freedom

PNH affects approximately 20,000 people worldwide, causing symptoms like anemia, fatigue, and blood clots, potentially leading to kidney disease. PiaSky works by binding to C5, blocking the complement cascade, and delivering rapid and sustained inhibition. It is recycled within the bloodstream, allowing small volume SC administration every four weeks. This differs from current treatments and provides an option for people with specific C5 gene mutations who do not respond to existing therapies.

Roche’s Chief Medical Officer, Levi Garraway, emphasized the potential of PiaSky to offer patients more freedom in their daily lives by reducing the need for frequent clinic visits. The CHMP’s recommendation marks a significant step towards making PiaSky available to paroxysmal nocturnal hemoglobinuria patients in Europe. A final decision from the European Commission is expected soon.

Paroxysmal Nocturnal Hemoglobinuria

Roche’s PiaSky® Gains Approval as First Monthly SC Treatment for Paroxysmal Nocturnal Hemoglobinuria in US, Japan, and China

PiaSky is the first monthly SC treatment approved in the US, Japan, and China for paroxysmal nocturnal hemoglobinuria based on the COMMODORE studies. It is being investigated in a broad clinical development program, including five ongoing Phase III studies and three earlier-phase studies in complement-mediated diseases, such as atypical hemolytic uremic syndrome and sickle cell disease.

Founded in 1896, Roche is a global leader in biotechnology and in-vitro diagnostics, committed to scientific excellence and personalized healthcare. Recognized as one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices, Roche partners with stakeholders worldwide to improve access to healthcare. Genentech, in the US, is a member of the Roche Group, and Roche is the majority shareholder in Chugai Pharmaceutical, Japan.

This release includes forward-looking statements about Roche’s efforts to improve healthcare access and develop innovative treatments. These statements are based on current beliefs and expectations but are subject to risks and uncertainties that could cause actual results to differ materially.


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Resource: Roche, June 28, 2024

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