Roche has received positive news from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP), which has recommended the approval of PiaSky (crovalimab) for the treatment of paroxysmal nocturnal haemoglobinuria (PNH). This recommendation marks a significant step forward in offering a new treatment option for adults and adolescents (12 years and older, weighing at least 40 kg) with PNH who are either new to or have previously been treated with C5 inhibitors.
PiaSky, described as a novel recycling monoclonal antibody, works by inhibiting the complement protein C5, a crucial part of the immune system involved in PNH. Unlike existing treatments that require frequent intravenous infusions, PiaSky offers the convenience of a monthly subcutaneous injection after an initial phase of intravenous and weekly subcutaneous loading doses. This potential approval could provide patients and caregivers greater flexibility and reduce the treatment burden associated with managing PNH.
Levi Garraway, M.D., Ph.D., Roche’s chief medical officer, highlighted the impact of this recommendation on patients’ daily lives, noting that PiaSky’s monthly self-administration option could offer more freedom and convenience compared to current treatment regimens.
PNH is a rare and serious blood disorder affecting approximately 20,000 people worldwide. It is characterized by the destruction of red blood cells by the body’s complement system, leading to symptoms such as anaemia, fatigue, and an increased risk of blood clots and kidney disease. C5 inhibitors like PiaSky have demonstrated efficacy in managing PNH by blocking this destructive process.
The CHMP’s recommendation is supported by data from the phase III COMMODORE 2 study, which showed that PiaSky effectively controlled the disease and was well-tolerated by patients who had not previously received C5 inhibitors. The study compared PiaSky’s performance with eculizumab, a current standard of care, and found comparable safety profiles between the two treatments.
Pending approval from the European Commission, PiaSky would become the first monthly subcutaneous treatment option available for PNH in the European Union. This decision underscores Roche’s commitment to advancing treatment options for rare diseases and improving outcomes for patients affected by complement-mediated disorders.
Roche continues to explore the potential of PiaSky in a broad clinical development program, including studies in atypical haemolytic uremic syndrome and sickle cell disease, aiming to address unmet medical needs in these challenging conditions.