EU Approval for Roche’s PiaSky as First Monthly Subcutaneous Treatment for PNH
Roche has announced that the European Commission has approved PiaSky (crovalimab) as a new treatment option for adults and adolescents (12 years and older, weighing at least 40 kg) with paroxysmal nocturnal haemoglobinuria (PNH), a rare and life-threatening blood disorder. This condition leads to the destruction of red blood cells by the complement system, part of the body's innate immune defense, resulting in symptoms such as anemia, fatigue, blood clots, and potentially kidney disease. PiaSky is the first treatment in the European Union that can be administered subcutaneously once a month, offering patients the option to self-administer the medication after receiving appropriate training.
PiaSky provides an alternative to the existing C5 inhibitors, which typically require frequent intravenous infusions. This new treatment option is expected to ease the burden on patients with PNH and their caregivers by reducing the frequency and inconvenience of hospital visits for treatment.
C5 inhibitors work by blocking part of the complement system cascade and have been effective in managing PNH. PiaSky has been specifically designed to meet the needs of PNH patients while addressing some of the limitations of current therapies. It features an innovative recycling technology that allows the medicine to bind to and inhibit the C5 protein multiple times, enabling longer action in the body with a smaller volume of medication. This technology allows for the drug’s monthly subcutaneous administration.
The European Commission's approval of PiaSky is based on findings from the Phase III COMMODORE 2 study, which included patients with PNH who had not previously been treated with C5 inhibitors. The study demonstrated that monthly subcutaneous injections of PiaSky were as effective in controlling the disease as bi-weekly intravenous infusions of eculizumab, a commonly used C5 inhibitor. PiaSky also showed a similar safety profile to eculizumab, with comparable rates of adverse events. Additionally, the approval was supported by data from two other Phase III trials: the COMMODORE 1 study, which involved patients transitioning from other C5 inhibitors, and the COMMODORE 3 study, which included patients new to C5 inhibitor treatment in China.
PiaSky has now been approved in several countries worldwide, including the United States and Japan, based on the results of the COMMODORE studies. The treatment is being further explored in an extensive clinical development program that includes five Phase III studies and three earlier-phase studies focusing on diseases mediated by the complement system, such as PNH, atypical hemolytic uremic syndrome, and sickle cell disease.
About PiaSky (crovalimab)
PiaSky (crovalimab) is a novel monoclonal antibody developed to inhibit the complement protein C5, a key player in the complement system, which is part of the body's first line of defense against infections. Engineered by Chugai Pharmaceutical Co., Ltd., PiaSky is designed to meet the needs of individuals with complement-mediated diseases. The treatment offers the potential for self-administration after initial intravenous infusions and weekly subcutaneous loading doses during the first month of treatment.
PiaSky operates by binding to the C5 protein, blocking the final step in the complement cascade, and providing rapid and sustained inhibition of the complement system. Its recycling ability within the bloodstream allows for a small-volume subcutaneous injection every four weeks. Additionally, PiaSky binds to a different site on the C5 protein compared to current treatments, potentially offering a new option for patients with specific C5 gene mutations who do not respond to existing therapies.
About the COMMODORE 2 Study
The Phase III COMMODORE 2 study was a randomized, open-label trial designed to assess the effectiveness and safety of PiaSky® (crovalimab) compared to eculizumab in individuals with paroxysmal nocturnal haemoglobinuria who had not previously received C5 inhibitors. The study's co-primary efficacy endpoints included transfusion avoidance and control of hemolysis, which is the ongoing destruction of red blood cells, measured by lactate dehydrogenase levels. Adult participants were randomly assigned in a 2:1 ratio to receive either subcutaneous PiaSky every four weeks or intravenous eculizumab every two weeks. Adolescents under 18 were included in a non-randomized treatment arm, receiving subcutaneous PiaSky every four weeks.
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