EU Approves Roche's PiaSky as First Monthly Subcutaneous Treatment for PNH
Tuesday, August 27, 2024
Roche announced that the European Commission has granted approval for PiaSky® (crovalimab), a new monoclonal antibody that inhibits the complement protein C5, for treating adults and adolescents (12 years and older, weighing 40 kg or more) with paroxysmal nocturnal haemoglobinuria (PNH).
PiaSky® (crovalimab) is a novel monoclonal antibody that inhibits the complement protein C5, designed to block the complement system—a crucial part of the immune system. Engineered by Chugai Pharmaceutical, PiaSky offers the potential for self-administration following proper training, with initial treatment involving an intravenous infusion and weekly SC doses during the first month.
This condition, which is rare and life-threatening, leads to the destruction of red blood cells by the complement system—a key part of the body's innate immune response. Symptoms of PNH include anaemia, fatigue, and blood clots, and the condition can lead to kidney disease.
PNH patients often face the burden of frequent and lengthy intravenous infusions, which can disrupt both their lives and those of their families. PiaSky offers a more flexible treatment option, allowing for less frequent administration at home, providing patients with greater independence.
PiaSky is the first subcutaneous (SC) treatment for PNH available in the European Union, offering patients the option to self-administer the medication after receiving proper training. This marks a significant advancement compared to existing C5 inhibitors, which require regular intravenous infusions, and could greatly reduce the treatment burden on patients and caregivers.
PiaSky provides a new option in PNH treatment by combining effective disease control with a cutting-edge recycling technology that allows for monthly subcutaneous administration. This will reduce the treatment burden for many PNH patients in Europe.
PiaSky is now approved as the first monthly SC treatment for PNH in multiple regions, including the US and Japan, based on the COMMODORE studies. Ongoing clinical trials continue to explore its potential in treating various complement-mediated diseases, including PNH, atypical haemolytic uremic syndrome, and sickle cell disease.
PiaSky functions by binding to C5, blocking the final step of the complement cascade, thereby providing rapid and sustained inhibition. Its recycling mechanism allows for smaller volume SC administration every four weeks. Additionally, PiaSky targets a different C5 binding site compared to existing treatments, offering a potential option for patients with specific C5 gene mutations who do not respond to current therapies.
C5 inhibitors have been proven effective in managing PNH, and PiaSky has been developed to address the limitations of existing treatments. The innovative recycling technology of PiaSky allows for monthly SC administration by enabling the medicine to repeatedly bind to and inhibit the C5 protein, thus maintaining its efficacy with a smaller dosage.
This approval was supported by results from the Phase III COMMODORE 2 study, which involved PNH patients who had not previously been treated with C5 inhibitors. The study showed that PiaSky, administered via SC injections every four weeks, provided effective disease control and was well-tolerated.
It was found to be non-inferior to eculizumab, an existing standard C5 inhibitor, with a similar safety profile. Additional data from the COMMODORE 1 and COMMODORE 3 studies further supported this approval, including results from patients in China who were new to C5 inhibitor treatment.
The COMMODORE 2 study is a Phase III, randomised, open-label trial comparing the efficacy and safety of PiaSky® (crovalimab) with eculizumab in PNH patients who have not previously been treated with C5 inhibitors. The primary efficacy measures were transfusion avoidance and control of haemolysis, as indicated by lactate dehydrogenase levels. Adult participants were randomised in a 2:1 ratio to receive either SC PiaSky every four weeks or intravenous eculizumab every two weeks. Adolescents under 18 were included in a non-randomised treatment arm and received SC PiaSky every four weeks.
Source: roche.com
