Swiss pharmaceutical giant, Roche, recently announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) gave its new monoclonal antibody, Crovalimab, which is sold under the brand name PiaSky, a positive opinion. Crovalimab is meant to block the complement protein C5, which is an important part of the innate immune response.

Crovalimab functions by binding to C5 and inhibiting its activity, preventing the chain of events that results in the destruction of red blood cells in conditions like paroxysmal nocturnal hemoglobinuria (PNH), a rare and serious blood disorder that causes the destruction of red blood cells (hemolysis).

In a statement, Roche said that this significant development marks a potential shift in the treatment landscape for PNH, offering new hope for patients across Europe.

 The statement also said that adults and adolescents (12 years of age and older a weight of 40 kg) who are either new to or have previously received treatment with C5 inhibitors should use the drug.

PiaSky will become the first monthly service, if the European Commission approves. subcutaneous (SC) treatment for PNH in the European Union, with the option for patients to self-administer after adequate training. This is poised to offer a more convenient and less disruptive alternative to current C5 inhibitors that require regular intravenous (IV) infusions.

“People living with PNH face lifelong treatment, often requiring frequent intravenous infusions and time-consuming clinic visits,” Roche’s Chief Medical Officer and Head of Global Product Development, Dr. Levi Garraway, said.

“With the option to self-administer once a month, today’s recommendation may therefore offer patients and carers in Europe more freedom in their day-to-day lives,” he added.

PNH is a rare and life-threatening blood condition affecting approximately 20,000 people worldwide. It is characterized by the destruction of red blood cells by the complement system, leading to symptoms such as anemia, fatigue, and blood clots, as well as kidney disease. C5 inhibitors, which block part of the complement system cascade, have proven effective in treating PNH. The bloodstream recycles PiaSky, a novel C5 inhibitor, enabling it to bind and inhibit the C5 protein multiple times. This results in longer action in the body with a small volume of medicine, enabling SC administration every four weeks following an initial IV infusion and weekly SC loading doses in the first month of treatment.

 The Phase III COMMODORE 2 study, which demonstrated that PiaSky, administered as SC injections every four weeks, achieved disease control and was well-tolerated in people with PNH who had not previously received C5 inhibitors, forms the basis of the CHMP's recommendation. The study showed that PiaSky was non-inferior with comparable safety to eculizumab, an existing standard of care C5 inhibitor given intravenously every two weeks. The rate of adverse events in people treated with PiaSky was similar to those treated with eculizumab (78% versus 80%, respectively). Additional supportive data came from the COMMODORE 1 study in patients switching from other C5 inhibitors, as well as the COMMODORE 3 study in newly treated patients in China.

 According to the COMMODORE studies, PiaSky is already the first monthly SC treatment approved for PNH in the US, Japan, and China. A broad clinical development programme, including five ongoing Phase III studies and three earlier phase studies, is also investigating PiaSky in complement-mediated diseases such as atypical hemolytic uremic syndrome and sickle cell disease.

 In a unique way, PiaSky works by attaching to a different part of the C5 protein than other treatments. This could make it a good choice for people with certain C5 gene mutations who don't respond to other treatments.