Roche has announced that the European Medicine Agency’s human medicines committee has recommended approval of PiaSky (crovalimab) to treat paroxysmal nocturnal haemoglobinuria (PNH), a rare blood condition.
If approved, PiaSky will be the first monthly subcutaneous treatment for PNH in the EU, with the option of self-administration following adequate training.
Affecting approximately 20,000 people worldwide, PNH is a life-threatening blood condition where red blood cells are destroyed by the complement system in the immune system, leading to symptoms such as anaemia, fatigue and blood clots that can eventually lead to kidney disease.
The Committee for Medicinal Products for Human Use (CHMP) has recommended the novel recycling monoclonal antibody that inhibits the complement protein C5 for use in adults and adolescents aged 12 years or older with a weight of 40kg, who are either new to or have been previously treated with C5 inhibitors.
The recommendation was based on results from the phase 3 COMMODORE 2 study, which has been evaluating PiaSky in PNH patients who have not previously received treatment with C5 inhibitors.
Results showed that PiaSky, administered every four weeks, achieved disease control and was well-tolerated, with comparable safety to eculizumab, an existing standard of care C5 inhibitor.
In addition, the application included two other phase 3 studies part of a broad clinical development programme, the COMMODORE 1 study and the COMMODORE 3 study, evaluating PiaSky in PNH patients switching from currently approved C5 inhibitors and in PNH patients new to C5 inhibitor treatment in China.
Commenting on the recommendation, Levi Garraway, chief medical officer and head of global product development, Roche, said: “With the option to self-administer once a month, [the] recommendation [could] offer [PNH] patients and caregivers in Europe more freedom in their day-to-day lives.”
PiaSky, engineered by Chugai Pharmaceutical, is already approved in the US, Japan and China to treat PNH and is designed to block the complement system to address certain needs of people living with complement-mediated diseases.
A final decision from the European Commission regarding the approval of PiaSky is anticipated in the near future.
