Roche’s PNH treatment PiaSky now approved in the EU
Patients can be trained to self-administer therapy
The European Commission has approved Roche’s PiaSky (crovalimab) to treat adults and adolescents with paroxysmal nocturnal hemoglobinuria (PNH), ages 12 and older who weigh at least 40 kilograms (88 pounds).
PiaSky is the first treatment for PNH in the European Union that’s administered subcutaneously (under the skin) once a month, and patients can be trained to administer it themselves. The therapy will likely be more convenient than other, similar treatments, which require regular infusions.
In PNH, a part of the immune system called the complement system becomes activated and causes hemolysis, or the breakdown of circulating red blood cells, resulting in a range of symptoms. Patients with anemia, which refers to low numbers of red blood cells, often require blood transfusions.
Like Soliris (eculizumab) and Ultomiris (ravulizumab), PiaSky blocks complement activation by targeting a protein called C5. However, PiaSky is a novel recycling antibody that acts longer in the body than Soliris or Ultomiris, which generally require regular intravenous (into-the-vein) infusions.
“The PiaSky approval brings a new option to the PNH treatment landscape, combining the disease control achievable through C5 inhibition with a cutting-edge recycling technology that enables monthly subcutaneous administration,” Levi Garraway, MD, PhD, Roche’s chief medical officer and head of global product development, said in a company press release.
More flexible PNH treatment ‘essential’
“More flexible treatment options such as PiaSky, which are just as effective but less frequent and can be given more quickly at home, are essential to give people with PNH greater control over their treatment and more independence,” said Alexander Röth, MD, a blood disease specialist at University Hospital Essen in Germany. “People with PNH are often burdened with life-long, frequent intravenous infusions with time-consuming clinic visits, meaning that their lives, as well as their caregivers’ and families’ lives, may revolve around the demands of their treatment,” Röth said.
As with its recent approval in the U.S., the approval of PiaSky by the European Commission was mainly based on evidence from COMMODORE 2 (NCT04434092), a Phase 3 clinical trial that enrolled 204 people with PNH who had not previously been treated with a C5 inhibitor.
The study showed that patients treated with PiaSky once monthly and those treated with Soliris every other week achieved similar results in terms of hemolysis control and need for blood transfusions. The rates of side effects were also comparable in the two treatment groups.
The application requesting the approval of PiaSky also included supportive data from two additional Phase 3 clinical studies: COMMODORE 1 (NCT04432584), which enrolled people with PNH switching from currently approved C5 inhibitors, and COMMODORE 3 (NCT04654468), which enrolled people new to C5 inhibitors in China.
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