NEW APPROVAL | FDA Endorses Piasky for Paroxysmal Nocturnal Hemoglobinuria
The Food and Drug Administration (FDA) has granted approval for Piasky (crovalimab-akkz) for the treatment of adults and pediatric patients aged 13 and older with rare anaemia paroxysmal nocturnal hemoglobinuria (PNH), provided they weigh at least 40kg.
Piasky, developed by Roche, is a monoclonal antibody that targets complement protein C5. Crovalimab binds to C5 to prevent its cleavage, thus preventing the formation of the membrane attack complex (MAC). This inhibits terminal complement-mediated intravascular haemolysis. This approval is substantiated by data from the phase 3 COMMODORE 2 study (ClinicalTrials.gov Identifier: NCT04434092), which compared crovalimab to eculizumab in PNH patients who had not previously received complement inhibitor therapy.
Participants in the study were randomly allocated in a 2:1 ratio to receive either crovalimab (n=135) or eculizumab (n=69). The primary endpoints were the proportion of patients achieving transfusion avoidance (defined as being free from packed red blood cell transfusions) and the proportion of patients achieving haemolysis control (measured by lactate dehydrogenase levels ≤1.5 times the upper limit of normal) from baseline through week 25.
The results indicated that treatment with crovalimab met both primary endpoints, demonstrating non-inferiority to eculizumab with respect to transfusion avoidance (65.7% vs. 68.1%, respectively; treatment difference, -2.8% [95% CI, -15.7, 11.1]) and hemolysis control (79.3% vs. 79.0%, respectively; odds ratio, 1.02% [95% CI, 0.57-1.82]).
Furthermore, crovalimab was also found to be non-inferior to eculizumab regarding the proportion of patients with breakthrough haemolysis and stabilized haemoglobin levels.
The efficacy of crovalimab was also assessed in 12 pediatric patients with PNH. Out of these, nine patients achieved transfusion avoidance and haemoglobin stabilization, with no occurrences of breakthrough hemolysis during the 24-week treatment duration.
The most frequently reported adverse reactions associated with crovalimab included infusion-related reactions, respiratory tract infections, viral infections, and Type III hypersensitivity reactions.
Piasky is supplied as 340mg/2mL solution in a single-dose vial for intravenous or subcutaneous use.
Furthermore, on 27 June 2024, the Committee for Medicinal Products for Human Use (CHMP) in the EU adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Piasky.
Source: OncLive article / Roche, Media Press Release
