FDA Accepts Biologics License Application for Crovalimab for Paroxysmal Nocturnal Hemoglobinuria


Crovalimab achieved disease control and non-inferiority compared to eculizumab, which is the current standard-of-care for individuals with paroxysmal nocturnal hemoglobinuria.

The FDA has accepted a biologics license application (BLA) for crovalimab (Genentech), an investigational anti-C5 recycling monoclonal antibody, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).1 The BLA was based on results from the phase 3 COMMODORE 2 study (NCT04434092), which demonstrated that individuals with PNH achieved disease control and the treatment was well tolerated.1

Image credit: Richelle | stock.adobe.com

Image credit: Richelle | stock.adobe.com

“This filing acceptance reinforces the value of crovalimab, which was engineered to be recycled in the bloodstream with the goal of offering a sustained response while reducing treatment burden,” Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development at Genentech, said in a statement. “Crovalimab could provide an option to self-administer as infrequently as every 4 weeks, thereby reducing clinic visits for [individuals] with this lifelong condition.”1

Crovalimab is recycling monoclonal antibody designed to block a vital part of the immune system that acts as the first line of defense against infections.1 The COMMODORE 2 study included individuals with PNH who had not been previously treated with complement inhibitors.1 In the study, crovalimab achieved disease control and non-inferiority when used every 4 weeks as a subcutaneous injection compared to eculizumab, which is the current standard-of-care for this patient population.1,2 The drug was also found to be safe and effective for those who have not been previously treated with a complementary inhibitor.2

Adverse events in the study occurred in approximately 78% of those treated with crovalimab and 80% treated with eculizumab, with the most common being infusion-related reactions.1

The study’s co-primary endpoints were measure transfusion avoidance and control of hemolysis, the ongoing destruction of red blood cells measured by lactate dehydrogenases levels.1 Individuals in the study were randomized 2:1 to be treated with crovalimab every 4 weeks subcutaneously or eculizumab every 2 weeks intravenously. Individuals aged less than 18 years were included in a non-randomized treatment arm, with crovalimab administered every 4 weeks subcutaneously.1 Furthermore, the study authors found that crovalimab can control hemolysis and help individuals avoid blood transfusion.2

Results from the phase 3 COMMODORE 1 study (NCT04432584) were also used for the BLA, demonstrating a consistent benefit-risk profile of crovalimab for those with PNH who were switching from their currently approved C% inhibitors.1 The study was used to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic properties of the drug for those who were previously on an approved C5 inhibitor.2

In the study, investigators included individuals aged 18 years and older who were currently treated with eculizumab, Additionally, the non-randomized arm of the study included pediatric patients who were currently treated with eculizumab, those treated with ravulizumab, those treated with off-label doses of eculizumab, or individuals with a known mutation in the C5 gene who do not respond to current therapies.1

The data from both studies were recently presented at the European Hematology Association 2023 Hybrid Congress. The data will also be submitted to other regulatory authorities around the world, with submission ongoing.1

The drug is currently being evaluated in atypical hemolytic uremic syndrome, sickle cell disease, and other complement mediated diseases, according to the statement.1


  1. FDA accepts application for Genentech’s crovalimab for the treatment of PNH, a rare life-threatening blood condition. News release. Genentech. September 4, 2023. Accessed September 5, 2023. https://www.gene.com/media/press-releases/15000/2023-09-04/fda-accepts-application-for-genentechs-c
  2. Hunter E. Crovalimab shows non-inferiority to current standard of care for paroxysmal nocturnal hemoglobinuria. Pharmacy Times. February 8, 2023. Accessed September 5, 2023. https://www.pharmacytimes.com/view/crovalimab-shows-non-inferiority-to-current-standard-of-care-for-paroxysmal-nocturnal-hemoglobinuria
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