FDA Accepts BLA for Crovalimab for Rare Blood Condition

The FDA has accepted Genentech’s biologics license application (BLA) for crovalimab, a novel monoclonal antibody, to treat patients with paroxysmal nocturnal hemoglobinuria (PNH).

PNH is a rare and life-threatening blood condition, which affects about 20,000 people worldwide. In PNH, red blood cells are destroyed by the complement system, part of the innate immune system. This causes symptoms such as anemia, fatigue and blood clots, and can lead to kidney disease.

Crovalimab is a novel C5 inhibitor that is recycled within the bloodstream and blocks part of the complement system cascade. If approved, crovalimab will be the first monthly subcutaneous treatment for PNH, with an option for patients to self-administer.

Levi Garraway, M.D., Ph.D.

“Crovalimab could provide an option to self-administer as infrequently as every four weeks, thereby reducing clinic visits for people with this lifelong condition,” Levi Garraway, M.D., Ph.D., chief medical officer and head of global product development, at Genentech said in a press release.

The BLA is based on results from the phase 3 COMMODORE 2 study in people with PNH who have not been previously treated with complement inhibitors. Results from the study demonstrated that crovalimab, administered every four weeks, achieved disease control and was non-inferior with comparable safety to Alexion’s Soliris (eculizumab), a current standard of care, given intravenously every two weeks.

In the study, 79.3% of patients treated with crovalimab achieved hemolysis control from week five to week 25 compared with 79.0% Solaris. Additionally, 65.7% achieved transfusion avoidance from baseline to week 25 with crovalimab and 68.1% with Soliris.

Adverse events in the study occurred in 78% of participants treated with crovalimab and 80% treated with Soliris, with the most common adverse events being an infusion-related reaction. The application also included data from the phase 3 COMMODORE 1 study, which supported the favorable benefit-risk profile of crovalimab in people with PNH switching from currently approved C5 inhibitors. Data from the COMMODORE 1 and 2 studies were recently presented at the European Hematology Association 2023 Hybrid Congress.

Regulatory applications have also been accepted in the European Union, China and Japan.