Sanofi’s big week includes a promising PhIII for an orphan disease drug, with plans for a pitch to the FDA
The biopharma R&D food chain is paying off with a plan at Sanofi to pitch regulators on a new drug for an orphan disease called cold agglutinin disease.
The pharma giant ushered out a statement Tuesday morning — after it spelled out plans to radically restructure the company, abandoning cardio and diabetes research altogether — saying that their C1s inhibitor sutimlimab had cleared the pivotal study.
Thirteen of the 24 patients in the small study met the composite endpoint, according to investigators, with “62.5% (n=15) of patients achieving a hemoglobin ≥ 12 g/dL or an increase of at least 2 g/dL and 71% (n=17) of patients remaining transfusion-free after week 5.”
Mean total bilirubin, a marker of hemolysis in CAD, achieved near normalization after the first week of treatment, said researchers. And none of the serious adverse events recorded among the patients were tied to the drug.
Sanofi and the patients are continuing on with a second leg of the study to assess durability.
“CAD is a disease in which the immune system attacks red blood cells and causes a cascade of symptoms for patients. In our study, sutimlimab achieved clinically meaningful results by impacting the central mechanism of CAD, bringing about marked improvements in patients’ hemolysis, anemia and fatigue,” said R&D chief John Reed. “We are eager to share these results with regulatory authorities beginning with the U.S. FDA in the near future in an effort to provide patients with a first-in-class targeted therapy that we believe has the potential to change the treatment paradigm for CAD.”