FDA grants Orphan Designation to Bausch’ Rifaximin for SCD

“Early data suggests that rifaximin may be beneficial in reducing vaso-occlusive crisis in patients with sickle cell disease, and we are currently finalizing trial protocols to further evaluate its potential in this patient population,” said Joseph C. Papa, chairman and CEO, Bausch Health. “We expect to start a Phase 2 trial in the first half of 2021 evaluating a novel rifaximin formulation for a potential sickle cell disease treatment, and we are hopeful to be able to bring this investigational oral formulation forward to help sickle cell patients.” 

Approximately 100,000 Americans have sickle cell disease, which is a group of inherited red blood cell disorders that affects hemoglobin, the protein that carries oxygen through the body. Patients with sickle cell disease have crescent or “sickle” shaped red blood cells that can block blood flow through the body, which can lead to serious problems, including stroke, eye problems, infections and unpredicted episodes of vaso-occlusive crisis, which is described as sharp, intense, stabbing or throbbing pain that can strike nearly anywhere in the body and often requires intravenous pain medication. Sickle cell disease is a lifelong illness, and its only cure is a blood and bone marrow transplant.1