Bausch Health Companies Inc (NYSE/TSX: BHC), a Canada-based specialty pharmaceutical company, and its gastroenterology business, Salix Pharmaceuticals, have received Orphan Drug Designation from the United States Food and Drug Administration (FDA) for rifaximin intended for the treatment of sickle cell disease, it was reported on Thursday.
The US FDA's Orphan Drug Designation program grants orphan status to medicines that are aimed at the treatment of a rare disease or condition, and incentives for firms to study and further develop medicines to bring forward novel treatments.
Joseph C Papa, chairman and CEO, Bausch Health, said, 'Early data suggests that rifaximin may be beneficial in reducing vaso-occlusive crisis in patients with sickle cell disease, and we are currently finalising trial protocols to further evaluate its potential in this patient population. 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.'
AbbVie's ELAHERE receives full US FDA approval
Johnson & Johnson's OPSYNVI receives US FDA approval
Outlook Therapeutics receives positive CHMP opinion for ONS-5010 in wet AMD treatment
Human Immunology Biosciences receives ODD from FDA for felzartamab
Eton Pharmaceuticals acquires PKU GOLIKE, expanding rare disease portfolio
Vertex receives FDA clearance for VX-407 Investigational New Drug Application for ADPKD
Phanes Therapeutics receives FDA Fast Track designation for PT886
Bio-Thera Solutions signs new licensing agreement with SteinCares
FDA accepts resubmission of Citius Pharmaceuticals, Inc's BLA for LYMPHIR
GSK Plc reports positive Phase III Jemperli data for endometrial cancer
ImmVira receives FDA Fast Track designation for MVR-T3011 IT