Gloperba® is the first and only liquid oral version of the anti-gout medicine colchicine indicated for the prophylaxis of painful gout flares in ...

Scilex (SCLX) announced that FDA has approved the company’s request for the orphan drug designation for colchicine for the treatment of ...

Scilex Holding said the Food and Drug Administration granted orphan drug designation for colchicine for the treatment of pericarditis. The company's Gloperba, licensed from Romeg Therapeutics, is a ...

There have been continued calls to reform orphan drug legislation in the US and the EU, particularly the 40-year-old Orphan Drug Act and the European Union's 1999 Orphan Drug Regulation.

FDA grants orphan drug designation to HLX22 for gastric cancer, with a phase 2 trial showing improved progression-free ...

Sanofi SNY announced that the FDA has granted orphan drug designation to its investigational BTK inhibitor, rilzabrutinib, for the treatment of warm autoimmune hemolytic anemia (wAIHA) and ...

Rare diseases, often referred to as orphan diseases, affect small patient populations, yet collectively they impact more than ...

The FDA has granted orphan drug designation to rilzabrutinib for IgG4-related disease and warm autoimmune hemolytic anemia, according to a press release from the manufacturer. There are currently no ...

Inebilizumab-cdon (Uplizna; Amgen) was approved as the first and only treatment for immunoglobulin G4–related disease ...

Rhythm Pharmaceuticals, Inc. has announced that it received orphan drug designation from Japan's Ministry of Health, Labour and Welfare for setmelanotide, a treatment aimed at acquired ...

Government incentives, advances in technology, and an army of patient advocates have spun a successful market—but abuses of the system and exorbitant prices could cause a backlash. Along with ...