The FDA designation marks a significant milestone in the development of a novel treatment for a rare neurodevelopmental disorderPlan to initiate ...

NS Pharma, a unit of Nippon Shinyaku (OTCPK:NPNKF), said on Monday that the U.S. FDA has granted orphan drug designation to ...

NS-229 is an investigational selective Janus kinase 1 inhibitor designed to prevent tissue damage caused by EGPA by suppressing immune cell activation.

A Phase 2 study evaluating TPST-1495 in patients with FAP is set to begin this year, conducted by the Cancer Prevention Clinical Trials Network and funded by the National Cancer Institute (NCI) ...

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 ...

Under the Orphan Drug Act, the FDA may grant orphan designation to a drug or biological product intended to treat a rare disease or condition, which is generally a disease or condition that ...

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

Del-desiran is the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. Del-desiran has also received Breakthrough Therapy, Orphan Drug and Fast Track designations ...

EMA orphan drug designation is awarded to drugs intended to treat rare diseases that are life-threatening or chronically debilitating Preeclampsia, a serious obstetric complication, is a leading ...

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 ...