The FDA has approved Gamifant as the first-ever treatment for macrophage activation syndrome in Still’s disease for adults ...

Shares of Cero Therapeutics Holdings drew increased retail investor chatter on Tuesday after the company said that the FDA ...

Jaguar Health (NASDAQ:JAGX) (Jaguar) family company Napo Pharmaceuticals (Napo) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation (ODD) to ...

NEW YORK, May 14, 2025 (GLOBE NEWSWIRE) -- Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has received seven ...

New FDA Orphan Drugs: Amplimexon, AP23573, TQ-1017 The FDA has approved orphan drug status for imexon injection (Amplimexon), a non-prodrug rapamycin analog (AP23573), and an abuse-deterrent once ...

PYC Therapeutics Limited (AU:PYC) has released an update. PYC Therapeutics has received Orphan Drug Designation from the US FDA for its pioneering drug candidate, VP-001, aimed at treating ...

Mesoblast Secures FDA Orphan-Drug Approval For Ryoncil May 14, 2025 — 11:06 pm EDT Written by RTTNews.com for RTTNews -> ...

The Food and Drug Administration (FDA) has granted Orphan Drug designation to LQT-1213, a first-in-class serum glucocorticoid regulated kinase 1 (SGK1) inhibitor, for the treatment of long QT ...

FDA Approves Orphan-Drug Designation for Jaguar Health’s Crofelemer for Treatment of Diarrhea in Cholera ACCESSWIRE. Updated December 17, 2024 8:46 AM.

NEW YORK, May 14, 2025 (GLOBE NEWSWIRE) -- Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has received seven ...