Latest News and Press Releases

FDA grants orphan drug status to Attralus AT-02 for AL amyloidosis, a novel therapy targeting toxic amyloid removal

Eledon Pharmaceuticals Announces Orphan Drug Designation Granted to Tegoprubart for the Prevention of Allograft Rejection in Liver Transplantation

Transpire Bio today announced that the U.S. FDA has granted orphan drug designation (ODD) for two of its products in development.

European Medicines Agency recognizes potential of lead compound MP1032 in addressing a high unmet medical need in Duchenne Muscular Dystrophy (DMD)Designation is paving the way for safer long-term...

An Orphan Drug Designation Could Provide Significant Benefits to Regen SAN DIEGO, July 23, 2025 (GLOBE NEWSWIRE) -- Regen BioPharma, Inc. (OTC Pink: RGBP and RGBPP) today provides further insight...

ImCheck’s Announces EMA Orphan Drug Designation for ICT01 as Treatment for Acute Myeloid Leukemia Following the FDA ODD, European designation underscores ICT01’s potential as a novel immunotherapy...

ImCheck’s ICT01 Receives FDA Orphan Drug Designation for Treatment of Acute Myeloid Leukemia Clinical data showing unprecedented remission rates in newly diagnosed AML patients support advancing...

In an ongoing Phase 1 trial published in Nature Medicine, MB-101 was well-tolerated and 50% of patients achieved stable disease or better with two partial responses and two complete responses lasting...

SAN FRANCISCO, July 07, 2025 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of...

FDA grants Orphan Drug Designation to Medicovestor’s ADoBind MC001, a novel ADC targeting hard-to-treat pancreatic cancer.