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Tue, Jul 29, 2025

Orphan drug

Home » Topics » Regulatory » Orphan drug
  • Doctor examining child in wheelchair
    July 23, 2025

    Precision Biosciences’ PBGENE-DMD gains US orphan drug designation

    The U.S. FDA has granted Precision Biosciences Inc.’s PBGENE-DMD orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). PBGENE-DMD uses two complementary Arcus nucleases delivered via a one-time administration in a single AAV to excise exons 45-55 of the dystrophin gene in order to restore near full-length dystrophin protein within the body to improve functional outcomes.
  • July 17, 2025

    FDA grants orphan drug designation to Amphix Bio’s AMFX-200

    Amphix Bio LLC has been granted U.S. FDA orphan drug designation for its lead candidate AMFX-200 for the treatment of acute spinal cord injury (SCI). AMFX-200 is an FGFR (fibroblast growth factor receptor) and ITGB1 (integrin β1) agonist peptide amphiphile scaffold. In preclinical models of acute SCI, a single injection of AMFX-200 into the spinal cord enabled motor neurons from the brain to regrow past the injury site.
  • Obesity doctor patient weight
    July 16, 2025

    Palatin Technologies reports efficacy of PL-7737 in obesity model

    Palatin Technologies Inc. has announced preclinical results for PL-7737, an oral selective melanocortin MC4 receptor (MC4R) agonist, showing effectiveness in rodent models of obesity.
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