Oddsmakers placing their bets on which drugs will be in play for round 3 of the U.S. Inflation Reduction Act (IRA) price negotiations are doing some reshuffling, thanks to an orphan drug provision tucked into the Trump administration’s One Big Beautiful Bill Act (OBBBA) that was signed into law on the Fourth of July.
The FDA has granted orphan drug designation to Vanda Pharmaceuticals Inc.’s VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera (PV).
Arnatar Therapeutics Inc.’s ART-4, an upregulating antisense oligonucleotide (ASO) therapeutic candidate for the treatment of Alagille syndrome (ALGS), has been awarded U.S. orphan drug and rare pediatric disease designations by the FDA. Approximately 95% of ALGS cases are caused by haploinsufficient mutations in the Jagged-1 (JAG1) gene, leading to insufficient JAG1 protein levels and impaired liver bile duct development.
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.
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.
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.
The U.S. FDA has granted orphan drug designation to Klotho Neurosciences Inc.’s secreted-Klotho (s-KL) promoter, gene and delivery system (KLTO-202 or s-KL-AAV.myo) for the treatment of amyotrophic lateral sclerosis (ALS).
Brightpath Biotherapeutics Co. Ltd.’s iPS cell-derived BCMA CAR-natural killer T cell therapy candidate has been awarded orphan drug designation by the FDA for the treatment of multiple myeloma.
Diagonal Therapeutics Inc.’s DIAG-723 has been awarded orphan drug designation by the FDA for the treatment of hereditary hemorrhagic telangiectasia (HHT). Additionally, the EMA has provided a positive opinion for orphan drug designation, confirming that DIAG-723 meets the criteria for designation as an orphan drug in the E.U.
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