The FDA has awarded orphan drug designation to Cure Rare Disease’s CRD-003 for the treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy caused by biallelic mutations in the FKRP gene.
A failed July inspection of manufacturer Catalent Indiana LLC has delayed another U.S. FDA approval, the latest being that of Scholar Rock Inc.’s selective anti-latent myostatin antibody, apitegromab, which was expected to become the first therapy to enhance skeletal muscle in patients with spinal muscular atrophy.
NS Pharma Inc.’s NS-051 (NCNP-04) has been awarded orphan drug designation by the FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping.
Two months after starting the phase I/II Synrgy trial with its gene therapy, CAP-002, enrolling 12 pediatric patients with rare disease STXBP1 encephalopathy, Capsida Biotherapeutics paused the study following the death of the trial’s first patient.
Neuronos Ltd., a subsidiary of Beyond Air Inc., has announced the granting of orphan drug designation by the FDA to BA-101 for the treatment of glioblastoma (GBM). The company is advancing development of BA-101 toward first-in-human studies.
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.
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