Revir Therapeutics Inc.’s RTX-117 has been awarded orphan drug designation by the FDA for Charcot-Marie-Tooth disease. RTX-117 is a small-molecule therapy designed to activate eIF2B to restore translation of cap-dependent mRNAs to normalize protein expression.
The FDA has granted orphan drug designation to FRF-001, the FOXG1 Research Foundation’s lead gene therapy candidate for the treatment of FOXG1 syndrome. This follows the FDA’s earlier award of rare pediatric disease designation to the investigational therapy.
The U.S. FDA released a trio of draft guidances to help sponsors in developing and monitoring cell and gene therapies, as well as other regenerative medicine therapies.
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.
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