Orphan Drug Production

QR-1123, a first-in-class investigational antisense oligonucleotide for the treatment of vision loss associated with autosomal dominant retinitis pigmentosa, received an orphan drug designation from the FDA, ProQR announced in a press release.

The FDA announced it has approved elexacaftor/ivacaftor/tezacaftor, the first triple combination therapy for patients with the most common cystic fibrosis mutation.

OCU400, a gene therapy candidate, has received a second orphan drug designation from the FDA for the treatment of CEP290 mutation associated retinal disease, Ocugen announced in a press release.

The FDA has granted orphan drug designation to ADP-A2M4, an investigational specific peptide-enhanced affinity receptor T-cell therapy, for the treatment of soft tissue sarcomas.