Rare Pediatric Disease

Treatment with vamorolone, an anti-inflammatory steroidal drug, improved motor outcomes in boys with Duchenne muscular dystrophy compared with corticosteroid-naive individuals, according to findings published in PLoS Medicine.

Innova Therapeutics Inc. has been granted rare pediatric disease designation by the FDA for its IVT-8086 osteosarcoma treatment.

The FDA granted rare pediatric disease designation to EDIT-301, an investigational autologous cell therapy for the treatment of sickle cell disease, according to the agent’s manufacturer.

QR-421a has received rare pediatric disease designation from the FDA for the treatment of retinitis pigmentosa, according to a press release from ProQR Therapeutics.

The FDA granted rare pediatric disease designation to ARU-1801, an investigational autologous gene therapy, for the treatment of sickle cell disease, according to the agent’s manufacturer.

Sepofarsen has received rare pediatric disease designation from the FDA for the treatment of Leber’s congenital amaurosis 10, according to a press release from ProQR Therapeutics.

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