Rare Pediatric Disease
FDA approves Miplyffa for Niemann-Pick disease type C
The FDA announced in a press release that it has approved Miplyffa to treat neurological symptoms associated with Niemann-Pick disease type C in patients aged 2 years and older.
FDA grants orphan drug designation for ATSN-201 for X-linked retinoschisis
ATSN-201 received orphan drug designation from the FDA for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.
Oral achondroplasia therapy receives FDA breakthrough therapy designation
An oral medication to treat children with achondroplasia has received breakthrough therapy designation from the FDA, BridgeBio Pharma announced.
Ilya Pharma’s investigational treatment receives rare pediatric disease designation
The FDA has issued a rare pediatric disease designation to Ilya Pharma for its topical emilimogene sigulactibac treatment for STING-associated vasculopathy with onset in infancy, according to a press release.
FDA grants rare pediatric disease designation to Descartes-08 for juvenile dermatomyositis
The FDA has granted rare pediatric disease designation to Descartes-08, an autologous mRNA cell therapy, for the treatment of juvenile dermatomyositis, Cartesian Therapeutics announced in a press release.
FDA grants rare pediatric disease designation for Leber congenital amaurosis gene therapy
Opus Genetics received rare pediatric disease designation from the FDA for its ocular gene therapy designed to treat patients with a form of Leber congenital amaurosis, according to a press release.
Gene therapy for X-linked retinoschisis receives rare pediatric disease designation
The FDA granted rare pediatric disease designation to ATSN-201, a gene therapy candidate for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.
FDA grants rare pediatric disease designation for Duchenne muscular dystrophy treatment
The FDA has granted rare pediatric disease designation to an investigational oligonucleotide for the treatment of boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping, according to the manufacturer.
Meta Pharmaceuticals receives FDA rare pediatric disease designation for META-001-PH
Meta Pharmaceuticals has received FDA rare pediatric disease designation for its investigational new drug META-001-PH, according to a press release.
FDA clears investigational new drug application to alpha kinase inhibitor for ROSAH syndrome
The FDA has cleared an investigational new drug application for an alpha-kinase 1 inhibitor for clinical evaluation in those with retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache, or ROSAH, syndrome.