Rare Pediatric Disease

The FDA has approved Lenmeldy as the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy.

The FDA has granted rare pediatric disease designation to an investigational therapy for the treatment of Duchenne muscular dystrophy in those with mutations amenable to exon 44 skipping, according to the manufacturer.

The FDA has granted rare pediatric disease designation to an alpha-kinase 1 inhibitor to treat patients with retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache, or ROSAH, syndrome.

The first two female pediatric patients with Rett syndrome have been dosed in an ongoing phase 1/2 trial of investigational AAV9 gene therapy NGN-401, according to the manufacturer.

A Bay Area biotech company announced the FDA has lifted the partial clinical hold on EryDel SpA’s investigational new drug application for EryDex to treat ataxia-telangiectasia, a rare pediatric neurological disorder.

HG004, a gene replacement therapy, received rare pediatric disease designation from the FDA for the treatment of inherited retinal diseases caused by RPE65 mutations, according to a press release from HuidaGene.

The FDA has granted rare pediatric disease designation to vitamin A metabolic and reparative respiratory drugs manufactured by Advent Therapeutics to stop the development of bronchopulmonary dysplasia, according to a press release.

The FDA has approved Skyclarys, Reata Pharmaceuticals’ oral, once-daily medication to treat Friedreich’s ataxia in people aged 16 years and older.

SEATTLE — Clinicians should consider disease-modifying therapeutics when treating patients with rare pediatric disorders, according to an expert at the 2022 American Academy of Neurology annual meeting.

The FDA granted rare pediatric disease designation to IMX-110 for treatment of children with rhabdomyosarcoma.