Rare Pediatric Disease
FDA approves Gomekli for neurofibromatosis type 1 with plexiform neurofibromas
The FDA has approved an oral, small molecule MEK inhibitor for adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection.
FDA receives biologics license application for apitegromab in spinal muscular atrophy
A Boston-area biopharmaceutical firm has submitted a biologics license application to the FDA for its investigational, fully human monoclonal antibody to treat individuals with spinal muscular atrophy.
Part B of phase 1/2 X-linked retinoschisis gene therapy trial initiated
Atsena Therapeutics initiated part B of the phase 1/2 LIGHTHOUSE study investigating ATSN-201 for the treatment of X-linked retinoschisis, according to a press release.
Retinitis pigmentosa therapy receives rare pediatric disease designation
The FDA granted rare pediatric disease designation to VG901 for the treatment of retinitis pigmentosa caused by mutations in the CNGA1 gene, according to a press release from ViGeneron.
Gene therapy receives rare pediatric disease designation for inherited retinal dystrophy
The FDA granted rare pediatric disease designation for HORA-PDE6b, a gene therapy for patients with inherited retinal dystrophy caused by mutations of the PDE6B gene, according to a press release from eyeDNA Therapeutics.
Atsena completes dosing in part A of X-linked retinoschisis gene therapy trial
Atsena Therapeutics completed patient dosing in part A of the phase 1/2 LIGHTHOUSE clinical trial investigating a gene therapy designed for the treatment of X-linked retinoschisis, according to a press release.
FDA grants rare pediatric disease, fast track designations to Stargardt disease therapy
The FDA granted rare pediatric disease and fast track designations to gildeuretinol, an investigational oral therapy for the treatment of Stargardt disease, according to a press release from Alkeus Pharmaceuticals.
FDA grants BPGbio rare pediatric disease designation for epidermolysis bullosa treatment
The FDA granted rare pediatric disease designation for BPM31510T for the treatment of epidermolysis bullosa, BPGbio announced in a press release.
FDA approves second Niemann-Pick disease type C treatment
The FDA has approved Aqneursa as an oral treatment for neurological symptoms associated with Niemann-Pick disease type C, a rare genetic disease, in adults and children weighing at least 15 kg, according to a press release.
Cell-based therapies, enzyme inhibitors at vanguard of muscular dystrophy treatment
ORLANDO, Fla. — Cell-based therapies and enzyme inhibitors currently in development are likely to become the next-generation treatments for ambulatory and non-ambulatory forms of muscular dystrophy, according to a speaker.