FDA Extends Review Period for Gene Therapies for Beta-Thalassemia, Cerebral Adrenoleukodystrophy
The FDA extended the target dates for beti-cel and eli-cel to allow time to review additional clinical information for both applications.
Inborn Errors Of Metabolism
Intravitreal Gene Therapy Fast Tracked for X-Linked Retinitis Pigmentosa
4D-125 is a gene therapy candidate designed to deliver a functional copy of the RPGR gene to photoreceptors in the retina.
Antisense Oligonucleotide Fast Tracked for Myotubular and Centronuclear Myopathies
The Company is currently evaluating DYN101 in the phase 1/2 UNITE-CNM trial in patients 16 years of age and older with CNM caused by mutations in the MTM1 or DNM2 gene.
FDA Requests Clinical Outcomes Data for Galactosemia Treatment
Galactosemia is a rare genetic metabolic disease caused by an inability to metabolize galactose.
Generic Version of Carbaglu Now Available
Carglumic acid is a carbamoyl phosphate synthetase 1 activator.
Gene Therapy Gets Priority Review for Cerebral Adrenoleukodystrophy
Elivaldogene autotemcel (eli-cel) is a one-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells, resulting in the production of ALDP.
Visomitin Gets Orphan Drug Status for Leber Hereditary Optic Neuropathy
Visomitin is a cardiolipin peroxidation inhibitor designed to sustain and restore mitochondrial function and interrupt apoptosis in mitochondrial conditions.
Enzastaurin Gets Orphan Drug Status for Vascular Ehlers-Danlos Syndrome
Vascular EDS is the most severe subtype of Ehlers-Danlos syndrome.
FDA Panel Votes on Bardoxolone for CKD Caused by Alport Syndrome
The Committee voted “no on the question of whether the provided evidence demonstrated that bardoxolone is effective in slowing the progression of CKD in patients with Alport syndrome and that its benefits outweigh its risks.”
November 2021: 5 Notable Drug Approvals
The new approvals include Besremi, Fyarro, Livtencity, Voxzogo, and Vuity.
