Cuvrior Gets FDA Approval for Wilson Disease
The approval was based on data from a phase 3 study that compared Cuvrior with penicillamine in 53 adults with Wilson disease.
Inborn Errors Of Metabolism
AMPK Activator Gets Fast Track Status for X-Linked Adrenoleukodystrophy
PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator.
More Data Needed to Make Decision on Bardoxolone in Alport Syndrome
The CRL stated that the application for bardoxolone could not be approved in its current form due to a lack of data supporting claims of slowing kidney function loss and reducing kidney failure progression.
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.
Setmelanotide Gets Priority Review for Bardet-Biedl Syndrome and Alström Syndrome
The application is supported by data from a phase 3 study that evaluated the efficacy and safety of setmelanotide for the treatment of obesity and hyperphagia in patients with BBS and Alström syndrome.
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