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.
The approval was based on data from a phase 3 study that compared Cuvrior with penicillamine in 53 adults with Wilson disease.
PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator.
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.
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.
Galactosemia is a rare genetic metabolic disease caused by an inability to metabolize galactose.
Carglumic acid is a carbamoyl phosphate synthetase 1 activator.
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 is a cardiolipin peroxidation inhibitor designed to sustain and restore mitochondrial function and interrupt apoptosis in mitochondrial conditions.
Vascular EDS is the most severe subtype of Ehlers-Danlos 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.