NICE Recommends Elosulfase Alfa for Morquio A Syndrome
The UK’s National Institute for Health and Care Excellence (NICE) has recommended the routine use of BioMarin’s Vimizin (elosulfase alfa) by the National Health Service (NHS) for patients with a rare skeletal disorder, Morquio A syndrome.
The costly once-a-week intravenous infusion replaces the enzyme lacking in people with the syndrome. The enzyme is needed to break down large sugar molecules that the body’s cells are unable to use.
NICE previously rejected the drug in November 2021 because of its high price. Vimizin costs more than $1,000 for each 5-milligram vial. The agency did not disclose the new price that was negotiated with the company for NHS use.
NICE estimates that 90 NHS patients will gain routine access to the product.
Mucopolysaccharidoses comprise a group of lysosomal storage disorders caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG). Mucopolysaccharidosis IVA (MPS IVA, Morquio A Syndrome) is characterized by the absence or marked reduction in N-acetylgalactosamine-6-sulfatase activity. The sulfatase activity deficiency results in the accumulation of the GAG substrates, KS and C6S, in the lysosomal compartment of cells throughout the body. The accumulation leads to widespread cellular, tissue, and organ dysfunction. Vimizim is intended to provide the exogenous enzyme N-acetylgalactosamine-6-sulfatase that will be taken up into the lysosomes and increase the catabolism of the GAGs KS and C6S. Elosulfase alfa uptake by cells into lysosomes is mediated by the binding of mannose-6-phosphate-terminated oligosaccharide chains of elosulfase alfa to mannose-6-phosphate receptors.
In the absence of an animal disease model that recapitulates the human disease phenotype, elosulfase alfa pharmacological activity was evaluated using human primary chondrocytes from two MPS IVA patients. Treatment of MPS IVA chondrocytes with elosulfase alfa induced clearance of KS lysosomal storage from the chondrocytes.
March 22, 2022
