RWD shakes confidence in elusulfase alfa for MPS 4A

Draft guidance published by NICE (London, UK) has not recommended the use of elusulfase alfa, otherwise known as Vimizin®, for mucopolysaccharidosis type 4A (MPS 4A) on the NHS. The decision comes after new research evidence was collected, including real-world data (RWD). MPS 4A is a rare condition that shortens the life of individuals, by lacking the N-acetylgalactosamine-6-sulfatase enzyme. Consequently, glycosaminoglycans accumulate in tissues and organs, leading to skeletal abnormalities, vision loss and a gradually increasing reliance on wheelchairs. Life expectancy for individuals with MPS 4A is reduced; on average, to around 25 years old. Since the treatment has been available...