Patients with a very rare inherited lysosomal storage disease will now get treatment with Biomarin’s Vimizim funded on the National Health Service in England and Wales after cost regulators issued a final nod for the drug.
As part of its Highly Specialised Technologies Programme, NICE has recommended Vimizim (elosulfase alfa) for the treatment of mucopolysaccharidosis type IVa (also known as MPS IVa and Morquio A syndrome), which affects just 88 people in England, under a managed access agreement between the drugmaker and NHS England.
People born with the disease lack an enzyme that breaks down large sugar molecules the body can’t utilise, leading to their accumulation in tissues and organs causing a wide range of symptoms such as joint and skeletal abnormalities, hearing and vision loss, heart valve disease and pain.
Vimizim replaces this enzyme, offering the first treatment that targets the underlying cause of the disease, at a cost of around £395,000 per patient, per year. But under the agreed managed access arrangement, all clinically suitable patients in England will be allowed treatment with the drug over the next five years, while clinical data on its use is collected for future evaluation of provision.
“The Committee concluded that the combined funding arrangements specified in the managed access agreement offered acceptable value for money in the context of the uncertainty of the clinical benefits and will be used to inform a future review of this guidance,” said Meindert Boysen, Technology Appraisals Programme Director at NICE.
Vimizim was rejected by the Scottish Medicines Consortium back in September despite a “powerful testimony” from patients about the impact of the disease. “While elosulfase alfa has the potential to improve quality of life in the short term, the case presented by the company was not robust enough to convince the Committee about the longer-term benefits when balanced against its extremely high cost,” the SMC said at the time.