Patients with a very rare inherited lysosomal storage disease are now likely to get treatment with Biomarin’s Vimizin funded on the National Health Service in England and Wales after all, under a managed access agreement between the drugmaker and NHS England.

The National Institute for Health and Care Excellence has provisionally recommended Vimizin (elosulfase alfa) for the treatment of mucopolysaccharidosis type IVa (also known as MPS IVa and Morquio A syndrome), a condition affecting just 88 people England that carries a life expectancy of just 25 years.

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. However, at £395,000 per patient, per year, it doesn’t come cheap. 

The Institute had wavered over recommending the drug because of its high cost and uncertainty over the magnitude of its benefit. However, an agreed managed access arrangement will allow all clinically suitable patients in England treatment with the drug over the next five years, while clinical data on its use is collected for future evaluation of provision.

Acceptable value for money

“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,” explained Meindert Boysen, Technology Appraisals Programme Director at NICE.

Christine Lavery, chief executive of the MPS Society, said the decision has given patients “hope for a future where their improved health allows them to reach their full potential”.

In yet another example of the postcode lottery of care, 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.