The National Institute for Health and Care Excellence (NICE) has published draft guidance in which it has decided not to recommend BioMarin’s rare disease med Vimizin (elosulfase alfa).

The draft guidance, which has been published for public consultation, follows an evaluation of new evidence on the use of Vimizin for the treatment of mucopolysaccharidosis type 4A (also known as MPS 4A and Morquio A syndrome).

This new evidence was gathered through research and collection of real-world data as part of a managed access agreement, which has made the treatment available for NHS use since 2015.

According to NICE, this evidence from the managed access agreement demonstrates some long-term benefits with Vimizin which suggest the condition becomes stable.

Despite this, NICE’s independent committee has concluded that BioMarin’s cost-effectiveness modelling is ‘not robust or plausible’ – in particular because it does not adequately capture disease progression.

On top of this, the committee were ‘concerned’ about how the real-world data was used to model long-term wheelchair use, how the quality-of-life values of patients and carers were captured and the ‘assumptions made about body weight and drug dose’.

“We are disappointed not to be able to recommend elosulfase alfa for use within the NHS. The length of the managed access agreement has been extended twice to allow the company more time for its submission. But despite this, there are still significant concerns with the company’s analysis and modelling. It is important to recognise this recommendation is not intended to affect those people who have begun treatment with elosulfase alfa on the NHS before this guidance was published,” said Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE.

“We will continue to work with the company to try to address these concerns ahead of the next committee meeting in January 2022,” he added.

Mucopolysaccharidosis type Iva is an extremely rare and life-limiting inherited lysosomal storage disease.

People with this condition are born lacking an enzyme called N-acetylgalactosamine-6-sulfatase which breaks down large sugar molecules that the body’s cells can’t use.

As a result, the accumulation of glycosaminoglycans in the cells of tissues and organs cause a wide range of symptoms that usually appear in early childhood and worsen over time.