The positive draft guidance follows the development of a managed access scheme by the company and NHS England that fulfils the conditions for the drug’s use specified by NICE in its previous draft guidance.

Elosulfase alfa treats mucopolysaccharidosis type IVa (also known as MPS IVa and Morquio A syndrome), an extremely rare - it affects around 88 people in England - but life-limiting disease. People born with the disease lack an enzyme – N-acetylgalactosamine-6-sulfatase – that breaks down large sugar molecules (glycosaminoglycans) the body’s cells can’t use. The resulting accumulation of glycosaminoglycans in the cells of tissues and organs causes a wide range of symptoms that typically appear in early childhood and worsen over time. These include joint and skeletal abnormalities, hearing and vision loss, heart valve disease, pain, fatigue, and progressive loss of endurance leading to increasing dependence on wheelchairs.

MPS IVa leads to reduced life expectancy - the average life expectancy in people with this condition is about 25 years - primarily because of respiratory failure and heart problems (63% and 15% of deaths respectively).

Elosulfase alfa is an enzyme replacement therapy consisting of the N-acetylgalactosamine-6-sulfatase enzyme. It replaces the enzyme lacking in people with MPS IVa.  

Before elosulfase alfa became available there was no treatment for the underlying disease. Because the condition is so rare and the symptoms so diverse, there is no standard treatment or pathway of care. Management may include surgery for skeletal problems, respiratory support, drugs to manage heart disease, dental and eye care, pain relief, and hearing aids and ventilating tubes to manage deafness and middle ear effusions.

Commenting on the draft guidance, Meindert Boysen, Technology Appraisals Programme Director at NICE, said: “MPS IVa is a condition that severely affects both life expectancy and quality of life. In our previous draft guidance the independent Evaluation Committee concluded that, although elosulfase alfa improved some aspects of health compromised by the disease, and that health and quality of life improved significantly in some patients after treatment, the magnitude of overall long-term benefit offered by elosulfase alfa was uncertain. The drug is also very expensive at a cost of £395,000 per patient per year. The Committee recommended that further exploration of its benefits and costs in routine clinical practice was needed.

“We are therefore pleased that the company and NHS England have agreed a scheme that will allow patients with this condition to access elosulfase alfa while at the same time generating valuable evidence on its use through research and collection of ‘real-world’ data directly relevant to patients in the UK. 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.” 

Ends

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Notes to Editors

About the draft guidance

1. The draft guidance on elosulfase alfa, including details of the Managed Access Agreement, will be available on the NICE website from 00:01, 23 November 2015.
2. The draft guidance states:

1.1 Elosulfase alfa, within its marketing authorisation, is recommended for funding for treating mucopolysaccharidosis type IVa (MPS IVa) according to the conditions in the managed access agreement for elosulfase alfa.

About elosulfase alfa

1. Elosulfase alfa (Vimizim, BioMarin) is a recombinant form of the human N‑acetylgalactosamine‑6‑sulfatase enzyme. It is intended to replace the enzyme lacking in people with mucopolysaccharidosis type IVa (MPS IVa). Elosulfase alfa has a marketing authorisation in the UK for treating MPS IVa in people of all ages. It is given by intravenous infusion, over 4 hours, once a week. The recommended dosage is 2 mg/kg body weight each week, and is anticipated to continue lifelong.
2. Elosulfase alfa is available in 5 ml vials containing 5 mg of elosulfase alfa, at a net list price of £750 per vial (excluding VAT). NICE estimates that the average cost per year for elosulfase alfa is £394,680 per patient (based on the recommended dosage of 2 mg/kg/year and an average body weight of 25.3 kg). It is estimated that up to 77 people may want to have treatment with elosulfase alfa; the cost of elosulfase alfa could amount to £30 million per year for treating this many people.
3. The company has proposed a patient access scheme, in which elosulfase alfa would be provided at a discounted cost; the discount is commercial in confidence.