NICE draft guidance conditionally recommends elosulfase alfa (Vimizim) for treatment of very rare life-limiting genetic disorder

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.  

Commenting on the draft guidance, Professor Carole Longson, Director of the Health Technology Evaluation Centre at NICE, said: “MPS IVa is a serious condition that severely affects both life expectancy and quality of life. We are therefore pleased to be able to provisionally recommend elosulfase alfa as a treatment option for people with this condition.

“After considering the comments received as part of the consultation on the previous draft guidance, as well as the testimony of patient and clinical experts at its recent meeting, the independent Evaluation Committee felt that, although elosulfase alfa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 very expensive – the average cost per year for elosulfase alfa is £394,680 per patient based on the list price; although a confidential discount has been offered by the company as part of a patient access scheme. Overall, although the Committee was satisfied that there was sufficient evidence that some patients did well on elosulfase alfa, further exploration of its benefits and costs in routine clinical practice were needed.

“The Committee concluded that the company, clinical and patient experts and NHS England should make arrangements, as part of a managed access agreement, to generate evidence on the use of elosulfase alfa for treating MPS IVa through research and collection of ‘real-world’ data directly relevant to patients in the UK, including continued support of the MPS IVa registry. The Committee also felt that a protocol for starting and stopping treatment with the drug for clinical reasons should be developed.”

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.

NICE has not yet issued final guidance to the NHS; these decisions may change after consultation. Consultees, including the company, healthcare professionals, patient/carer organisations and members of the public are now able to comment on the preliminary recommendations which are available for public consultation until midday on 23 September. Comments received during this consultation will be fully considered by the Committee at its next meeting in October.

Ends

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Notes to Editors

About the draft guidance

1. The draft guidance on elosulfase alfa will be available on the NICE website from 00:01, 2 September 2015. Embargoed copies of the draft guidance are available from the NICE press office on request.
2. The draft guidance states:

1.1 Elosulfase alfa is recommended within its marketing authorisation, as an option for treating mucopolysaccharidosis type IVa (MPS IVa) only in the context of a managed access agreement, and provided that the company makes elosulfase alfa  available with the discount agreed in the patient access scheme.

1.2 The Committee recommends that the managed access agreement must include, as a minimum:

• A protocol that sets out the clinical criteria for starting and stopping treatment with elosulfase alfa.
• Assurance from the company that it will collaborate with relevant patient organisations and NHS England to collect the data set out in Section 5.7 in the UK and continue to support the MPS IVa registry. The data will be used by NICE to inform a review no more than 5 years after publication of the guidance.
• Agreement between the company and NHS England to set the total costs of elosulfase alfa during data collection, which is in addition to the discount in the patient access scheme, to manage financial risk.
• Agreement between the company and NHS England that ensures patients started on elosulfase alfa during the managed access agreement should be able to continue treatment until they and their NHS clinicians consider it appropriate to stop.

 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/week 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.