Please login to the form below

Not currently logged in

BioMarin's real world data sways NICE on orphan drug Vimizim

Rare genetic disorder treatment recommended in final draft guidance

NICE has issued final draft guidance recommending BioMarin Pharma's Vimizim (elosulfase alfa) for the treatment of the rare genetic disorder Morquio A syndrome.

The UK health technology assessment body had initially balked at the orphan drug's high cost, but was swayed by a combination of a previously-agreed patient access scheme and new real world data for Vimizim, which was being assessed under NICE's highly specialised technologies programme.

Meindert Boysen, technology appraisals programme director at NICE, said: “In our previous draft guidance the independent evaluation committee concluded that, although elosulfase alfa improved some aspects of health compromised by the disease, and that health and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.”

Morquio A syndrome, or MPS Iva is an extremely rare, life-limiting disease, currently affecting around 88 people in England. People born with the disease lack an enzyme - Nacetylgalactosamine-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, and the average life expectancy for patients with this conditions is about 25 years.

Symptoms include skeletal abnormalities, hearing and vision loss, heart valve disease, pain, and fatigue and progressive loss of endurance leading to increasing dependence on wheelchairs.

Before Vimizim became available there was no treatment for the underlying disease, with management of the condition falling to options such as surgery for skeletal problems, respiratory supports, drugs to manage heart disease, dental and eye care, pain relief, and hearing aids and ventilating tubes to manage deafness and middle ear effusions. 

Article by
Nikhil Patel

23rd November 2015

From: Regulatory



Featured jobs

Subscribe to our email news alerts


Add my company
Syneos Health™

Syneos Health™ is the only fully integrated biopharmaceutical solutions organization. Our company, including a Contract Research Organization (CRO) and Contract...

Latest intelligence

It’s all about patient outcomes… right?
Lessons from history: a design thinking perspective...
Emma Walmsley 2
30 Women Leaders in UK Healthcare (part 3)
Continuing our special feature on Women Leaders in UK Healthcare...
Louise Houson
30 Women Leaders in UK Healthcare
The enormous challenges facing UK healthcare mean it needs great leaders. PME’s Group Editor Andrew McConaghie introduces 30 outstanding innovators and trailblazers helping to shape the future...