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Rare genetic disorder treatment recommended in final draft guidance

NICE has issued final draft guidance recommending BioMarin Pharma's Vimizim (elosulfase alfa) for the treatment of the rare genetic disorder Morquio A syndrome.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Meindert Boysen, technology appraisals programme director at NICE, said: “In our previous draft guidance the independent evaluation committee concluded that, although elosulfase alfa improved some aspects of health compromised by the disease, and that health and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



COVID-19 Updates and Daily News

Featured jobs


Add my company
Research Partnership

We are the largest independent healthcare market research and consulting agencies in the world. Trusted partner to the global pharmaceutical...

Latest intelligence

The search for treatments for Parkinson’s disease
The research requires resilience but pharma is urged to ‘keep innovating, keep trying’...
Margot Hannah, OPEN Health - A personal perspective of LGBT+ and diversity.
Ditch the Label – it’s just me....
The impact of COVID-19 on clinical trials
As all industry sectors work to assess both the impact and the optimal path forward, the effects are expected to be diverse and long-lasting...