NICE firms up NHS use of Vimizim for Morquio A syndrome

In 2015, NICE said that BioMarin's Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome, while real-world data was collected on its efficacy. Now – almost seven years later – it has upgraded the drug to routine NHS use.

In the meantime, NICE ha collected clinical data from 69 people treated with Vimizim (elosulfase alfa), which suggests that it is able to slow the progression of Morquio A – also known as mucopolysaccharidosis type 4A.

Morquio stunts growth in children born with the disease, squashing organs and causing severe disability. The average life expectancy is around 25 years, primarily because of respiratory failure and heart problems.

The final draft guidance removes uncertainty for patients in England and Wales with the disease, and has been welcomed by the MPS Society, which said the recommendation and continued access to the treatment "will give hope to people with MPS 4A, their families and carers of a better future."

Its an endorsement of NICE's approach of allowing access whilst collecting long term clinical and cost effectiveness data, the first time this was attempted by the NHS in England.

"It means this revolutionary drug, which works by replacing a missing enzyme that is needed to break down sugar, will help almost 100 children a year, giving them and their families real hope of better quality of life," said NHS medical director Prof Steve Powis.

Vimizim was launched in the UK with a price of £395,000 per patient per year, making it one of the most expensive drugs used by the NHS, so the initial agreement with NICE – 18 months after the drug was approved in Europe – came as a massive relief to the patient community.

The decision to allow access came after an acrimonious period in which the MPS Society clashed with NHS England, winning a legal victory when a court ruled that criteria for assessing treatments was unfair.

NHS England handed the decision on access to NICE, which lead a collaboration between the MPS Society, Rare Diseases Research Partners, clinicians, NHS England and BioMarin to collect the real-world data on Vimizim. The company also offered a confidential discount on its list price.

"It has been a long and at times difficult journey, but due to the willingness of all parties to find innovative solutions…the long-term future of many within our MPS IVA community has been greatly improved," said the organisation's chief executive Bob Stevens.

"We want to thank the patients, clinicians and staff who have worked together to make this possible," he added.

Given by intravenous infusion once a week, Vimizim replaces the enzyme lacking in people with MPS 4A. It is BioMarin's top-selling drug, on course to make $650 to $700 million this year.