NHS England negotiates price cuts for ultra-rare Morquio A syndrome drugs

People in England who suffer from the ultra-rare Morquio A syndrome should in future be able to access the only drug for their condition after a ground-breaking agreement was reached between NHS England and the manufacturer.

NHS England has successfully negotiated a deal on elosulfase alfa (brand name Vimizim) with manufacturers BioMarin Pharma. The five year, fixed-fee agreement – which is the first of its kind but could provide a model for future decisions on high-cost treatments for rare conditions – will see a managed access scheme put in place for patients, as well as a mechanism to monitor how well the medicine has worked in practice before future funding decisions are taken.

NICE’s previous evaluation consultation document concluded that while the drug did improve some aspects of quality of life, the long-term benefits were uncertain. It also pointed out that the drug was expensive.

The establishment of a treatment scheme under the terms agreed by NHS England with BioMarin is now approved in draft guidance issued today, with final NICE guidance expected in December.

Confirming the deal, NHS England’s National Director of Commissioning Operations, Dame Barbara Hakin, said: “This agreement will enable us to start treating people with this debilitating condition, assess whether this treatment provides real benefits in the long term and, assuming it does, then continue to provide it in a sustainable way.

“This drug is not a cure but it can have benefits for patients, though long-term outcomes remain uncertain.

“NICE was right to highlight the very high list price, which would have a disproportionate impact on the availability of other treatments.  It is good news for all our patients that we have been able to come to a more reasonable arrangement that works for the NHS more broadly.”

Morquio A syndrome (full name Mucopolysaccharidosis IV Type A, or MPS IVA) is a rare condition that affects under 100 people in England.

It is a severely debilitating, inherited disorder caused by the lack of a key enzyme in the body which ordinarily breaks down large sugar molecules the body can’t otherwise process.

The accumulation of these molecules in the body’s cells causes a range of symptoms including joint and skeletal abnormalities, hearing and vision loss, heart valve disease, pain, fatigue, and reduced life expectancy.  The average life expectancy in people with this condition is about 25 years.

On average, around three children are born with the disorder per year in England, and 88 patients are currently identified by the MPS Society and treating centres.

Elosulfase alfa is currently the only treatment available for the cause of Morqiou A syndrome.  It works by replacing the defective enzyme via regular infusions. While it is not a cure, and patients will need to take the infusions for their entire lifetime, the treatment has been shown to slow progression of the symptoms and allow some patients a longer and better quality of life.