RARE Daily

NICE Recommends BioMarin’s Vimizin for MPS IVA

March 22, 2022

Britain’s National Institute for Health and Care Excellence, or NICE, published final draft guidance recommending elosulfase alfa for routine use in the National Health Service for the treatment of mucopolysaccharidosis type IVA.

Elosulfase alfa, made and marketed by BioMarin Pharmaceutical under the trade name Vimizin, becomes the first disease modifying treatment recommended by NICE for routine NHS use for people with this rare, severely life-limiting condition.

Mucopolysaccharidosis type IVA, also known as MPS IVA and Morquio A syndrome, which affects around 90 people in England, is an extremely rare, life-limiting, inherited lysosomal storage disease. People born with the disease lack an enzyme 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 not receiving treatment with elosulfase alfa is about 25 years—primarily because of respiratory failure and heart problems.

Elosulfase alfa replaces the enzyme lacking in people with MPS IVA and is delivered once a week through intravenous infusion.

NICE’s final draft guidance follows the collection of real-world data from 69 people who received NHS-funded treatment since 2015 as part of a managed access agreement. NICE’s close collaboration with the MPS Society, Rare Diseases Research Partners, clinicians, NHS England and the company were key to the success of the data collection, which enabled new evidence to be presented on the benefits of this treatment to patients.

Clinical trial evidence and data collected as part of the managed access agreement, along with expert clinical opinion, shows some long-term benefits with elosulfase alfa treatment which suggest it slows progression of MPS IVA.

“The arrangement to give access to elosulfase alfa while further data was collected on its clinical and cost-effectiveness was the first of its kind to have been attempted in the NHS in England,” said Helen Knight, acting interim director of medicines evaluation at NICE. “Today’s announcement demonstrates the value of this approach, with patient groups, clinicians, academics, companies, NICE and NHSE all working together to improve the lives of people with rare diseases.”

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.