NICE backs Vimizim for routine NHS use

The UK’s medicine costs watchdog the National Institute for Health and Care Excellence (NICE) has published final draft guidance recommending US biotech company BioMarin Pharmaceutical (Nasdaq: BMRN) Vimizim (elosulfase alfa) for routine use in the National Health Service (NHS) for the treatment of mucopolysaccharidosis type 4A (also known as MPS 4A and Morquio A syndrome).

Elosulfase alfa becomes the first disease modifying treatment recommended by the NICE for routine NHS use for people with this rare, severely life-limiting condition.

The final draft guidance follows the collection of ‘real-world’ data from 69 people who received NHS-funded treatment since 2015 as part of a managed access agreement.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