NHS England defers decision on Morquio drug by '12-16 weeks'

NICE has said that doubts exist about the benefits and cost of this treatment in its recent draft guidance, and is therefore “minded not to recommend” the treatment for patients with Morquio A Syndrome. The manufacturer now has the opportunity to present additional evidence to NICE to address these concerns. If NICE ultimately does recommend the NHS funds this treatment then NHS England will do so. NHS England believes the manufacturer has an ethical duty to continue to support those patients it enrolled in clinical trials of elosulfase alfa whilst the NICE process is still ongoing.

BioMarin is disappointed by NHS England’s decision not to provide interim funding for Vimizim. Vimizim is the only enzyme replacement therapy for people with Morquio A syndrome, a rare genetic condition that is progressively degenerative and life limiting. BioMarin continues to work closely with all stakeholders to ensure that a long term funding solution for Vimizim is found for all patients with Morquio A syndrome, so that they are able to access this treatment in England.