NICE recommends elosulfase alfa for Morquio A syndrome

16 December 2015

The UK's National Institute for Health and Care Excellence (NICE) has recommended Vimizim (elosulfase alfa) for the treatment of a very rare life-limiting genetic disorder, it has been announced.

The final guidance recommends the drug for the treatment of Morquio A syndrome, also known as mucopolysaccharidosis type IVa and MPS IVa.

It follows the development of a managed access scheme by US biotech company BioMarin Pharmaceutical (Nasdaq: BMRN) and NHS England that fulfils the conditions for the drug’s use specified by NICE in its previous guidance.

This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Try before you buy

Free

7 day trial access

Take a Free Trial

All the news that moves the needle in pharma and biotech
Exclusive features, podcasts, interviews, data analyses and commentary from our global network of life sciences reporters.
Receive The Pharma Letter daily news bulletin, free forever.

Become a subscriber

£820

Or £77 per month

Subscribe Now

Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
Daily roundup of key events in pharma and biotech.
Monthly in-depth briefings on Boardroom appointments and M&A news.
Choose from a cost-effective annual package or a flexible monthly subscription

The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK