Genzyme UK has been awarded a ‘Company Award’ from EURORDIS, the largest European patient organisation in the field of rare diseases.

The EURORDIS Company Award recognises the accomplishments of companies dedicated to rare diseases. Genzyme was honoured for pioneering the development and delivery of therapies for rare diseases, the main thrust of its business.

The company, which is now owned by Sanofi, was also recognised for its longstanding support of patient organisations - including EURORDIS - as well as initiatives to increase patient access to Genzyme treatments.

Genzyme markets a number of treatments for rare diseases, including Cerezyme for Gaucher’s disease and Fabrazyme for Fabry’s disease.

In recent years, however, the firm has come under pressure for rival companies such as Shire, which also make similar drugs as Genzyme. This prompted the 2010 takeover of the firm by Sanofi.

EURORDIS Award recipients are selected by the EURORDIS board of directors, based on over 100 nominations received from EURORDIS members (rare disease patient advocacy groups from across Europe), volunteers and staff, with the aim of promoting leadership and excellence for people living with rare diseases.

Genzyme’s Senior VP of rare diseases in Europe, Hilde Furberg, accepted the award this week during the EURORDIS Black Pearl Gala Dinner in Brussels, as EURORDIS recognises the sixth International Rare Disease Day on 28 February 2013.

“Genzyme has been partnering with patient advocacy organisations globally from our earliest days to be sure the community’s needs and perspectives are always represented within our organization, and the contributions of these organisations remain central to Genzyme and our ability to bring therapies to patients,” said Genzyme’s head of rare diseases, Rogerio Vivaldi.

Yann Le Cam, chief executive of EURORDIS, said: “We are honoured to acknowledge the commitment and achievements of this year’s recipients of the EURORDIS Awards on the occasion of Rare Disease Day 2013. Each of this year’s ten awardees contributes in their own unique way toward fostering the goals of cooperation embodied in this year’s Rare Disease Day slogan: ‘Rare Disorders without Borders.’”

Changes afoot in NHS for orphan drugs

From April firms such as Genzyme will be faced with a new system of having their drugs assessed for NHS funding.

Because of the rarity of the disease this drug treats – and the high costs involved – a specialised agency called the Advisory Group for National Specialised Services (AGNSS) has traditionally considered high cost, low volume treatments.

But from April, NICE will be able to assess these types medicines instead of the AGNSS, under reforms from the UK Government, meaning pharma must prepare for a new process.