Eurordis is relentlessly striving to improve its governance, and transparency policies and practices. Two new steps are of direct interest to you: we have adopted guidelines on the development of position papers, which you can find in a new section of our website, along with information on the European Public Affairs Committee (EPAC); we have also adopted a new approach to identify potential Eurordis representatives in EU decision making committees and expert groups. When time permits, we will call for expressions of interest from our members; a job description will be included. Candidates will then go through a selection process. Two such calls are launched in this newsletter to look for future representatives of the rare disease patient community in the:
a) EMEA Paediatric Committee,
b) EMEA Patients and Consumers Working Party.
These are two high-profile, demanding and challenging positions of great importance for people living with a rare disease in Europe. We are looking forward to receiving many expressions of interest.

Yann Le Cam
Chief Executive Officer

An unprecedented comparative study of EU and US orphan drug development initiated by Eurordis and conducted in partnership with the FDA and the EMEA in 2006, has led to an official proposal to develop a parallel orphan drug designation procedure on both sides of the Atlantic. A potentially great outcome for rare disease patients, who could benefit from faster access to orphan drugs.

The Eurordis Rare Disease Patient Solidarity Project will culminate in the creation of new networks to better service patients living with rare diseases throughout Europe, among which a network of help lines. Seven help lines are currently partnering in this exciting project to develop a Europe-wide network of help lines for rare diseases. Two expected outcomes of the project are a common tool and methodology for Europe-wide analysis of calls, and a Europe-wide service to put very isolated patients in contact with each other.

The fourth European Conference on Rare Diseases will take place in Lisbon on the 27th and 28th of November 2007. Portuguese rare disease patient groups are looking forward to the event, which should help them raise awareness of rare diseases in the country. Preparations are well underway, with a recent Eurordis meeting of rare disease patient organisations in Lisbon.

Dietmar Klucken suffers from a very rare disease called Neurodegeneration with brain iron accumulation (NBIA). His family is relentless in their battle to raise awareness on such a rare disease and foster research in a field where so little has been done so far. Dietmar’s mother, Angelika, tells her story.

Treatment of acute myeloid leukaemia
Antisense oligonucleotide 5'-d [P-Thio](CCCTG CTCCC CCCTG GCTCC)-3'

Treatment of mantle cell lymphoma
Temsirolimus

New designations October 06

Prevention of necrotizing enterocolitis
Heparin-binding epidermal growth factor-like growth factor (HB-EGF), amino acids 74-148

Prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection
Human cytomegalovirus immunoglobulin

Prevention of Graft-versus-Host disease
Recombinant fusion protein consisting of the extracellular portion of CD95 fused to the Fc part of a human IgG1 molecule

New Marketing Authorisations

Sprycel
[Dasatinib]
Bristol-Myers Squibb Pharma, UK
 

What is SPRYCEL?
SPRYCEL contains the active substance dasatinib. It is available as round or oval white tablets containing 20 mg, 50 mg or 70 mg of dasatinib.

What is SPRYCEL used for?
SPRYCEL is an anticancer medicine. It is used to treat adult patients who have leukaemia (cancer of the white blood cells):
-It is used in chronic myeloid leukaemia (CML), a type of leukaemia where granulocytes (a type of white blood cell) start growing out of control, in all phases of the disease (chronic, accelerated and blast). SPRYCEL is used when patients cannot tolerate, or when their disease is not responding to other treatments including imatinib (another anticancer medicine).
- It is also used in acute lymphoblastic leukaemia (ALL), a type of leukaemia where lymphocytes (another type of white blood cell) multiply too quickly, or blast phase CML, when the patients are ‘Philadelphia chromosome positive’ (Ph+). This means that some of their genes have rearranged themselves to form a special chromosome called the Philadelphia chromosome. This chromosome produces an enzyme that leads to the development of leukaemia. SPRYCEL is used when patients cannot tolerate, or when their disease is not responding to other treatments.

Because the number of patients with these two forms of leukaemia is low, the diseases are rare, and SPRYCEL was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 23 December 2005.

The medicine can only be obtained with a prescription.

All Orphan Drugs in Europe (in English) >

European Public Assessment Reports (EPARs)(multilingual) >

Editorial Team: Yann Le Cam, Jérôme Parisse-Brassens (Editor and Writer), Julia Fitzgerald, Nathacha Appanah (Writer), David Oziel (Site of the Month), Anja Helm, François Houÿez, Flaminia Macchia

Translation Team: Conchi Casas Jorde (Spanish), Ana Cláudia Jorge and Victor Ferreira (Portuguese), Roberta Ruotolo (Italian), Trado Verso (French), Ulrich Langenbeck (German)

© 2007 Eurordis