In our fight for rare disease patients I have sometimes quoted Antoine de Saint Exupéry, 'There are no solutions, only forces to be set in motion. Once these forces are created, solutions will follow.' Now is the time: the time to create the conditions for the implementation of solutions for all rare diseases in all Member States. Are we going to see real political commitment from the European Union, the Commission, the Parliament, and the Council, to support people living with rare diseases? Are we going to see commitment in the areas of European public health, research and drug development? At European and national levels? A commitment that can be sustained in the long term, based on proper governance and adequate financial resources?

To achieve this goal of widespread and sustainable political committment, energy and effort are also needed from each one of us. Let Michelangelo be our inspiration: "I hope that I may always desire more than I can accomplish." We don't want merely "reasonable" weak laws. We want texts that are as great as the suffering of patients and of the challenges ahead. Stefan Zweig's perceptive vision of the world guides us, 'Reason is a regulatory force; it is never in itself a creative one'. In the field of rare diseases, difficulties aren't what is stopping us from acting; it is our lack of ambition that makes things difficult. Let's aim for a strong, shared vision for rare disease policy. Let's aim to be the builders and the witnesses of a new decade of progress for patients.

This is why the future Commission's Communication on Rare Diseases and Recommendations to Member States are crucial. The legislative process has already started. The Commission is writing a draft text, to which we are contributing. As we have done in the past, we have put together a drafting group, with some of the key stakeholders in Europe, and this group has worked throughout the summer. Our internal consultation process has once more proved useful. The draft text will be extensively debated at the European Conference on Rare Diseases 2007 Lisbon on 27-28 November 2007 - a great opportunity to launch a public consultation process. Be part of the debate: register now for the Lisbon conference. Your opinion is important. If you belong to a patient group that is not member of Eurordis yet, why not become part of our network? Now is the right time to do so. Your involvement is essential to win this political battle and - more importantly - to contribute to the implementation of its outcomes during the next few years. We must show the strength, diversity and energy of our movement.

Yann Le Cam
Chief Executive Officer

Treatment of idiopathic thrombocytopenic purpura
Recombinant human soluble Fc-gamma receptor Iib

Treatment of cardiogenic shock
Pyridoxalated haemoglobin polyoxyethylene

Treatment of cutaneous T-cell lymphoma
Panobinostat lactate

Treatment of orthostatic hypotension in patients with pure autonomic failure
L-threo-3,4-dihydroxyphenylserine

Treatment of orthostatic hypotension in patients with multiple system atrophy
L-threo-3,4-dihydroxyphenylserine

Treatment of idiopathic thrombocytopenic purpura
Eltrombopag olamine

Treatment of malaria
Dihydroartemisinin, piperaquine

Treatment of cystic fibrosis
Ciprofloxacin (inhalation use)

New designations July 07

Treatment of glycogen storage disease type II (Pompe's disease)
Recombinant adeno-associated viral vector containing human acid alpha-glucosidase-gene

Treatment of cryopirin-associated periodic syndromes (Familial Cold Urticaria Syndrome (FCUS), Muckle-Wells Syndrome (MWS), and Neonatal Onset Multisystem Inflammatory Disease (NOMID), also known as Chronic Infantile Neurological Cutaneous Articular Syndrome (CINCA))
Rilonacept

Treatment of aspiration pneumonitis requiring intubation and mechanical ventilation
Lusupultide

Treatment of Guillain-Barré syndrome
Fampridine

Treatment of narcolepsy
1-{3-[3-(4-chlorophenyl)propoxy]propyl}piperidine, hydrochloride

New designations June 07

Treatment of renal cell carcinoma
Talactoferrinum alfa

Treatment of renal cell carcinoma
Everolimus

Treatment of osteosarcoma
5(S)-(2´-hydroxy ethoxy)-20(S)- camptothecin

Treatment of pancreatic cancer
Cisplatin (liposomal)

Treatment of gastric carcinoid
R-1-[2,3-dihydro-2-oxo-1-pivaloylmethyl-5-(2-pyridyl)-1 H-1,4-benzodiazepin-3-yl]-3-(3-methylaminophenyl) urea

Treatment of haemophilia B (congenital factor IX deficiency)
Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1

New Marketing Authorisations

Siklos
[hydroxycarbamide]
Addmedica SAS, France

Siklos is used in adults and in children who have sickle cell syndrome (a genetic disease where the red blood cells change shape and become inflexible when they are not carrying oxygen). It is used to prevent painful vaso-occlusive crises that are recurrent (keep coming back). Vaso-occlusive crises happen when blood vessels become blocked by the abnormal red blood cells, restricting the flow of blood to an organ. They can include acute chest syndrome, a life-threatening condition when the patient has sudden chest pain, fever, hard breathing or signs of fluid in the lungs on an X-ray. Because the number of patients with sickle cell syndrome is low, the disease is considered 'rare', and Siklos was designated an 'orphan medicine' (a medicine used in rare diseases) on 9 July 2003.

Tracleer
[Bosentan monohydrate]
Actelion Registration Ltd., UK

Tracleer received a new authorisation for the treatment of patients suffering from systemic sclerosis in whom poor circulation caused by the disease has led to the development of 'digital ulcers' (sores on the fingers and toes). Tracleer is intended to reduce the number of new digital ulcers that are formed. Tracleer was designated an 'orphan medicine' (a medicine used in rare diseases) on 17 March 2003.

Soliris
[eculizumab]
Alexion Europe SAS, France

Soliris is used to treat patients with paroxysmal nocturnal haemoglobinuria (PNH), a rare, lifethreatening genetic disease that causes the red blood cells to be broken down too quickly. This results in anaemia (low red blood cell counts), thrombosis (formation of blood clots in the blood vessels) and dark urine. Because the number of patients with PNH is low, the disease is considered 'rare', and Soliris was designated an 'orphan medicine' (a medicine used in rare diseases) on 17 October 2003.

Diacomit
[stiripentol]
Biocodex, France

Diacomit is an anti-epileptic medicine. It is used in children with a very rare type of epilepsy called 'severe myoclonic epilepsy in infancy' (SMEI), which is also known as Dravet's syndrome. This type of epilepsy affects young children. Diacomit is used as an add-on to clobazam and valproate (other anti-epileptic medicines) to treat generalised tonic- clonic seizures (major fits, including loss of consciousness) when these are not adequately controlled with clobazam and valproate. Because the number of patients with SMEI is low, the disease is rare, and Diacomit was designated as an 'orphan medicine' (a medicine used in rare diseases) on 5 December 2001.

Glivec
[Imatinib mesilate]
Novartis Europharm Ltd, UK

Glivec is an anticancer medicine. It received two additional authorisations for two new indications:
-Myelodysplastic or myeloproliferative diseases (MD/MPD), a group of diseases in which the body produces large numbers of one or more types of abnormal blood cells. Glivec is used to treat adults with MD/MPD who have re-arrangements of the gene for platelet-derived growth factor receptor (PDGFR). Glivec was designated an 'orphan medicine' (a medicine used in rare diseases) for this indication on 23 December 2005.
-Advanced hypereosinophilic syndrome (HES) or chronic eosinophilic leukaemia (CEL), diseases in which eosinophils (another type of white blood cell) start growing out of control. Glivec is used to treat adults with HES or CEL who have a specific re- arrangement of 2 genes called FIP1L1 and PDGFRa. Glivec was designated an 'orphan medicine' (a medicine used in rare diseases) for this indication on 28 October 2005.

All Orphan Drugs in Europe (in English) >

European Public Assessment Reports (EPARs)(multilingual) >

Editorial Team: Yann Le Cam, Jérôme Parisse-Brassens, Julia Fitzgerald, Nathacha Appanah (Writer), Anja Helm, François Houÿez

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

© 2007 Eurordis