EU/3/13/1184 - orphan designation for treatment of sickle cell disease
OrphanHuman
Overview
This medicine is now known as rivipansel.
On 5 August 2013, orphan designation (EU/3/13/1184) was granted by the European Commission to Pfizer Limited, United Kingdom, for (1R,3R,4R,5S)-3-O-[2-O-benzoyl-3-O-(sodium(2S)-3-cyclohexyl-propanoate-2-yl)-β-D-galactopyranosyl]-4-O-(α-L-fucopyranosyl)-5-orothylamido-cyclohexane-1-carboxylic acid ethyl-2-amidyl-ethyloxy-2-acetyl-(8-amino-1,3,6-naphthalene-tris sodium sulfonate) amide for the treatment of sickle-cell disease.
The sponsorship was transferred to Pfizer Europe MA EEIG, Belgium, in November 2018.
The sponsorship was transferred to FGK Representative Service GmbH, Germany in April 2020.
Sickle-cell disease is a genetic disease in which the red blood cells become rigid and sticky, and change from being disc-shaped to being crescent-shaped (like a sickle). The change in shape is caused by the presence of an abnormal form of haemoglobin, the protein in red blood cells that carries oxygen around the body. In patients with sickle-cell disease, the abnormal red blood cells attach to the walls of blood vessels and block them, restricting the flow of oxygen-rich blood to the internal organs such as the heart, lungs and spleen. Because the abnormal red blood cells have a shorter life span, they release haemoglobin into the blood circulation rather than carrying it to the internal organs where it is needed. As a result, the disease causes severe pain and damage to these organs as well as repeated infections and anaemia (low red-blood-cell counts).
Sickle-cell disease is a severe disease that is long-lasting and may be life-threatening because of damage to the heart and the lungs, anaemia and infections.
At the time of designation, sickle-cell disease affected approximately 1.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 76,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).
*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 509,000,000 (Eurostat 2013).
At the time of designation, the only medicine authorised in the EU to treat sickle-cell disease was hydroxycarbamide. The main treatment for sickle-cell disease was blood transfusion. This was usually combined with 'iron chelators' (medicines used to reduce the high iron levels in the body caused by repeated blood transfusions), which are necessary in patients with long-term anaemias such as sickle-cell disease. In some cases, haematopoietic (blood) stem-cell transplantation was used (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow) to allow the patient to produce red blood cells containing normal haemoglobin.
The sponsor has provided sufficient information to show that medicine might be of significant benefit for patients with sickle-cell disease because early studies show that it might reduce the duration of 'vaso-occlusive crises' (painful episodes caused by sickle-shaped red blood cells obstructing blood vessels and restricting blood flow to an organ) in patients already treated with currently authorised medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is expected to work by blocking proteins called selectins that are present on the surface of the cells lining the blood vessels. Selectins are thought to be involved in the attachment of sickle cells and inflammatory cells to the blood vessels, leading to the blockage of the blood flow and pain in patients with sickle-cell disease. By blocking selectins, the medicine is expected to prevent the deposit of sickle and inflammatory cells in blood vessels, thereby allowing better blood flow and improving the symptoms of the disease.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with sickle-cell disease were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for sickle cell disease. Orphan designation of the medicine had been granted in the United States for the treatment of vaso-occlusive crisis in patients with sickle-cell disease.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 July 2013 recommending the granting of this designation.
- the seriousness of the condition;
- the existence of alternative methods of diagnosis, prevention or treatment;
- either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.
Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.
Key facts
- Intended use
- Treatment of sickle cell disease
- Orphan designation status
- Positive
- EU designation number
- EU/3/13/1184
- Date of designation
- Sponsor
FGK Representative Service GmbH
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: