List of the latest marketing authorisations and medicinal products designations
Detailed information on European orphan medicinal products designation applications is available on theEMA website. A full list of designated and authorised orphan medicinal products in Europe available at:ec.europa.eu.
Learn more about the Orphan designation process in Europe
The Voice of Rare Disease
Patients in Europe
Patients in Europe
Recent marketing authorisations
Holoclar (ex vivo expanded autologous human corneal epithelial cells containing stem cells)
Treatment of corneal diseases
Chiesi Farmaceutici S.p.A.
Italy
17/02/2015
Chiesi Farmaceutici S.p.A.
Italy
17/02/2015
What is Holoclar and what is it used for?
Holoclar is a stem-cell treatment used in the eye to replace damaged cells on surface (epithelium) of the cornea, the transparent layer in front of the eye covering the iris (the coloured part).
It is used in adult patients with moderate to severe limbal stem-cell deficiency caused by burns, including chemical burns, to the eyes. Patients with this condition do not have enough limbal stem cells which normally act as a regeneration system, replenishing the outer corneal cells when they get damaged and when they age.
Holoclar is a type of advanced therapy product called a ‘tissue engineered product’. It consists of cells taken from the patient’s limbus (at the edge of the cornea) and then grown in a laboratory so that they can be used to repair the damaged corneal surface.
Because the number of patients with limbal stem-cell deficiency due to burns to the eyes is low, the disease is considered ‘rare’, and Holoclar was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 7 November 2008.
It is used in adult patients with moderate to severe limbal stem-cell deficiency caused by burns, including chemical burns, to the eyes. Patients with this condition do not have enough limbal stem cells which normally act as a regeneration system, replenishing the outer corneal cells when they get damaged and when they age.
Holoclar is a type of advanced therapy product called a ‘tissue engineered product’. It consists of cells taken from the patient’s limbus (at the edge of the cornea) and then grown in a laboratory so that they can be used to repair the damaged corneal surface.
Because the number of patients with limbal stem-cell deficiency due to burns to the eyes is low, the disease is considered ‘rare’, and Holoclar was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 7 November 2008.
Cerdelga (eliglustat)
Treatment of type-1 Gaucher disease
Genzyme Europe BV
Netherlands
19/01/2015
Genzyme Europe BV
Netherlands
19/01/2015
What is Cerdelga and what is it used for?
Cerdelga is a medicine used for the long-term treatment of adult patients with type-1 Gaucher disease. Gaucher disease is a rare inherited disorder, in which people do not have enough of an enzyme called glucocerebrosidase (also known as acid beta-glucosidase). This enzyme normally breaks down a fat called glucosylceramide (or glucocerebroside), and without it, the fat builds up in the body, typically in the liver, spleen and bone. This produces the symptoms of the disease: anaemia (low red blood cell counts), tiredness, easy bruising and a tendency to bleed, an enlarged spleen and liver, and bone pain and breaks.
Cerdelga is used in patients who have type-1 Gaucher disease, which is the type that usually affects the liver, spleen and bones. Cerdelga is used in patients whose body breaks down this medicine at normal speed (known as ‘intermediate’ or ‘extensive metabolisers’) or at slow speed (‘poor metabolisers’).
Because the number of patients with Gaucher disease is low, the disease is considered ‘rare’, and Cerdelga was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 4 December 2007.
Cerdelga contains the active substance eliglustat
Cerdelga is used in patients who have type-1 Gaucher disease, which is the type that usually affects the liver, spleen and bones. Cerdelga is used in patients whose body breaks down this medicine at normal speed (known as ‘intermediate’ or ‘extensive metabolisers’) or at slow speed (‘poor metabolisers’).
Because the number of patients with Gaucher disease is low, the disease is considered ‘rare’, and Cerdelga was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 4 December 2007.
Cerdelga contains the active substance eliglustat
Ofev (nintedanib)
Treatment of Idiopathic Pulmonary Fibrosis (IPF)
Boehringer Ingelheim International GmbH
Germany
15/01/2015
Boehringer Ingelheim International GmbH
Germany
15/01/2015
What is Ofev and what is it used for?
Ofev is a medicine used to treat adults with idiopathic pulmonary fibrosis (IPF). IPF is a long-term disease in which hard fibrous tissue continuously forms in the lungs, causing persistent cough and severe shortness of breath. ‘Idiopathic’ means that the cause of the disease is unknown.
Because the number of patients with IPF is low, the disease is considered ‘rare’, and Ofev was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 26 April 2013.
Ofev contains the active substance nintedanib.
Because the number of patients with IPF is low, the disease is considered ‘rare’, and Ofev was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 26 April 2013.
Ofev contains the active substance nintedanib.
Scenesse (afamelanotide)
Treatment of erythropoietic protoporphyria (EPP)
Clinuvel UK Limited
United Kingdom
22/12/2014
Clinuvel UK Limited
United Kingdom
22/12/2014
What is Scenesse and what is it used for?
Scenesse is an implant used to treat patients with erythropoietic protoporphyria (EPP), a rare disease that causes intolerance to light.
In patients with EPP, exposure to light can lead to symptoms such as pain and swelling of the skin, which prevent patients from being able to spend time outdoors or in places with bright light. Scenesse is used to help prevent or reduce these symptoms so that these patients can lead more normal lives.
Because the number of patients with EPP is low, the disease is considered ‘rare’, and Scenesse was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 May 2008.
Scenesse contains the active substance afamelanotide.
In patients with EPP, exposure to light can lead to symptoms such as pain and swelling of the skin, which prevent patients from being able to spend time outdoors or in places with bright light. Scenesse is used to help prevent or reduce these symptoms so that these patients can lead more normal lives.
Because the number of patients with EPP is low, the disease is considered ‘rare’, and Scenesse was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 May 2008.
Scenesse contains the active substance afamelanotide.
Cyramza (ramucirumab)
Treatment of advanced gastric cancer
Eli Lilly Nederland B.V.
The Netherlands
19/12/2014
Eli Lilly Nederland B.V.
The Netherlands
19/12/2014
What is Cyramza and what is it used for?
Cyramza is a cancer medicine used to treat adult patients with advanced gastric cancer (cancer of the stomach) or cancer of the area where the gullet (oesophagus) enters the stomach (known as gastro-oesophageal junction adenocarcinoma). Cyramza is used in combination with another medicine, paclitaxel, when the disease has worsened despite treatment with medicines containing platinum and fluoropyrimidines.
For patients whose disease has worsened despite treatment with a platinum or fluoropyrimidine, Cyramza can be given on its own if the combination with paclitaxel is not appropriate
Cyramza contains the active substance ramucirumab. Because the number of patients with gastric cancer is low, the disease is considered ‘rare’ in the EU, and Cyramza was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 4 July 2012.
For patients whose disease has worsened despite treatment with a platinum or fluoropyrimidine, Cyramza can be given on its own if the combination with paclitaxel is not appropriate
Cyramza contains the active substance ramucirumab. Because the number of patients with gastric cancer is low, the disease is considered ‘rare’ in the EU, and Cyramza was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 4 July 2012.
Lynparza (olaparib)
Treatment of high grade serous epithelial cancer of the ovary
AstraZeneca AB
Sweden
16/12/2014
AstraZeneca AB
Sweden
16/12/2014
What is Lynparza and what is it used for?
Lynparza is a cancer medicine used for the ‘maintenance’ treatment of adult patients with high grade serous epithelial cancer of the ovary (a type of advanced cancer of the ovary), including cancer of the fallopian tubes (part of the female reproductive system that connect the ovaries to the uterus) and cancer of the peritoneum (the membrane lining the abdomen).
Lynparza is used in patients who have mutations (defects) in one of the two genes known as BRCA1 and BRCA2 and who have recurrent disease (when the cancer has come back after previous treatment). Lynparza is given after treatment with platinum-based medicines, when the tumour is diminishing in size or has completely disappeared. It is given to those patients whose previous treatment with platinum-based medicines led to a durable response (lasting 6 months or more).
Lynparza contains the active substance olaparib.
Because the number of patients with ovarian cancer is low, the disease is considered ‘rare’ and Lynparza was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 6 December 2007.
Lynparza is used in patients who have mutations (defects) in one of the two genes known as BRCA1 and BRCA2 and who have recurrent disease (when the cancer has come back after previous treatment). Lynparza is given after treatment with platinum-based medicines, when the tumour is diminishing in size or has completely disappeared. It is given to those patients whose previous treatment with platinum-based medicines led to a durable response (lasting 6 months or more).
Lynparza contains the active substance olaparib.
Because the number of patients with ovarian cancer is low, the disease is considered ‘rare’ and Lynparza was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 6 December 2007.
Ketoconazole HRA (ketoconazole)
Treatment of Cushing’s syndrome
Laboratoire HRA Pharma
France
19/11/2014
Laboratoire HRA Pharma
France
19/11/2014
What is Ketoconazole HRA and what is it used for?
Ketoconazole HRA is a medicine used to treat adults and children above the age of 12 years with Cushing’s syndrome. Cushing’s syndrome is a disease characterised by an excess production of the hormone cortisol by the adrenal glands, two glands situated above the kidneys.
The active substance in Ketoconazole HRA is ketoconazole. Because the number of patients with Cushing’s syndrome is low, the disease is considered ‘rare’, and Ketoconazole HRA was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 23 April 2012.
The active substance in Ketoconazole HRA is ketoconazole. Because the number of patients with Cushing’s syndrome is low, the disease is considered ‘rare’, and Ketoconazole HRA was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 23 April 2012.
Imbruvica (ibrutinib)
Treatment of chronic lymphocytic leukaemia and mantle cell lymphoma
Janssen-Cilag International NV
Belgium
21 October 2014
Janssen-Cilag International NV
Belgium
21 October 2014
What is Imbruvica and what is it used for?
Imbruvica is a cancer medicine that is used to treat two types of blood cancer: chronic lymphocytic leukaemia and mantle cell lymphoma; both are cancers affecting a type of white blood cells called B lymphocytes.
In chronic lymphocytic leukaemia, Imbruvica is used in patients who have received at least one previous treatment, and in patients who have genetic mutations in their cancer cells called 17p deletion or TP53 mutation that make them unsuitable for treatment with a combination of chemotherapy medicines and immunotherapy (treatments that stimulate the immune system to kill cancer cells).
In mantle cell lymphoma, Imbruvica is used in patients whose disease does not respond to or has come back after previous treatment.
Because the number of patients with these diseases is low, they are considered ‘rare’, and Imbruvica was designated an ‘orphan medicine’ (a medicine used in rare diseases).
Imbruvica contains the active substance ibrutinib.
In chronic lymphocytic leukaemia, Imbruvica is used in patients who have received at least one previous treatment, and in patients who have genetic mutations in their cancer cells called 17p deletion or TP53 mutation that make them unsuitable for treatment with a combination of chemotherapy medicines and immunotherapy (treatments that stimulate the immune system to kill cancer cells).
In mantle cell lymphoma, Imbruvica is used in patients whose disease does not respond to or has come back after previous treatment.
Because the number of patients with these diseases is low, they are considered ‘rare’, and Imbruvica was designated an ‘orphan medicine’ (a medicine used in rare diseases).
Imbruvica contains the active substance ibrutinib.
Translarna (ataluren)
Treatment of Duchenne Muscular Dystrophy
PTC Therapeutics Limited
United Kingdom
21 July 2014
PTC Therapeutics Limited
United Kingdom
21 July 2014
What is Translarna and what is it used for?
Translarna is a medicine that contains the active substance ataluren. It is used to treat patients aged 5 years and older with Duchenne muscular dystrophy who are able to walk. Duchenne muscular dystrophy is a genetic disease that gradually causes weakness and loss of muscle function. Translarna is used in the small group of Duchenne patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene. Because the number of patients with Duchenne muscular dystrophy is low, the disease is considered ‘rare’, and Translarna was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 27 May 2005.
Arzerra (ofatumumab)
Previously untreated chronic lymphocytic leukaemia (CLL)
Extension of indication: 5 August 2014
Glaxo Group Ltd
United Kingdom
Extension of indication: 5 August 2014
Glaxo Group Ltd
United Kingdom
What is Arzerra?
Arzerra is a cancer medicine that contains the active substance ofatumumab. It is available as a concentrate that is made up into a solution for infusion (drip into a vein).
What is Arzerra used for?
Arzerra is used to treat chronic lymphocytic leukaemia (CLL), a cancer of a type of white blood cells called lymphocytes. It is used together with chlorambucil or bendamustine (other cancer medicines) in previously untreated patients who cannot be treated with therapy based on another cancer medicine, fludarabine. It can also be used in patients whose disease has not responded to previous treatment with fludarabine and a medicine called alemtuzumab.
Because the number of patients with CLL is low, the disease is considered ‘rare’, and Arzerra was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 7 November 2008.
The medicine can only be obtained with a prescription.
Arzerra is used to treat chronic lymphocytic leukaemia (CLL), a cancer of a type of white blood cells called lymphocytes. It is used together with chlorambucil or bendamustine (other cancer medicines) in previously untreated patients who cannot be treated with therapy based on another cancer medicine, fludarabine. It can also be used in patients whose disease has not responded to previous treatment with fludarabine and a medicine called alemtuzumab.
Because the number of patients with CLL is low, the disease is considered ‘rare’, and Arzerra was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 7 November 2008.
The medicine can only be obtained with a prescription.
Gazyvaro (obinutuzumab)
Previously untreated chronic lymphocytic leukaemia (CLL)
Roche Registration Ltd
United-Kingdom
23 July 2014
Roche Registration Ltd
United-Kingdom
23 July 2014
What is Gazyvaro and what is it used for?
Gazyvaro is a cancer medicine that contains the active substance obinutuzumab. It is used with chlorambucil (another cancer medicine) to treat adult patients with previously untreated chronic lymphocytic leukaemia (CLL). CLL is a cancer of B-lymphocytes, a type of white blood cells. Gazyvaro is used in patients with CLL who also have other medical conditions that make them ineligible for treatment based on a medicine called fludarabine.
Because the number of patients with CLL is low, the disease is considered ‘rare’, and Gazyvaro was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 10 October 2012.
Because the number of patients with CLL is low, the disease is considered ‘rare’, and Gazyvaro was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 10 October 2012.
Sylvant (siltuximab)
Treatment of multicentric Castleman’s disease
Janssen-Cilag International NV
Belgium
22 May 2014
Janssen-Cilag International NV
Belgium
22 May 2014
What is Sylvant and what is it used for?
Sylvant is a medicine that contains the active substance siltuximab. It is used to treat multicentric Castleman’s disease in adults who tested negative for the human immunodeficiency virus (HIV) and the human herpesvirus-8 (HHV-8).
Castleman’s disease is a disorder of the lymphatic system (a network of vessels that transport fluid from tissues through the lymph nodes and into the bloodstream) in which cells in lymph nodes start growing abnormally, causing benign tumours. Multicentric means that the disease affects several lymph nodes as well as other organs in the body. Symptoms can include tiredness, sweating at night, fever, peripheral neuropathy (pins and needles due to nerve damage) and enlargement of liver and spleen.
Because the number of patients with Castleman’s disease is low, the disease is considered ‘rare’, and Sylvant was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 30 November 2007.
Castleman’s disease is a disorder of the lymphatic system (a network of vessels that transport fluid from tissues through the lymph nodes and into the bloodstream) in which cells in lymph nodes start growing abnormally, causing benign tumours. Multicentric means that the disease affects several lymph nodes as well as other organs in the body. Symptoms can include tiredness, sweating at night, fever, peripheral neuropathy (pins and needles due to nerve damage) and enlargement of liver and spleen.
Because the number of patients with Castleman’s disease is low, the disease is considered ‘rare’, and Sylvant was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 30 November 2007.
Vimizim (elosulfase alfa)
Treatment of mucopolysaccharidosis type IVA (MPS IVA)
BioMarin Europe Ltd
United Kingdom
28 April 2014
BioMarin Europe Ltd
United Kingdom
28 April 2014
What is Vimizim and what is it used for?
Vimizim is a medicine that contains the active substance elosulfase alfa. It is used to treat patients with mucopolysaccharidosis type IVA (MPS IVA, also known as Morquio A syndrome). This disease is caused by the lack of an enzyme called N-acetylgalactosamine-6-sulfatase, which is needed to break down substances in the body called glycosaminoglycans (GAGs). If the enzyme is not present, or only present in very small quantities, GAGs cannot be broken down and they build up in bones and organs. This causes the signs of the disease, the most noticeable being short bones, difficulty moving and breathing, clouding of the eyes and hearing loss.
Because the number of patients with MPS IVA is low, the disease is considered ‘rare’, and Vimizim was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 24 July 2009.
Because the number of patients with MPS IVA is low, the disease is considered ‘rare’, and Vimizim was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 24 July 2009.
Deltyba (delamanid)
Treatment of tuberculosis
Otsuka Novel Products GmbH
Germany
28 April 2014
Otsuka Novel Products GmbH
Germany
28 April 2014
What is Deltyba and what is it used for?
Deltyba is a tuberculosis medicine that contains the active substance delamanid. Tuberculosis is an infection caused by the bacterium Mycobacterium tuberculosis (M. tuberculosis). Deltyba is used in adults with tuberculosis that is affecting the lung and that is multi-drug resistant (resistant to at least isoniazid and rifampicin, two standard anti-tuberculosis medicines). It is used together with other standard medicines and when other combinations without this medicine cannot be used either because the disease is resistant to them or because of their side effects.
Because the number of patients with tuberculosis is low in the EU, the disease is considered ‘rare’, and Deltyba was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 1 February 2008.
Para-aminosalicylic acid Lucane (para-aminosalicylic acid (PAS))
Treatment of tuberculosis
Lucane Pharma SA
France
07 April 2014
Lucane Pharma SA
France
07 April 2014
What is Para-aminosalicylic acid Lucane and what is it used for?
Para-aminosalicylic acid Lucane is a tuberculosis medicine that contains the active substance para-aminosalicylic acid (PAS). It is used in combination with other medicines to treat adults and children from 28 days of age who have multi-drug resistant tuberculosis when combinations without this medicine cannot be used, either because the disease is resistant to them or because of their side effects.
Multi-drug resistance is when the bacteria causing tuberculosis (Mycobacterium tuberculosis) are resistant to treatment with at least isoniazid and rifampicin, two standard tuberculosis medicines.
Because the number of patients with tuberculosis is low in the EU, the disease is considered ‘rare’, and Para-aminosalicylic acid Lucane was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 17 December 2010.
Cholic acid FGK (cholic acid)
Treatment of inborn errors in primary bile acid synthesis
FGK Representative Service GmbH
Germany
04 April 2014
FGK Representative Service GmbH
Germany
04 April 2014
What is Cholic acid FGK and what is it used for?
Cholic acid FGK is a medicine that contains the active substance cholic acid. This is a ‘primary bile acid’, which is a main component of the bile (a fluid produced by the liver that helps to digest fats).
Cholic acid FGK is used for the life-long treatment of adults and children from one month of age who cannot produce enough primary bile acids such as cholic acid due to genetic abnormalities that result in a lack of liver enzymes. When these primary bile acids are lacking, the body produces abnormal bile acids instead, which can damage the liver potentially leading to life-threatening liver failure. The condition is known as ‘inborn errors in primary bile acid synthesis’.
Cholic acid FGK is authorised for use in those patients who lack one of the following liver enzymes: sterol 27-hydroxylase; 2-methylacyl-CoA racemase; or cholesterol 7α-hydroxylase.
Because the number of patients with inborn errors in primary bile acid synthesis is low, the condition is considered ‘rare’, and Cholic acid FGK was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 28 October 2009.
Cometriq (cabozantinib)
Treatment of medullary thyroid carcinoma
TMC Pharma Services Ltd
United-Kingdom
21 March 2014
TMC Pharma Services Ltd
United-Kingdom
21 March 2014
What is Cometriq and what is it used for?
Cometriq is a cancer medicine that contains the active substance cabozantinib. It is used to treat adults with medullary thyroid cancer, a type of cancer originating in the cells in the thyroid gland that produce the hormone calcitonin. Cometriq is used when the cancer cannot be removed by surgery and has progressed or spread to other parts of the body.
The benefits of Cometriq may be smaller for patients whose cancer does not have a mutation in a gene called the ‘re-arranged during transfection’ (RET) gene, and this should be taken into account when deciding whether to start treatment.
Because the number of patients with medullary thyroid cancer is low, the disease is considered ‘rare’, and Cometriq was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 6 February 2009.
Sirturo (bedaquiline)
Treatment of tuberculosis
Janssen-Cilag International N.V.
Belgium
5 March 2014
Janssen-Cilag International N.V.
Belgium
5 March 2014
What is Sirturo and what is it used for?
Sirturo is a tuberculosis medicine that contains the active substance bedaquiline. Tuberculosis is an infection caused by the bacterium Mycobacterium tuberculosis. Sirturo is used in combination with other tuberculosis medicines in adults with tuberculosis that is affecting the lung and that is multi-drug resistant (resistant to at least isoniazid and rifampicin, two standard tuberculosis medicines). It is given when combinations without Sirturo cannot be used, either because the disease is resistant to them or because of their side effects.
Because the number of patients with tuberculosis is low in the EU, the disease is considered ‘rare’, and Sirturo was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 26 August 2005.
Adempas (riociguat)
Treatment of Pulmonary arterial hypertension (PAH), including treatment of chronic thromboembolic pulmonary hypertension (CTEPH)
Bayer Pharma AG
Germany
27 March 2014
Bayer Pharma AG
Germany
27 March 2014
What is Adempas and what is it used for?
Adempas is a medicine that contains the active substance riociguat. It is used to increase the ability to carry out physical activity in adults with the following forms of pulmonary hypertension (high blood pressure in the blood vessels of the lungs):
- Chronic thromboembolic pulmonary hypertension (CTEPH, where the blood vessels of the lungs are blocked or narrowed with blood clots). Adempas is used to treat patients with CTEPH who cannot be operated on, or in whom CTEPH remains or returns after surgery.
- Pulmonary arterial hypertension (PAH, where the walls of the blood vessels of the lungs are thickened and the vessels become narrowed). Adempas can be used on its own or in combination with other medicines for PAH called ‘endothelin receptor antagonists’.
Adempas is used in patients with functional class II to III CTEPH or PAH. The ‘class’ reflects the seriousness of the disease: ‘class II’ involves slight limitation of physical activity while ‘class III’ involves marked limitation of physical activity.
Because the number of patients with CTEPH or with PAH is low, the diseases are considered ‘rare’, and Adempas was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 20 December 2007.
Opsumit (macitentan)
Treatment of pulmonary arterial hypertension (PAH)
Actelion Registration Ltd
United-Kingdom
20 December 2013
Actelion Registration Ltd
United-Kingdom
20 December 2013
What is Opsumit and what is it used for?
Opsumit is a medicine that contains the active substance macitentan. It is used for the long-term treatment of pulmonary arterial hypertension (PAH), a condition in which there is abnormally high blood pressure in the arteries of the lungs, causing symptoms such as breathlessness and fatigue.
Opsumit is used for adults whose PAH is classified as ‘WHO functional class II to class III’. The ‘class’ reflects the seriousness of the disease: patients with class II PAH have slight limitation of physical activity and those with class III disease have marked limitation of physical activity. Opsumit can be used alone or in combination with other PAH medicines; for further information, see the package leaflet.
Because the number of patients with PAH is low, the disease is considered ‘rare’, and Opsumit was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 27 September 2011.
Orphacol (cholic acid)
Treatment of inborn errors in primary bile acid synthesis
Laboratoire CTRS
France
12 September 2013
Laboratoire CTRS
France
12 September 2013
What is Orphacol and what is it used for?
Orphacol is a medicine containing cholic acid, a substance found in the bile which is used to digest fats.
It is used to treat adults and children from one month of age who have a genetic abnormality that makes them unable to produce bile. Orphacol is used in patients who do not have enough of two specific liver enzymes (3β-Hydroxy-Δ5-C27-steroid oxidoreductase or Δ4-3-Oxosteroid-5β-reductase). This makes their liver unable to produce enough of the main components of bile, called primary bile acids, such as cholic acid. When these primary bile acids are lacking, the body produces abnormal bile acids instead which can damage the liver, potentially leading to life-threatening liver failure.
Because the number of patients with inborn errors in primary bile acid synthesis is low, the condition is considered ‘rare’, and Orphacol was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 18 December 2002.
Defitelio (defibrotide)
Treatment of severe hepatic veno-occlusive disease (VOD)
Gentium S.p.A
Italy
18 October 2013
Gentium S.p.A
Italy
18 October 2013
What is Defitelio and what is it used for?
Defitelio is a medicine containing the active substance defibrotide. It is used to treat severe veno-occlusive disease (VOD) in patients undergoing haematopoietic (blood) stem-cell transplantation. VOD is a condition in which the veins in the liver become blocked, leading to liver dysfunction. Defitelio is used in adults and in children from one month of age.
Because the number of patients with VOD is low, the disease is considered ‘rare’, and Defitelio was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 29 July 2004.
Imnovid (pomalidomide)
Treatment of multiple myeloma
Celgene Europe Ltd
United Kingdom
5 August 2013
Celgene Europe Ltd
United Kingdom
5 August 2013
What is Imnovid and what is it used for?
Imnovid is an anticancer medicine that contains the active substance pomalidomide. It is used in combination with dexamethasone (an anti-inflammatory medicine) to treat multiple myeloma (a cancer of the bone marrow). It is used in adults who have received at least two prior therapies, including both lenalidomide and bortezomib, and whose disease progressed after the last treatment.
Because the number of patients with multiple myeloma is low, the disease is considered ‘rare’, and Imnovid was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 October 2009.
Procysbi (cysteamine (as mercaptamine bitartrate))
Treatment of nephropathic (kidney)cystinosis
Raptor Pharmaceuticals Europe
The Netherlands
06/09/2013
Raptor Pharmaceuticals Europe
The Netherlands
06/09/2013
What is Procysbi and what is it used for?
Procysbi is a medicine that contains the active substance mercaptamine (also known as cysteamine). It is used in patients with nephropathic (kidney) cystinosis. Cystinosis is an inherited disease in which excess amounts of cystine, an amino acid naturally found in the body, build up within cells, especially in the kidneys and the eyes, damaging them.
Because the number of patients with cystinosis is low, the disease is considered ‘rare’, and Procysbi was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 20 September 2010.
Procysbi is a ‘hybrid medicine’. This means that it is similar to a ‘reference medicine’ containing the same active substance, but Procysbi is available in a formulation that allows for a delayed release of the active substance in the body. The reference medicine for Procysbi is Cystagon.
Iclusig (ponatinib)
Treatment of chronic myeloid leukaemia (CML) ; acute lymphoblastic leukaemia (ALL)
Ariad Pharma Ltd
United-Kingdom
01/07/2013
Ariad Pharma Ltd
United-Kingdom
01/07/2013
What is Iclusig and what is it used for?
Iclusig is an anticancer medicine that contains the active substance ponatinib. It is used to treat adults with the following types of leukaemia (cancer of the white blood cells):
- chronic myeloid leukaemia (CML) in its different stages known as chronic, accelerated and blast phases;
- acute lymphoblastic leukaemia (ALL) in patients who are ‘Philadelphia-chromosome-positive’ (Ph+). Ph+ means that some of the patient’s genes have rearranged themselves to form a special chromosome called the Philadelphia chromosome that leads to the development of leukaemia. The Philadelphia chromosome is found in some ALL patients but is present in most patients with CML.
Iclusig is used in patients who cannot tolerate or do not respond to dasatinib or nilotinib (other anticancer medicines) and for whom subsequent treatment with imatinib is not considered appropriate. It is also used in patients who have a genetic mutation called ‘T315I mutation’ which makes them resistant to treatment with imatinib, dasatinib or nilotinib.
Because the numbers of patients with CML and ALL are low, the diseases are considered ‘rare’, and Iclusig was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 2 February 2010.
Revlimid (lenalidomide)
Extension of indication:
Treatment of myelodysplastic syndromes
Celgene Europe Ltd.
United Kingdom
25/04/2013
Treatment of myelodysplastic syndromes
Celgene Europe Ltd.
United Kingdom
25/04/2013
What is Revlimid?
Revlimid is a medicine that contains the active substance lenalidomide. It is available as capsules (2.5 mg, 5 mg, 7.5 mg, 10 mg, 15 mg and 25 mg).
What is Revlimid used for?
Revlimid is an anticancer medicine originally indicated to treat adults with multiple myeloma.
On 25 April 2013, the CHMP adopted the following new therapeutic indication for Revlimid: myelodysplastic syndromes.
On 25 April 2013, the CHMP adopted the following new therapeutic indication for Revlimid: myelodysplastic syndromes.
Revlimid is indicated for the treatment of patients with transfusion-dependent anaemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.
Bosulif (bosutinib)
Treatment of adults with chronic myeloid leukaemia (CML)
Pfizer Limited
United-Kingdom
27/03/2013
Pfizer Limited
United-Kingdom
27/03/2013
Bosulif is an anticancer medicine that contains the active substance bosutinib. It is used to treat adults with chronic myeloid leukaemia (CML), a cancer of the white blood cells in which granulocytes (a type of white blood cell) start growing out of control.
Bosulif is used in patients who are ‘Philadelphia-chromosome-positive’ (Ph+). This means that some of the patient’s genes have re-arranged themselves to form a special chromosome called the Philadelphia chromosome. Bosulif is used to treat three stages of CML called ‘chronic phase’, ‘accelerated phase’ and ‘blast phase’. It is only used when the CML has already been treated with one or more tyrosine kinase inhibitors (medicines for CML which work in a similar way to Bosulif) and when the tyrosine kinase inhibitors called imatinib, nilotinib and dasatinib are not considered appropriate treatment options.
Because the number of patients with CML is low, the disease is considered ‘rare’, and Bosulif was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 4 August 2010.
NexoBrid (concentrate of proteolytic enzymes enriched in bromelain)
Treatment of partial deep dermal and full thickness burns
Teva Pharma GmbH
Germany
27/03/2013
Teva Pharma GmbH
Germany
27/03/2013
What is NexoBrid?
NexoBrid is a medicine that contains the active substance ‘concentrate of proteolytic enzymes enriched in bromelain’. It is available as a powder and gel, which are mixed together to make a gel (2 g/22 g or 5 g/55 g).
NexoBrid is a medicine that contains the active substance ‘concentrate of proteolytic enzymes enriched in bromelain’. It is available as a powder and gel, which are mixed together to make a gel (2 g/22 g or 5 g/55 g).
What is NexoBrid used for?
NexoBrid is used in adults to remove eschar (dead tissue which is dried-out, thick, leathery and black) from deep partial thickness and full thickness burns of the skin caused by heat or fire. Deep partial thickness burns (sometimes called ‘second degree’ burns) extend into a deep region of an inner layer of the skin called the dermis, while full thickness burns (sometimes called ‘third degree’ burns) extend even deeper, through the whole dermis.
Because the number of patients with deep partial thickness and full thickness thermal burn wounds is low, the disease is considered ‘rare’, and NexoBrid was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 30 July 2002.
The medicine can only be obtained with a prescription.
NexoBrid is used in adults to remove eschar (dead tissue which is dried-out, thick, leathery and black) from deep partial thickness and full thickness burns of the skin caused by heat or fire. Deep partial thickness burns (sometimes called ‘second degree’ burns) extend into a deep region of an inner layer of the skin called the dermis, while full thickness burns (sometimes called ‘third degree’ burns) extend even deeper, through the whole dermis.
Because the number of patients with deep partial thickness and full thickness thermal burn wounds is low, the disease is considered ‘rare’, and NexoBrid was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 30 July 2002.
The medicine can only be obtained with a prescription.
Glybera (alipogene tiparvovec)
uniQure biopharma B.V.
The Netherlands
25/10/2012
What is Glybera?
Glybera is a medicine that contains the active substance alipogene tiparvovec. It is available as a solution for injection.
Glybera is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.
Glybera is a medicine that contains the active substance alipogene tiparvovec. It is available as a solution for injection.
Glybera is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.
What is Glybera used for?
Glybera is used to treat adults with lipoprotein lipase deficiency who have severe or multiple attacks of pancreatitis (inflammation of the pancreas) despite maintaining a low-fat diet.
Lipoprotein lipase deficiency is a rare disease in which patients have a defect in the gene for lipoprotein lipase, an enzyme responsible for breaking down fats. Patients with this disease need to be on a strict low-fat diet and are prone to recurring attacks of pancreatitis, which is a severe and life-threatening complication.
Glybera is only for patients whose disease has been confirmed by appropriate genetic testing and who have detectable levels of the lipoprotein lipase enzyme in their blood.
Because the number of patients with lipoprotein lipase deficiency is low, the disease is considered ‘rare’, and Glybera was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 March 2004.
The medicine can only be obtained with a prescription.
Glybera is used to treat adults with lipoprotein lipase deficiency who have severe or multiple attacks of pancreatitis (inflammation of the pancreas) despite maintaining a low-fat diet.
Lipoprotein lipase deficiency is a rare disease in which patients have a defect in the gene for lipoprotein lipase, an enzyme responsible for breaking down fats. Patients with this disease need to be on a strict low-fat diet and are prone to recurring attacks of pancreatitis, which is a severe and life-threatening complication.
Glybera is only for patients whose disease has been confirmed by appropriate genetic testing and who have detectable levels of the lipoprotein lipase enzyme in their blood.
Because the number of patients with lipoprotein lipase deficiency is low, the disease is considered ‘rare’, and Glybera was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 March 2004.
The medicine can only be obtained with a prescription.
Adcetris ( brentuximab vedotin)
Treatment of Hodgkin lymphoma
Takeda Global Research and Development Centre (Europe) Ltd
United Kingdom
25/10/2012
Takeda Global Research and Development Centre (Europe) Ltd
United Kingdom
25/10/2012
What is Adcetris?
Adcetris is a medicine that contains the active substance brentuximab vedotin. It is available as a powder that is made up into a solution for infusion (drip into a vein).
Adcetris is a medicine that contains the active substance brentuximab vedotin. It is available as a powder that is made up into a solution for infusion (drip into a vein).
What is Adcetris used for?
Adcetris is used to treat adults with Hodgkin lymphoma (HL, a type of cancer that originates from blood cells in the lymphatic system, a part of the immune system) when the tumour cells are CD30-positive (when they have a protein called CD30 on their surface). It is used:
when the cancer has come back or has not responded to an autologous stem cell transplant (a transplant of the patient's own blood-producing cells);
Adcetris is used to treat adults with Hodgkin lymphoma (HL, a type of cancer that originates from blood cells in the lymphatic system, a part of the immune system) when the tumour cells are CD30-positive (when they have a protein called CD30 on their surface). It is used:
when the cancer has come back or has not responded to an autologous stem cell transplant (a transplant of the patient's own blood-producing cells);
when the cancer has come back or has not responded to at least two previous therapies and when autologous stem cell transplant or multi-agent chemotherapy (a combination of anticancer medicines) are not treatment options.
Adcetris is also used to treat systemic anaplastic large cell lymphoma (sALCL, a CD30-positive cancer of white blood cells called T lymphocytes), when the cancer has come back or has not responded to other treatments.
Because the number of patients with HL and sALCL is low, the diseases are considered ‘rare’, and Adcetris was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 15 January 2009.
The medicine can only be obtained with a prescription.
Because the number of patients with HL and sALCL is low, the diseases are considered ‘rare’, and Adcetris was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 15 January 2009.
The medicine can only be obtained with a prescription.
Signifor (pasireotide)
Treatment of Cushing’s disease
Novartis Europharm Limited
United-Kingdom
24/04/2012
Novartis Europharm Limited
United-Kingdom
24/04/2012
What is Signifor?
Signifor is a medicine that contains the active substance pasireotide. It is available as a solution for injection.
Signifor is a medicine that contains the active substance pasireotide. It is available as a solution for injection.
What is Signifor used for?
Signifor is used to treat adults with Cushing’s disease when surgery has failed or is not an option.
Cushing’s disease is caused by a tumour of the pituitary gland (a gland located at the base of the brain) releasing too much of a hormone called ACTH that stimulates the production of too much cortisol (a hormone also known as the ‘stress hormone’ because it is released in response to stress).
Because the number of patients with Cushing’s disease is low, the disease is considered ‘rare’, and Signifor was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 October 2009.
Signifor is used to treat adults with Cushing’s disease when surgery has failed or is not an option.
Cushing’s disease is caused by a tumour of the pituitary gland (a gland located at the base of the brain) releasing too much of a hormone called ACTH that stimulates the production of too much cortisol (a hormone also known as the ‘stress hormone’ because it is released in response to stress).
Because the number of patients with Cushing’s disease is low, the disease is considered ‘rare’, and Signifor was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 October 2009.
Dacogen (decitabine)
acute myeloid leukaemia (AML)
Janssen-Cilag International NV, Belgium
20/09/2012
Janssen-Cilag International NV, Belgium
20/09/2012
What is Dacogen?
Dacogen is a powder that is made up into a solution for infusion (drip into a vein). It contains the active substance decitabine.
Dacogen is a powder that is made up into a solution for infusion (drip into a vein). It contains the active substance decitabine.
What is Dacogen used for?
Dacogen is used to treat adults aged 65 or older with acute myeloid leukaemia (AML), a type of cancer affecting the white blood cells. It is used in patients with newly diagnosed AML who are not eligible for initial treatment with standard chemotherapy (anticancer medicines).
Because the number of patients with AML is low, the disease is considered ‘rare’, and Dacogen was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 June 2006.
The medicine can only be obtained with a prescription.
Dacogen is used to treat adults aged 65 or older with acute myeloid leukaemia (AML), a type of cancer affecting the white blood cells. It is used in patients with newly diagnosed AML who are not eligible for initial treatment with standard chemotherapy (anticancer medicines).
Because the number of patients with AML is low, the disease is considered ‘rare’, and Dacogen was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 June 2006.
The medicine can only be obtained with a prescription.
Revestive (teduglutide)
Short bowel syndrome
Nycomed Danmark APS
Denmark
30/08/2012
What is Revestive?
Revestive is a medicine that contains the active substance teduglutide. It is available as a powder and a solvent to be made up into a solution for injection.
What is Revestive used for?
Revestive is used to treat adults with short bowel syndrome. Short bowel syndrome is a condition in which nutrients and fluids are not properly absorbed by the gut, usually following the surgical removal of a large portion of the small intestine. Revestive is used after ‘intestinal adaptation’ has occurred (changes in the function of the bowel to compensate for its reduced size following surgery).
Because the number of patients with short bowel disease is low, the disease is considered ‘rare’, and Revestive was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 11 December 2001.
The medicine can only be obtained with a prescription.
Nycomed Danmark APS
Denmark
30/08/2012
What is Revestive?
Revestive is a medicine that contains the active substance teduglutide. It is available as a powder and a solvent to be made up into a solution for injection.
What is Revestive used for?
Revestive is used to treat adults with short bowel syndrome. Short bowel syndrome is a condition in which nutrients and fluids are not properly absorbed by the gut, usually following the surgical removal of a large portion of the small intestine. Revestive is used after ‘intestinal adaptation’ has occurred (changes in the function of the bowel to compensate for its reduced size following surgery).
Because the number of patients with short bowel disease is low, the disease is considered ‘rare’, and Revestive was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 11 December 2001.
The medicine can only be obtained with a prescription.
Jakavi (ruxolitinib)
myelofibrosis
Novartis Europharm Limited, UK
United-Kingdom
23/08/2012
What is Jakavi?
Jakavi is a medicine that contains the active substance ruxolitinib. It is available as tablets (5, 15 and 20 mg).
Novartis Europharm Limited, UK
United-Kingdom
23/08/2012
What is Jakavi?
Jakavi is a medicine that contains the active substance ruxolitinib. It is available as tablets (5, 15 and 20 mg).
What is Jakavi used for?
Jakavi is used to treat adults with myelofibrosis who have splenomegaly (enlarged spleen) or symptoms related to the disease such as fever, night sweats, bone pain and weight loss.
Myelofibrosis is a disease in which the bone marrow becomes very dense and rigid and produces abnormal, immature blood cells. Jakavi can be used in three types of the disease: primary myelofibrosis (also known as chronic idiopathic myelofibrosis, where the cause is unknown), post polycythaemia vera myelofibrosis (where the disease is linked to an overproduction of red blood cells) and post essential thrombocythaemia myelofibrosis (where the disease is linked to an overproduction of platelets, components that help the blood to clot).
Because the number of patients with these diseases is low, they are considered ‘rare’, and Jakavi was designated an ‘orphan medicine’ (a medicine used in rare diseases) for chronic idiopathic myelofibrosis on 7 November 2008 and for myelofibrosis secondary to polycythaemia vera or essential thrombocythaemia on 3 April 2009.
The medicine can only be obtained with a prescription.
Jakavi is used to treat adults with myelofibrosis who have splenomegaly (enlarged spleen) or symptoms related to the disease such as fever, night sweats, bone pain and weight loss.
Myelofibrosis is a disease in which the bone marrow becomes very dense and rigid and produces abnormal, immature blood cells. Jakavi can be used in three types of the disease: primary myelofibrosis (also known as chronic idiopathic myelofibrosis, where the cause is unknown), post polycythaemia vera myelofibrosis (where the disease is linked to an overproduction of red blood cells) and post essential thrombocythaemia myelofibrosis (where the disease is linked to an overproduction of platelets, components that help the blood to clot).
Because the number of patients with these diseases is low, they are considered ‘rare’, and Jakavi was designated an ‘orphan medicine’ (a medicine used in rare diseases) for chronic idiopathic myelofibrosis on 7 November 2008 and for myelofibrosis secondary to polycythaemia vera or essential thrombocythaemia on 3 April 2009.
The medicine can only be obtained with a prescription.
Kalydeco (ivacaftor)
cystic fibrosis
Vertex Pharmaceuticals (U.K.) Ltd.
United-Kingdom
23/07/2012
What is Kalydeco?
Kalydeco is a medicine that contains the active substance ivacaftor. It is available as tablets (150 mg).
What is Kalydeco used for?
Kalydeco is used to treat cystic fibrosis in patients aged six years and above who have the G551D mutation in their gene for the protein called cystic fibrosis transmembrane conductance regulator (CFTR). Cystic fibrosis is an inherited disease that affects the cells that secrete mucus in the lungs, and the cells that secrete digestive juices from the glands in the gut and pancreas. In cystic fibrosis these secretions become thick, blocking the airways and the flow of digestive juices. This leads to problems with the digestion and absorption of food, resulting in poor growth, and long-term infection and inflammation of the lungs because of excess mucus not being cleared away. Because the number of patients with cystic fibrosis is low, the disease is considered ‘rare’, and Kalydeco was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 July 2008.
The medicine can only be obtained with a prescription.
Vertex Pharmaceuticals (U.K.) Ltd.
United-Kingdom
23/07/2012
What is Kalydeco?
Kalydeco is a medicine that contains the active substance ivacaftor. It is available as tablets (150 mg).
What is Kalydeco used for?
Kalydeco is used to treat cystic fibrosis in patients aged six years and above who have the G551D mutation in their gene for the protein called cystic fibrosis transmembrane conductance regulator (CFTR). Cystic fibrosis is an inherited disease that affects the cells that secrete mucus in the lungs, and the cells that secrete digestive juices from the glands in the gut and pancreas. In cystic fibrosis these secretions become thick, blocking the airways and the flow of digestive juices. This leads to problems with the digestion and absorption of food, resulting in poor growth, and long-term infection and inflammation of the lungs because of excess mucus not being cleared away. Because the number of patients with cystic fibrosis is low, the disease is considered ‘rare’, and Kalydeco was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 July 2008.
The medicine can only be obtained with a prescription.
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