2013-04-30

Orphan drug lastest designations and marketing authorisation





About EURORDIS

About Rare Diseases

Rare Disease Policy

Orphan Drugs & Treatments

Living with a Rare Disease

Services to Patients

Training Resources

News & Events

Home › Orphan Drugs & Treatments

Languages

EN

FR

DE

ES

IT

PT

RU

Latest orphan designations and/or marketing authorisations

ShareThis

ORPHAN DRUG DESIGNATIONS
March 2013
Treatment of neuronal ceroid lipofuscinosis type 2

Recombinant human tripeptidyl-peptidase 1
Treatment of Stargardt’s disease

Ramiprilat
Treatment of Churg-Strauss Syndrome

Mepolizumab
Treatment of mantle cell lymphoma

1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H- pyrazolo [3,4-d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one
Treatment of congenital alpha-1 antitrypsin deficiency

Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-Lglutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl] acetate salt
Prevention of graft rejection following solid organ transplantation

Murine IgM monoclonal antibody binding to alpha beta T-cell receptor
Treatment of sickle cell disease

Poloxamer 188
Treatment of chronic non-infectious uveitis

Gevokizumab
Treatment of Niemann-Pick disease, type C

Recombinant human heat shock protein 70
Treatment of hepatocellular carcinoma

4-[2-(6-methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate
Treatment of systemic sclerosis

2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid

February 2013
Treatment of achromatopsia caused by mutations in the CNGB3 gene

Recombinant adeno-associated viral vector containing the human CNGB3 gene
Treatment of amyloid light-chain amyloidosis

Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid
Treatment of moderate and severe traumatic brain injury

Progesterone
Treatment of high altitude pulmonary oedema

Cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys
Treatment of chronic thromboembolic pulmonary hypertension

Treprostinil sodium
Treatment of systemic sclerosis

Terguride
Treatment of Duchenne muscular dystrophy

Humanised monoclonal antibody against myostatin
Treatment of acute myeloid leukaemia

L-asparaginase encapsulated in erythrocytes
January 2013
Treatment of follicular lymphoma

Lenalidomide
Treatment of familial adenomatous polyposis

Eflornithine in combination with sulindac
Treatment of growth hormone deficiency

Recombinant modified human growth hormone
Treatment of 5q spinal muscular atrophy

Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
Treatment of Wilson’s disease

Choline tetrathiomolybdate
Treatment of pancreatic cancer

Recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem cell antigen
Treatment of beta-thalassaemia intermedia and major

Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
Treatment of ovarian cancer

Chimeric monoclonal antibody against claudin 6
Treatment of glioma

1,2:5,6-Dianhydrogalactitol
Treatment of achondroplasia

Modified recombinant human C-type natriuretic peptide
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)

Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene
Treatment of systemic sclerosis

Terguride
Treatment of retinitis pigmentosa

Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor
December 2012
Treatment of lead toxicity

Erdosteine
Treatment of malignant mesothelioma

Maytansinoid-conjugated human monoclonal antibody against mesothelin
Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)

Alisertib
Treatment of acromegaly

Cyclo(-gamma-aminobutyryl-L-phenylalanyl-L-tryptophanyl-Dtryptophanyl-

L-lysyl-L-threonyl-L phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt
Treatment of perinatal asphyxia

Allopurinol sodium
Treatment of Duchenne muscular dystrophy

Exon 52 specific phosphorothioate oligonucleotide
Treatment of Duchenne muscular dystrophy

Exon 55 specific phosphorothioate oligonucleotide
Treatment of malaria

Artesunate
Treatment of chronic lymphocytic leukaemia

4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide
Treatment of very-long-chain-acyl-CoA dehydrogenase deficiency

Triheptanoin
Treatment of long-chain L-3-hydroxyacyl-CoA-dehydrogenase deficiency

Triheptanoin
Treatment of chronic lymphocytic leukaemia

Humanised single chain monoclonal antibody against CD37
Treatment of non-infectious uveitis

Voclosporin
November 2012
Treatment of mercury toxicity

Erdosteine
Treatment of cutaneous T-cell lymphoma

Naloxone hydrochloride dehydrate
Treatment of glioma

IL-12-secreting dendritic cells, loaded with autologous tumour lysate
Treatment of malignant thymoma

Milciclib maleate
Treatment of systemic light chain amyloidosis

Ixazomib
Treatment of idiopathic pulmonary fibrosis

Tralokinumab
Treatment of neuroblastoma

Chimeric monoclonal antibody against GD2
Treatment of multiple myeloma

Panobinostat
Treatment of ovarian cancer

Alisertib
Treatment of glioma

Synthetic double-stranded siRNA oligonucleotide directed against claudin-5 complexed with polyethyleneimine (prior to administration of doxorubicin)
Treatment of senile systemic amyloidosis

tafamidis
Treatment of African tripanosomiasis

Melarsoprol
Treatment of retinitis pigmentosa

Adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5 (prior to administration of 17- dimethylaminoethylamino-17-demethoxygeldanamycin)
Treatment of dyskeratosis congénita

Recombinant human dyskerin
Treatment of tumour necrosis factor receptor-associated periodic syndrome

Canakinumab
Treatment of macular telangiectasia type 2

Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor
October 2012
Treatment of cystic fibrosis

Alpha-1 proteinase inhibitor (for inhalation use)

Treatment of fragile X syndrome

Mavoglurant

Treatment of acute lung injury

Asp-Arg-Val-Try-Ile-His-Pro

Treatment of pancreatic cancer

Mixture of two allogeneic human pancreatic cancer cell lines stably transduced with a retroviral vector encoding the murine alpha-(1,3)-galactosyltransferase gene

Treatment of ovarian cancer

Rucaparib

Treatment of traumatic spinal cord injury

[2-Cyano-3-cyclopropyl-3-hydroxy-N-(3-methyl-4-trifluoromethylphenyl)prop-2-enamide]

Treatment of lecithin cholesterol acyltransferase deficiency

Recombinant human lecithin cholesterol acyltransferase

Treatment of haemophilia A

Humanised monoclonal IgG4 antibody against tissue factor pathway inhibitor

Treatment of ovarian cancer

Lurbinectedin

Treatment of chronic lymphocytic leukaemia

Obinutuzumab

Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)

Belinostat

Treatment of acute myeloid leukaemia

Liposomal daunorubicin
September 2012
Diagnosis of positive folate receptor status in ovarian cancer

N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-Lalpha-aspartyl-L-cysteine to be used with folic acid

Diagnosis of positive folate receptor status in ovarian cancer

Folic acid to be used with N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-Lalpha-

aspartyl-L-cysteine

August 2012
Treatment of primary biliary cirrhosis

(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid

MARKETING AUTHORISATION

Bosulif [bosutinib]

Treatment of adults with chronic myeloid leukaemia (CML)

Pfizer Limited

United-Kingdom

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
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).
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.

Glybera (alipogene tiparvovec)

Treatment of lipoprotein lipase deficiency

uniQure biopharma B.V.

The Netherlands

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.
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.

Adcetris ( brentuximab vedotin)

Treatment of Hodgkin lymphoma

Takeda Global Research and Development Centre (Europe) Ltd

United Kingdom
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).
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);

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.

Signifor (pasireotide)

Treatment of Cushing’s disease

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.
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.

Dacogen (decitabine)

acute myeloid leukaemia (AML)

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.
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.

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.

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).
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.

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.
Detailed information on European orphan drug designation applications is available on the EMA website
A full list of designated and authorised orphan drugs in Europe available at: ec.europa.eu
Page created: 27/04/2012
Page last updated: 29/04/2013

Show more