Latest orphan drug designations and/or marketing authorisations

ShareThis

ORPHAN DRUG DESIGNATIONS

November 2013
Treatment of glioma
Autologous ex-vivo-expanded leucocytes treated with 5-aza-2’-deoxycytidine
Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Recombinant human insulin receptor monoclonal antibody-fusediduronate 2-sulfatase
Treatment of follicular thyroid cáncer
Sorafenib tosylate
Treatment of papillary thyroid cáncer
Sorafenib tosylate
Prevention of graft-versus-host disease
Defibrotide
Treatment of hepatocellular carcinoma
Tivantinib
Treatment of diffuse large B-cell lymphoma
Ibrutinib
Prevention of arteriovenous access dysfunction in patients undergoing surgical creation of an arteriovenous access for haemodialysis
Sirolimus
Treatment of eosinophilic oesophagitis
Human monoclonal antibody against human interleukin 13
Treatment of spinal cord injury
Synthetic 12 amino acids peptide designed after subcommissural organ spondin
Treatment of ovarian cáncer
Trebananib
Treatment of Stargardt’s disease
Soraprazan
October 2013
Treatment of congenital factor VII deficiency
Recombinant fusion protein linking coagulation factor VIIa with Albumin
Treatment of myotonic disorders
Mexiletine hydrochloride
Treatment of plasma cell myeloma
Recombinant human monoclonal IgM antibody targeting glucose-regulated protein 78
Treatment of sarcoidosis
L-Pyr-L-Glu-L-Gln-L-Leu-L-Glu-L-Arg-L-Ala-L-Leu-L-Asn-LSer-L-Ser
Treatment of complex regional pain syndrome
Zoledronic acid
Treatment of Allan-Herndon-Dudley syndrome
3,5-diiodothyropropionic acid
Treatment of Duchenne muscular dystrophy
Naproxcinod
Treatment of cystic fibrosis
Antisense oligonucleotide targeting the F508delta mutation of CFTR
Treatment of Wiskott-Aldrich-syndrome
Autologous CD34+ cells transduced with a lentiviral vector containing the human Wiskott-Aldrich syndrome gene
July & August 2013
Treatment of pulmonary alveolar proteinosis
Granulocyte macrophage colony stimulating factor
Autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors
Autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors
Prevention of ischaemia/reperfusion injury associated with solid organ transplantation
Human hemin
Treatment of malignant thymomas
Belinostat
Treatment of pancreatic cancer
(1-methyl-2-nitro-1H-imidazole-5-yl) methyl N,N’-bis(2-bromoethyl) diamidophosphate
Treatment of plasma cell myeloma
Daratumumab
Treatment of ovarian cancer
Fosbretabulin tromethamine
Treatment of amyotrophic lateral sclerosis
Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
Prevention of hepatitis B re-infection following liver transplantation
Recombinant human monoclonal antibody against hepatitis B Virus
Treatment of ataxia telangiectasia
Dexamethasone sodium phosphate encapsulated in human autologous erythrocytes
Treatment of carbamoylphosphate synthase-1 deficiency
Heterologous human adult liver-derived progenitor cells
Treatment of citrullinaemia type 1
Heterologous human adult liver-derived progenitor cells
Treatment of argininosuccinic aciduria
Heterologous human adult liver-derived progenitor cells
Treatment of hyperargininaemia
Heterologous human adult liver-derived progenitor cells
Treatment of N-acetylglutamate synthetase (NAGS) deficiency
Heterologous human adult liver-derived progenitor cells
Treatment of citrullinaemia type 2
Heterologous human adult liver-derived progenitor cells
Treatment of ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome)
Heterologous human adult liver-derived progenitor cells
Treatment of limbal stem cell deficiency
Ex-vivo expanded autologous human corneal epithelium containing stem cells
Treatment of osteosarcoma
Lipid-complexed cisplatin
Treatment of acromegaly
Octreotide acetate (oral use)
Treatment of nodal marginal zone lymphoma
Idelalisib
Treatment of chronic lymphocytic leukaemia/small lymphocytic lymphoma
Idelalisib
Treatment of acute myeloid leukaemia
Trans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bis-hydrochloride
Treatment of autosomal dominant polycystic kidney disease
Tolvaptan
Treatment of nontraumatic osteonecrosis
Human allogeneic bone marrow derived osteoblastic-like cells
Treatment of pancreatic cancer
Chimeric monoclonal antibody against claudin-18 splice variant 2
Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Pegylated recombinant anti-Pseudomonas aeruginosa PcrV Fab′antibody
Treatment of growth hormone deficiency
Recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains
Treatment of Behçets' disease
Apremilast
Treatment of eosinophilic oesophagitis
Budesonide
Treatment of mastocytosis
Cladribine
Treatment of congenital sucrase-isomaltase deficiency
Sacrosidase
Treatment of sickle cell disease
(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
Treatment of neuromyelitis optica
Eculizumab
Treatment of extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma)
Idelalisib
Treatment of splenic marginal zone lymphoma
Idelalisib

June 2013
Treatment of cystic fibrosis
4,6,4’–trimethylangelicin
Treatment of amyotrophic lateral sclerosis
Sodium chlorite
Treatment of retinitis pigmentosa
Expanded human allogeneic neural retinal progenitor cells extracted from neural retina
Treatment of pachyonychia congenital
Synthetic double-stranded siRNA oligonucleotide directed against the keratin 6a N171K mutation
Treatment of retinitis pigmentosa
Adenovirus associated viral vector serotype 5 containing the human pde6β gene
Treatment of acute liver failure
Immortalised human C3A hepatoblastoma cells
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Recombinant human alpha-N-acetylglucosaminidase
Treatment of soft tissue sarcoma
Genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor
Treatment of retinitis pigmentosa
Unoprostone isopropyl
Treatment of non-dystrophic myotonia
Mexiletine hydrochloride
Treatment of B-cell acute lymphoblastic leukaemia
Inotuzumab ozogamicin
Treatment of adrenocortical carcinoma
N-[2,6-bis(1-methylethyl)phenyl]-N’-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt
Treatment of graft-versus-host disease
Allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media
Treatment of transglutaminase-1-deficient autosomal recessive congenital ichthyosis
Recombinant human transglutaminase 1 encapsulated into liposomes
Treatment of cystic fibrosis
Recombinant human CXCL8 mutant
Treatment of malignant mesothelioma
N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride
Treatment of cytomegalovirus disease in patients with impaired cell mediated immunity
Maribavir
Treatment of adenosine deaminase-deficient-severe combined immunodeficiency
Autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene
Treatment of retinitis pigmentosa
Recombinant human nerve growth factor

April 2013
Treatment of follicular thyroid cancer
Lenvatinib
Treatment of glioma
4-[2-(6-methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate
Treatment of papillary thyroid cancer
Lenvatinib
Treatment of Duchenne muscular dystrophy
R,S-O-(3-piperidino-2-hydroxy-1-propyl)-nicotinic acid amidoxime dihydrochloride
Treatment of idiopathic pulmonary fibrosis
Nintedanib
Treatment of Niemann-Pick disease, type C
2-hydroxypropyl-β-cyclodextrin
Treatment of chronic lymphocytic leukaemia/small lymphocytic lymphoma
(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one
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

MARKETING AUTHORISATIONS

Procysbi (cysteamine (as mercaptamine bitartrate)

Treatment of nephropathic (kidney)cystinosis
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
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

Revlimid (lenalidomide)
Extension of indication:
Treatment of myelodysplastic syndromes
Celgene Europe Ltd.
United Kingdom
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.
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
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: 25/11/2013