Latest orphan designations and/or marketing authorisations
MARKETING AUTHORISATION
Bronchitol [mannitol]
cystic fibrosis
Pharmaxis Pharmaceuticals Limited
United Kingdom
13/04/2012
What is Bronchitol?
Bronchitol is a medicine that contains the active substance mannitol. It is available as capsules (40 mg) containing a dry powder for inhalation using an inhaler device.
What is Bronchitol used for?
Bronchitol is used for the treatment of cystic fibrosis in adults in addition to best standard of care.
Cystic fibrosis is an inherited disease that affects the cells in the lungs and the glands in the gut and pancreas that secrete fluids such as mucus and digestive juices. In cystic fibrosis these fluids become thick and viscous, 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 Bronchitol was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 7 November 2005.
The medicine can only be obtained with a prescription.
ORPHAN DRUG DESIGNATIONS
August 2012Treatment of glioma
Humanised monoclonal antibody against epidermal growth factor receptor
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Vatreptacog alfa (activated)
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Treatment of haemophilia B
Vatreptacog alfa (activated)
Treatment of Fabry disease
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Treatment of peripheral T-cell lymphoma (nodal, other extranodal and
leukaemic/disseminated)
Recombinant anti-CD3-bi-single-chain-Fv-diphtheria toxin fusion protein
Treatment of cutaneous T-cell lymphoma
Recombinant anti-CD3-bi-single-chain-Fv-diphtheria toxin fusion protein
Treatment of Alagille syndrome
(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
July 2012
Treatment of gastric cancer
Ramucirumab
Treatment of autosomal recessive congenital ichthyosis
Talarozole
Treatment of sickle cell disease
Human erythrocytes encapsulating inositol hexaphosphate
Treatment of Duchenne muscular dystrophy
Givinostat
Treatment of Becker muscular dystrophy
Ataluren
Treatment of sickle cell disease
Levoglutamide
Treatment of Cushing’s syndrome
2S, 4R ketoconazole
Treatment of progressive multifocal leukoencephalopathy
Recombinant human interleukin-7
Treatment of keratinopathic ichthyosis
Talarozole
Treatment of infection-associated haemolytic uraemic syndrome
Eculizumab
Treatment of neuroblastoma
16-base single-stranded peptide nucleic acid oligonucleotide linked to 7-amino acid peptide
Treatment of recessive X-linked ichthyosis
Talarozole
Treatment of glycogen storage disease type II (Pompe's disease)
Recombinant adeno-associated viral vector containing human acid alfa-glucosidase-gene
Treatment of hepatocellular carcinoma
Ramucirumab
Treatment of idiopathic pulmonary fibrosis
Recombinant human pentraxin-2
Prevention of poliomyelitis in patients with immunodeficiencies deemed at risk
1-[(2-Chloro-4-methoxyphenoxy)methyl]-4-[(2,6-dichlorophenoxy)methyl]benzene
Treatment of Familial Partial Lipodystrophy
Metreleptin
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Metreleptin
Treatment of Lawrence syndrome
Metreleptin
Treatment of Berardinelli-Seip syndrome
Metreleptin
Treatment of calciphylaxis
Hexasodium phytate
Treatment of congenital hypotransferrinaemia
Human apotransferrin
Treatment of progressive familial intrahepatic cholestasis
(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
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
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