viernes, 30 de septiembre de 2011

Register of designated Orphan Medicinal Products (alphabetical)

New designations August 2011 

Treatment of multiple myeloma

Treatment of focal segmental glomerulosclerosisFresolimumab

Treatment for mobilisation of progenitor cells prior to stem cell transplantationLow molecular weight dextran sulfate  

Prevention of ischaemia/reperfusion injury associated with solid organ transplantationMethyl O-4-O-[2-[2-[2-[2-[[N-[(1R)-1-[[4-(aminoiminomethyl)phenyl]methyl]-2-oxo-2-(1-piperidinyl)ethyl]- N2-[(4-methoxy-2,3,6-trimethylphenyl)sulfonyl]-L-α-asparaginyl-4-aminobutanoyl-N6-[5-[(3aS,4S,6aR)-hexahydro-2-oxo-1H-thieno[3,4-d]imidazol-4-yl]-1-oxopentyl]-Llysyl] amino]ethoxy]ethoxy]ethoxy]ethyl]-2,3-di-O-methyl-6-O-sulfo-α-Dglucopyranosyl-(1->4)- O-2,3-di-O-methyl-β-D-glucopyranuronosyl-(1->4)- O-2,3,6-tri-
O-sulfo-α-D-glucopyranosyl-(1->4)- O-2,3-di-O-methyl-α-L-idopyranuronosyl-(1->4)-3-
O-methyl-α-D-glucopyranoside 2,6-bis(hydrogen sulfate) octasodium salt

Treatment of pancreatic cancerMixture of seven synthetic fragments consisting of p21 RAS peptides

Treatment of post-polycythaemia vera myelofibrosisN-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl)phenyl]amino}pyrimidin-4-yl)benzamide, dihydrochloride salt

Treatment of post-essential thrombocythaemia myelofibrosisN-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl)phenyl]amino}pyrimidin-4-yl)benzamide, dihydrochloride salt

Treatment of primary myelofibrosisN-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl)phenyl]amino}pyrimidin-4-yl)benzamide, dihydrochloride salt

Treatment of acute lymphoblastic leukaemiaPegylated recombinant Erwinia chrysanthemi L-asparaginase

Treatment of hepatocellular carcinomaPeretinoin

Post-exposure prophylaxis of inhalation anthrax diseaseHuman anthrax monoclonal antibody

Treatment of 5q spinal muscular atrophy5-[1-(2,6-dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride

Treatment of acute liver failureCardiotrophin-1

Treatment of gastric cancerEverolimus

Treatment of Niemann-Pick disease, type CHydroxy-propyl-beta-cyclodextrin

Treatment of cystic fibrosisMultilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phospatidylinositol and cholesterol

Treatment of tuberculosisN-{[(5S)-3-(3-fluoro-4-thiomorpholin-4-ylphenyl)-2-oxo-1,3-oxazolidin-5-yl]methyl}acetamide

Treatment of chronic non-infectious uveitisSirolimus

New Marketing Authorisations

Tobi Podhaler
Novartis Europharm Limited
United Kingdom

What is Tobi Podhaler?
Tobi Podhaler is a medicine that contains the active substance tobramycin. It is available as capsules (28 mg) containing a dry powder for inhalation using a portable inhaler device.

What is Tobi Podhaler used for?
Tobi Podhaler is used to suppress chronic lung infection caused by bacteria called Pseudomonas aeruginosa in adults and children aged six years and over who have cystic fibrosis. Cystic fibrosis is an inherited disease that affects the cells in the lungs and the glands in the gut and pancreas which secrete fluids such as mucus and digestive juices. The accumulation of mucus in the lungs allows bacteria to grow more easily causing infections, lung damage and breathing problems. Bacterial lung infection with P. aeruginosa is frequent in cystic fibrosis patients.

Because the number of patients with cystic fibrosis and P. aeruginosa bacterial lung infection is low, the disease is considered 'rare' and Tobi Podhaler was designated an 'orphan medicine' (a medicine used in rare diseases) on 17 March 2003.

The medicine can only be obtained with a prescription.

All Orphan Drugs in Europe
European Public Assessment Reports (EPARs)  >>

open here please to see/read/learn about Orphan Drugs:
Register of designated Orphan Medicinal Products (alphabetical)

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