Orphan drugs in Portugal

October 28 ,2013 | By Márcio Barra

Tafamidis (vyndaqel), from Pfizer, sold in Portugal since 05 May 2012

What follows is a list of Orphan Drugs available in Portugal. This data has been compiled from two different databases, the INFOMED database, managed by the Portuguese National Competent Authrority on Medicines, INFARMED, and the OrphaNet database, from which sales numbers from Portugal were obtained, when available (note, sales numbers were, according to the Orphanet Website, last updated in September 28, 2013). The Portuguese Marketing approval date was also provided,

In the Market Status, you may find “no data” on some drugs. This means that the drug in question does not appear on the INFOMED database, only on the Orphanet Database. There may also be cases where the drug has a sales number, but it is not currently approved for sale by INFARMED. This could mean that the drug was sold under a Special Use Authorization request.

This page may be updated as more informations becomes available.

If you wish the table in a .PDF format, send an e-mail to marciobarra@ua.pt

Orphan Drugs in Portugal

Active Substance Brand Name Marketing authorization holder Market Status and Nº of units sold/consumed Dosage
Zinc acetate Wilzin Orphan Europe, S.A.R.L. Sold since 21/03/2007

 

3.163

25 mg
Zinc acetate Wilzin Orphan Europe, S.A.R.L. Sold since 21/03/2007

 

11.120

50 mg
Alglucosidase alfa Myozyme Genzyme Europe, B.V. Sold since 21/03/2007

 

5.284

50 mg
Alipogene tiparvovec Glybera Uniqure Biopharma B.V. Not approved for sale by INFARMED ( EMA’s approval date – 25/10/2012) 3 x 10^12 genome copies /ml
Ambrisentan Volibris Glaxo Group Limited Sold since 01/05/2010

 

901

10 mg
Ambrisentan Volibris Glaxo Group Limited Blister of 30 units sold since 01/07/2009

 

6.056

5 mg
Amifamipridine Firdapse BioMarin Europe Ltd Blister of 100 units – not sold

 

4.661

10 mg
Anagrelide Xagrid Shire Pharmaceuticals Contracts Ltd. Sold since 01/01/2004

 

82.818

0.5 mg
Azacitidina Vidaza Celgene Europe Limited Sold since 02/04/2009

 

4.722

25 mg/ml
Aztreonam Cayston Gilead Sciences International Ltd. Sold since 29/06/2011

 

4.047

75 mg/1 ml
Betaine Cystadane Orphan Europe, S.A.R.L. Not sold

 

78

1000 mg
Bosentan Tracleer Actelion Registration, Ltd Sold since 30/12/2002

 

99.513

125 mg
Bosentan Tracleer Actelion Registration, Ltd Sold since 23/02/2010

 

1.045

32 mg
Bosentan Tracleer Actelion Registration, Ltd Sold since 30/12/2002

 

13.913

62.5 mg
Buslfan   538 60 mg/10 ml
Bosutinib Bosulif Pfizer, Ltd. Sold since 09/09/2013 100 mg
Bosutinib Bosulif Pfizer, Ltd. Sold since 09/09/2013 500 mg
Brentuximab vedotina Adcetris Takeda Global Research & Development Centre (Europe) Ltd. Not sold 50 mg
Bromelain Nexobrid Teva Pharma GmbH. No data 2 g/22 g
Bromelain Nexobrid Teva Pharma GmbH. No data 5 g/55 g
Caffeine Peyona Chiesi Farmaceutici, S.p.A. No data 20 mg/ml
Cladribine LITAK Lipomed GmbH Sold since 21/03/2007

 

27

2 mg/ml
Clofarabine Evoltra Genzyme Europe, B.V Not sold

 

148

1 mg/ml
Dasatinib Sprycel Bristol-Myers Squibb Pharma EEIG Not sold

 

11.464

100 mg
Dasatinib Sprycel Bristol-Myers Squibb Pharma EEIG Sold since 02/01/2012

 

1.954

140 mg
Dasatinib Sprycel Bristol-Myers Squibb Pharma EEIG Sold since 01/10/2009

 

645

20 mg
Dasatinib Sprycel Bristol-Myers Squibb Pharma EEIG Sold since 01/10/2009

 

12.217

50 mg
Dasatinib Sprycel Bristol-Myers Squibb Pharma EEIG Sold since 02/01/2012

 

60

80 mg
Dasatinib Sprycel Bristol-Myers Squibb Pharma EEIG Not sold 70 mg
Decitabine Dacogen Janssen-Cilag International N.V. Not sold 50 mg
Desferrasirox Exjade Novartis Europharm, Ltd. Sold since 29/05/2008

 

407

125 mg
Desferrasirox Exjade Novartis Europharm, Ltd. Sold since 29/05/2008

 

833

125 mg
Desferrasirox Exjade Novartis Europharm, Ltd. Sold since 22/04/2008

 

784

250 mg
Desferrasirox Exjade Novartis Europharm, Ltd. Sold since 22/04/2008

 

1.200

250 mg
Desferrasirox Exjade Novartis Europharm, Ltd. Sold since 15/01/2008

 

4.513

500 mg
Desferrasirox Exjade Novartis Europharm, Ltd. Sold since 15/01/2008

 

6.496

500 mg
Eculizumab Soliris Alexion Europe SAS Not sold

 

39

300 mg
Estiripentol Diacomit Biocodex Not sold

 

2.160

250 mg
Estiripentol Diacomit Biocodex No data

 

450

250 mg
Estiripentol Diacomit Biocodex Not sold

 

4.854

250 mg
Estiripentol Diacomit Biocodex Not sold

 

1.890

500 mg
Estiripentol Diacomit Biocodex No data

 

1.803

500 mg
Estiripentol Diacomit Biocodex Not sold

 

5.124

500 mg
Everolimus Votubia Novartis Europharm, Ltd. Sold since 14/12/2012

 

732

5 mg
Everolimus Votubia Novartis Europharm, Ltd. Sold since 28/08/2012

 

90

2.5 mg
Everolimus Votubia Novartis Europharm, Ltd. No data 10 mg
Galsulfase Naglazyme BioMarin Europe Limited Sold since 21/03/2007

 

1.766

1 mg/ml
Hydrocortisone Plenadren ViroPharma SPRL – BVBA Approved, but no data on marketing status 20 mg
Hydrocortisone Plenadren ViroPharma SPRL – BVBA Approved, but no data on marketing status 5 mg
hydroxycarbamide Siklos Addmedica Not sold (EMA approval date -28/02/2011) 100 mg
hydroxycarbamide Siklos Addmedica Not sold (EMA approval date -28/02/2011) 1000 mg
Histamine Ceplene Meda AB Not sold 0.5 ml
Ibuprofen Pedea Orphan Europe, S.A.R.L. Sold since 21/03/2007

 

322

5 mg/ml
Icatibant Firazyr Shire Orphan Therapies GmbH Sold since 16/02/2009

 

13

30 mg/3 ml
Idursulfase Elaprase Shire Human Genetic Therapies AB Sold since 01/01/2007

 

321

2 mg/ml
Iloprost Ventavis Bayer Pharma A.G Sold since 21/03/2007

 

18.695

10 µg/ml
Ivacaftor Kalydeco Vertex Pharmaceuticals (U.K.) Limited Not sold 150 mg
Laronidase   No data

 

698

100 U/ml
Lenalidomide Revlimid Celgene Europe Limited Sold since 10/01/2008

 

4.522

10 mg
Lenalidomide Revlimid Celgene Europe Limited Sold since 10/01/2008

 

2.006

15 mg
Lenalidomide Revlimid Celgene Europe Limited Sold since 10/01/2008

 

6.573

25 mg
Lenalidomide Revlimid Celgene Europe Limited Sold since 10/01/2008

 

2.002

5 mg
Lenalidomide Revlimid Celgene Europe Limited No data 2.5 mg
Lenalidomide Revlimid Celgene Europe Limited No data 7.5 mg
Levodopa + Carbidopa Duodopa Abbvie, Lda. Sold since 27/01/2009 20 mg/ml + 5 mg/ml
Manitol Bronchitol Pharmaxis Pharmaceuticals Limited (No data, EMA’s authorization was on 2012) 40 mg
Mecasermin Increlex Ipsen Pharma SAS Sold since 12/04/2010

 

149

10 mg/ml
Mercaptamine Procysbi Raptor Pharmaceuticals Europe B.V. (No data, EMA’s approval date – 6/09/2013) 25 mg
Mercaptamine Procysbi Raptor Pharmaceuticals Europe B.V. (No data, EMA’s approval date – 6/09/2013) 75 mg
Mercaptopurine Xaluprine Nova Laboratories limited Sold since 01/01/2013

 

71

20 mg/ml
Mifamurtide Mepact Takeda France SAS Not sold 4 mg
Miglustat Zavesca Actelion Registration, Ltd. Sold since 01/02/2005

 

1.797

100 mg
Miglustat Zavesca Actelion Registration, Ltd. Sold since 01/02/2005

 

3.213

100 mg
Mitotane Lysodren Laboratoire HRA Pharma Sold since 21/03/2007

 

14.905

500 mg
Nelarabine Atriance Glaxo Group Limited Sold since 01/10/2008 5 mg/ml
Nilotinib Tasigna Novartis Europharm, Ltd. Sold since 23/09/2013

 

525

150 mg
Nilotinib Tasigna Novartis Europharm, Ltd. Sold since 20/06/2008

 

14.995

200 mg
Nitisinone Orfadin Swedish Orphan International AB Sold since 21/03/2007

 

1.380

10 mg
Nitisinone Orfadin Swedish Orphan International AB Sold since 21/03/2007

 

3.600

5 mg
Nitisinone Orfadin Swedish Orphan International AB Sold since 21/03/2007 2 mg
Ofatumumab Arzerra Glaxo Group Limited Sold since 09/01/2012 1000 mg/50 ml
Ofatumumab Arzerra Glaxo Group Limited Sold since 09/01/2012 100 mg/5 ml
Pasireotido Signifor Novartis Europharm, Ltd Not sold 0.3 mg/ml
Pasireotido Signifor Novartis Europharm, Ltd Not sold 0.6 mg/ml
Pasireotido Signifor Novartis Europharm, Ltd Not sold 0.9 mg/ml
Pirfenidona Esbriet InterMune UK Ltd Not sold 267 mg
Pegvisomant   No data

 

2.093

10 mg
Pegvisomant   No data

 

525

15 mg
Pegvisomant   No data

 

1.540

20 mg
Plerixafor Mozobil Genzyme Europe, B.V. Not sold

 

59

20 mg/ml
Pomalidomida Pomalidomida Celgene Celgene Europe Limited Not sold 1 mg
Pomalidomida Pomalidomida Celgene Celgene Europe Limited Not sold 2 mg
Pomalidomida Pomalidomida Celgene Celgene Europe Limited Not sold 3 mg
Pomalidomida Pomalidomida Celgene Celgene Europe Limited Not sold 4 mg
Ponatinib Iclusig ARIAD Pharma Ltd No data 15 mg
Ponatinib Iclusig ARIAD Pharma Ltd No data 45 mg
Romiplostim Nplate Amgen Europe, B.V Not sold

 

645

250 µg
Romiplostim Nplate Amgen Europe, B.V Not sold

 

75

500 µg
Romiplostim Nplate Amgen Europe, B.V Not sold 250 µg/0.5 ml
Romiplostim Nplate Amgen Europe, B.V Not sold 500 µg/1 ml
Rufinamide Inovelon Eisai Limited Sold since 01/07/2009

 

1

100 mg
Rufinamide Inovelon Eisai Limited Sold since 01/07/2009

 

328

200 mg
Rufinamide Inovelon Eisai Limited Sold since 01/07/2009 400 mg
Rufinamide Inovelon Eisai Limited Not sold 40 mg/ml
Ruxolitinib Jakavi Novartis Europharm, Ltd. Sold since 02/10/2013

 

180

15 mg
Ruxolitinib Jakavi Novartis Europharm, Ltd. Sold since 02/10/2013

 

1.140

20 mg
Ruxolitinib Jakavi Novartis Europharm, Ltd. Sold since 14/07/2009 5 mg
Sapropterin Kuvan Merck Serono Europe Ltd. Sold since 14/07/2009 100 mg
Sildenafil Revatio Pfizer, Ltd Sold since 19/04/2006

 

130.716

20 mg
Sildenafil Revatio Pfizer, Ltd Not sold 10 mg/ml
Sildenafil Revatio Pfizer, Ltd Sold since 12/07/2013 0.8 mg/ml
Sorafenib Nexavar Bayer Pharma A.G. Sold since 13/07/2007

 

48.930

200 mg
Tafamidis Vyndaqel Pfizer, Ltd. Sold since 05/05/2012

 

37.830

20 mg
Thalidomid Thalidomide Celgene Celgene Europe Limited Sold since 01/11/2008

 

83.730

50 mg
Teduglutide Revestive Nycomed Danmark ApS Not sold 5 mg
Temsirolímus Torisel Pfizer, Ltd. Sold since 26/03/2008

 

139

25 mg/ml
Thiotepa Tepadina Adienne S.r.l. Not sold

 

6

100 mg
Thiotepa Tepadina Adienne S.r.l. Not sold

 

63

15 mg
Tobramycin Tobi Podhaler Novartis Europharm, Ltd. Blister – 56 units – sold since 22/11/2011 28 mg
Trabectedin Yondelis Pharma Mar, S.A. Sold since 19/07/2010

 

124

0.25 mg
Trabectedin Yondelis Pharma Mar, S.A. Sold since 19/07/2010

 

326

1 mg
TArsenic Trioxide   No data

 

90

1 mg/ml
Velaglucerase alfa Vpriv Shire Pharmaceuticals (Ireland) Ltd. Not sold

 

330

200 U
Velaglucerase alfa Vpriv Shire Pharmaceuticals (Ireland) Ltd. Sold since 01/01/2010

 

290

400 U
Ziconotide Prialt Eisai Limited Not sold

 

2

0.1 mg/1 ml
Ziconotide Prialt Eisai Limited No data 25 µg/ml
 5-Aminolevulinic acid hydrochloride Gliolan Medac Gesellschalft fur Klinische Spezialpraparate GmbH Sold since 02/12/2008 1.5 g
Carglumic acid Carbaglu Orphan Europe, S.A.R.L. Sold since 21/03/2007 200 mg
Cholic Acid Orphacol Laboratoires CTRS (Cell Therapies Research & Services) No data 250 mg
Cholic Acid Orphacol Laboratoires CTRS (Cell Therapies Research & Services) No data 50 mg

Sources:

INFOMED

ORPHANET

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