Table 1 CDR for rare diseases

19/02/2004

Replagal

agalsidase alfa

Transkaryotic Therapies Inc.

Biologic/Enzyme replacement therapy

Fabry Disease

0.22^a

Do not list

24/02/2004

Fabrazyme

agalsidase beta

Genzyme Canada Inc.

Biologic/Enzyme replacement therapy

Fabry Disease

0.22^a

Do not list

14/07/2004

Remodulin

Treprostinil sodium

Northern Theraputics Inc.

Chemical/Vasodilating Agents

Pulmonary arterial hypertension

3.3^a

Do not list

24/11/2004

Zavesca

miglustat

Actelion Pharmaceuticals Canada Inc.

Chemical/Substrate reduction therapy, Enzyme Inhibitor; Glucosylceramide Synthase Inhibitor

Gaucher disease

1^a

Do not list

10/12/2004

Fabrazyme

agalsidase beta

Genzyme Canada Inc.

Biologic/Enzyme replacement therapy

Fabry Disease

0.22^a

Do not list

03/02/2005

Aldurazyme

laronidase

Genzyme Canada Inc.

Biologic/Enzyme replacement therapy

Mucopolysaccharidosis I

8^a

Do not list

25/01/2006

Somavert

pegvisomant

Pfizer Canada Inc.

Biologic/Growth hormone receptor antagonist

Acromegaly

5.5^a

Do not list

24/02/2006

Remodulin

Treprostinil sodium

Northern Theraputics Inc.

Chemical/Vasodilating Agents

Pulmonary arterial hypertension

3.3^a

List with criteria/condition

20/07/2006

Sutent

sunitinib

Pfizer Canada Inc.

Chemical/Antineoplastic Agent, Tyrosine Kinase Inhibitor; Antineoplastic Agent, Vascular Endothelial Growth Factor (VEGF) Inhibitor

Gastrointestinal stromal tumour

13^a

List with clinical criteria and/or conditions

10/10/2006

Myozyme

alglucosidase

Genzyme Canada Inc.

Biologic/Enzyme replacement therapy

Glycogen storage disease type II

0.8^a

List with clinical criteria and/or conditions

20/02/2007

Somatuline

lanreotide acetate

Ipsen Limited

Chemical/Somatostatin Analog

Acromegaly

5.5^a

List in a similar manner to other drugs in class

22/06/2007

Thelin

Sitaxsentan sodium

Encysive Canada Inc.

Chemical/Endothelin receptor antagonist

Pulmonary arterial hypertension

3.3^a

Do not list

13/07/2007

Elaprase

idursulfase

Shire Human Genetic Therapies (Canada) Inc.

Biologic/Enzyme replacement therapy

Mucopolysarccharidosis II

6.7^a

Do not list

29/08/2007

Xyrem

sodium oxybate

Valeant Canada Ltd.

Chemical/Central Nervous System Depressant

Narcolepsy

25^a

Withdrawn

05/05/2008

Thelin

Sitaxsentan sodium

Encysive Canada Inc.

Chemical/Endothelin receptor antagonist

Pulmonary arterial hypertension

3.3^a

Do not list

09/07/2008

Xyrem

sodium oxybate

Valeant Canada Ltd.

Chemical/Central Nervous System Depressant

Narcolepsy

25^a

Do not list

09/07/2008

Volibris

Ambrisentan

GlaxoSmithKline

Chemical/Endothelin receptor antagonist

Pulmonary arterial hypertension

3.3^a

List with clinical criteria and/or conditions

29/08/2008

Orencia

Abatacept

Bristol-Myers Squibb Canada

Biologic/Selective T Cell Costimulation Modulator

Juvenile idiopathic arthritis (JIA)

44.7^b

List with criteria/condition

12/06/2009

Omnitrope

somatropin (rDNA origin)

Sandoz Canada Inc.

Biologic/Growth hormone

Growth hormone deficiency in children and adults

37^c

List in a similar manner to other drugs in class

12/06/2009

Xeomin

clostridium botulinum neurotoxin type A, free of complexing proteins

Merz Pharmaceuticals GmbH

Biologic/Neuromuscular Blocker Agent, Toxin; Ophthalmic Agent, Toxin

Blepharospasm

16.43^d

List in a similar manner to other drugs in class

12/06/2009

Xeomin

clostridium botulinum neurotoxin type A, free of complexing proteins

Merz Pharmaceuticals GmbH

Biologic/Neuromuscular Blocker Agent, Toxin; Ophthalmic Agent, Toxin

Cervical dystonia

16.43^d

List in a similar manner to other drugs in class

18/09/2009

Soliris

eculizumab

Alexion Pharmaceuticals Inc.

Biologic/Monoclonal Antibody; Monoclonal Antibody, Complement Inhibitor

Paroxysmal nocturnal hemoglobinuria

0.2^a

Do not list

29/09/2009

Nplate

Romiplostim

Amgen Canada Inc.

Biologic/Colony Stimulating Factor; Hematopoietic Agent; Thrombopoietic Agent

Immune thrombocytopenic purpura

25^a

Do not list

05/02/2010

Adcirca

Tadalafil

Eli Lilly Canada Inc.

Chemical/Enzyme inhibitor

Pulmonary arterial hypertension

3.3^a

List with criteria/condition

07/07/2010

Ilaris

canakinumab

Novartis Pharmaceuticals Canada Inc.

Biologic/Interleukin-1 Beta Inhibitor; Interleukin-1 Inhibitor; Monoclonal Antibody

Cryopyrin-Associated Periodic Syndrome (CAPS)

0.3^e

Do not list

08/07/2010

Kuvan

sapropterin dihydrochloride

BioMarin Pharmaceutical (Canada) Inc.

Chemical/Enzyme Cofactor, activates residual PAH enzyme

Phenylketonuria

10^a

Do not list

28/10/2010

Vpriv

velaglucerase alfa

Shire Human Genetic Therapies (Canada) Inc.

Biologic/Enzyme replacement therapy

Gaucher disease

1^a

List with clinical criteria and/or conditions

02/12/2010

Cayston

aztreonam

Gilead Sciences Canada Inc.

Chemical/Antibiotic

Cystic fibrosis

7.4^a

List with clinical criteria and/or conditions

26/04/2011

Revolade

Eltrombopag olamine

GlaxoSmithKline

Chemical/Colony Stimulating Factor; Hematopoietic Agent; Thrombopoietic Agent

Immune thrombocytopenic purpura

25^a

Do not list

01/06/2011

Kuvan

sapropterin dihydrochloride

BioMarin Pharmaceutical (Canada) Inc.

Chemical/Enzyme Cofactor, activates residual PAH enzyme

Phenylketonuria

10^a

NR

20/09/2011

Banzel

Rufinamide

Eisai Limited

Chemical/Anticonvulsant, Triazole Derivative

Lennox-Gastaut Syndrome

15^a

List with clinical criteria and/or conditions

24/01/2012

Mozobil

plerixafor injection

Genzyme Canada Inc.

Chemical/Hematopoietic Agent; Hematopoietic Stem Cell Mobilizer

Non-Hodgkin’s lymphoma and multiple myeloma

11.9^a

Do not list

30/01/2012

Actemra

tocilizumab

Hoffmann-La Roche Ltd.

Biologic/Antirheumatic, Disease Modifying; Interleukin-6 Receptor Antagonist

Juvenile idiopathic arthritis

44.7^b

List

24/02/2012

Rituxan

Rituximab

Hoffmann-La Roche Ltd.

Biologic/Antineoplastic Agent, Anti-CD20; Antineoplastic Agent, Monoclonal Antibody; Antirheumatic, Miscellaneous; Immunosuppressant Agent; Monoclonal Antibody

Granulomatosis with Polyangiitis

9^a

List with clinical criteria and/or conditions

27/09/2012

Kalydeco

ivacaftor tablets

Vertex Pharmaceuticals (Canada) Inc.

Chemical/Cystic Fibrosis Transmembrane Conductance Regulator Potentiator

Cystic fibrosis

7.4^a

List with criteria/condition

05/10/2012

Esbriet

pirfenidone

InterMune Canada Inc.

Chemical/Anti-inflammatory Agent; Antifibrotic Agent

Idiopathic pulmonary fibrosis (IPF)

42.7^f

Do not list

07/01/2013

Soliris

eculizumab

Alexion Pharmaceuticals Inc.

Biologic/Monoclonal Antibody; Monoclonal Antibody, Complement Inhibitor

Atypical Hemolytic Uremic Syndrome

0.85^a

Do not list

04/02/2013

Humira

adalimumab

AbbVie Corporation

Biologic/Antirheumatic, Disease Modifying; Gastrointestinal Agent, Miscellaneous; Monoclonal Antibody; Tumor Necrosis Factor (TNF) Blocking Agent

Juvenile idiopathic arthritis (JIA)

44.7^b

List with clinical criteria and/or conditions

08/03/2013

Afinitor

Everolimus

Novartis Pharmaceuticals Canada Inc.

Chemical/Antineoplastic Agent, mTOR Kinase Inhibitor; Immunosuppressant Agent; mTOR Kinase Inhibitor

Tuberous sclerosis

8.8^a

Do not list

27/05/2013

Genotropin GHD-A

somatropin

Pfizer Canada Inc.

Biologic/Growth Hormone

Growth hormone deficiency

37^c

List with clinical criteria and/or conditions

27/05/2013

Genotropin GHD-P

somatropin

Pfizer Canada Inc.

Biologic/Growth Hormone

Growth hormone deficiency

37^c

List with clinical criteria and/or conditions

27/05/2013

Genotropin TS

somatropin

Pfizer Canada Inc.

Biologic/Growth Hormone

Turner syndrome

50^g

List with criteria/condition

06/06/2013

Jetrea

ocriplasmin

Alcon Canada Inc.

Biologic/Ophthalmic Agent; Vitreolytic

Vitreomacular adhesion

40^h

List with criteria/condition

29/07/2013

Actemra

tocilizumab

Hoffmann-La Roche Ltd.

Biologic/Antirheumatic, Disease Modifying; Interleukin-6 Receptor Antagonist

Juvenile idiopathic arthritis (JIA)

44.7^b

List with criteria/condition

30/09/2013

Adempas

Ricoiguat

Bayer Inc.

Chemical/Soluble Guanylate Cyclase (sGC) Stimulator

Pulmonary arterial hypertension

3.3^a

List with criteria/condition

16/12/2013

Opsumit

Macitentan

Actelion Pharmaceuticals Canada Inc.

Chemical/Endothelin Receptor Antagonist; Vasodilator

Pulmonary arterial hypertension

3.3^a

List with criteria/condition

27/02/2014

Signifor

pasireotide

Novartis Pharmaceuticals Canada Inc.

Chemical/Somatostatin Analog

Cushing's Disease

4^a

Do not list

04/03/2014

Firazyr

icatibant

Shire Human Genetic Therapies (Canada) Inc.

Chemical/Selective Bradykinin B2 Receptor Antagonist

Hereditary angioedema

1^a

List with clinical criteria and/or conditions

19/03/2014

Afinitor

Everolimus

Novartis Pharmaceuticals Canada Inc.

Chemical./Antineoplastic Agent, mTOR Kinase Inhibitor; Immunosuppressant Agent; mTOR Kinase Inhibitor

Tuberous Sclerosis

8.8^a

Do not list

01/05/2014

Kalydeco

ivacaftor

Vertex Pharmaceuticals (Canada) Inc,

Chemical/Cystic Fibrosis Transmembrane Conductance Regulator Potentiator

Cystic fibrosis

7.4^a

List with criteria/condition

08/07/2014

Juxtapid

lomitapide

Aegerion Pharmaceuticals (Canada) Ltd.

Chemical/Antilipemic Agent, Microsomal Triglyceride Transfer Protein (MTP) Inhibitor

Homozygous Familial Hypercholesterolemia

0.6ⁱ

Do not list

05/08/2014

Vimizim

elosulfase alfa

BioMarin Pharmaceutical (Canada) Inc.

Biologic/Enzyme replacement therapy

Mucopolysaccharidosis IV

0.3^j

Do not list

29/08/2014

Esbriet

pirfenidone

Hoffmann-La Roche Ltd.

Chemical/Anti-inflammatory Agent; Antifibrotic Agent

Idiopathic pulmonary fibrosis (IPF)

42.7^f

List with criteria/condition

15/09/2014

Elelyso

Taliglucerase alfa

Pfizer Canada Inc.

Biologic/Enzyme replacement therapy

Gaucher disease

1^a

Do not list

30/10/2014

Diacomit

Stiripentol

Biocodex

Chemical/Anticonvulsant

Dravet Syndrome

2.5^a

List with criteria/condition

09/02/2015

Soliris

eculizumab

Alexion Pharmaceuticals Inc.

Biologic/Monoclonal Antibody; Monoclonal Antibody, Complement Inhibitor

Atypical Hemolytic Uremic Syndrome

0.85^a

Do not list

23/04/2015

Ofev

Nintedanib

Boehringer Ingelheim (Canada) Ltd.

Chemical/Tyrosine Kinase Inhibitor

Idiopathic pulmonary fibrosis (IPF)

42.7^f

List with criteria/condition

08/05/2015

Kalydeco

Ivacaftor

Vertex Pharmaceuticals (Canada) Inc.

Chemical/Cystic Fibrosis Transmembrane Conductance Regulator Potentiator

Cystic fibrosis

7.4^a

List with criteria/condition

25/06/2015

Adempas

Riociguat

Bayer Inc.

Chemical/Soluble Guanylate Cyclase (sGC) Stimulator

Pulmonary arterial hypertension

3.3^a

List with criteria/condition

20/07/2015

Strensiq

Asfotase alfa

Alexion Pharmaceuticals Inc.

Biologic/Enzyme replacement therapy

Hypophophatasia

1^k

Ongoing

20/08/2015

Naglazyme

Galsulfase

BioMarin Pharmaceutical (Canada) Inc.

Biologic/Enzyme replacement therapy

Mucopolysaccharidosis VI

0.3^j

Ongoing

24/11/2015

Ilaris

canakinumab

Novartis Pharmaceuticals Canada Inc.

Biologic/Interleukin-1 Beta Inhibitor; Interleukin-1 Inhibitor; Monoclonal Antibody

Juvenile idiopathic arthritis

44.7^b

Ongoing

27/11/2015

Orkambi

lumacaftor/ivacaftor

Vertex Pharmaceuticals (Canada) Inc.

Chemical/CFTR combinations

Cystic fibrosis

7.4^a

Ongoing