Drugs Designated As Orphan Drugs, Australia, July 2008

Main Category: Regulatory Affairs / Drug Approvals
Also Included In: Pharmacy / Pharmacist;  Pharma Industry / Biotech Industry
Article Date: 22 Jul 2008 - 1:00 PDT

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The following drugs have been designated as "orphan drugs" for the purposes of Regulation 16J of the Therapeutic Goods Regulations, as at 8 July 2008.

Drug: Adefovir dipivoxil
Sponsor: Gilead Sciences Pty Ltd
Date of designation: 12 August 2005
Indication: in monotherapy or in combination with other therapies, for prevention of recurrence of HBV infection in liver transplant recipients.

Drug: agalsidase beta (FABRAZYME)
Sponsor: Genzyme Australasia Pty Ltd
Date of designation: 22 May 2000
Indication: for long term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry Disease
Registered in Australia: 3 May 2002

Drug: agalsidase alfa (REPLAGAL)
Sponsor: PPD Australia Pty Limited
Date of designation: 20 October 2000
Indication: for the long-term enzyme replacement therapy in patients with Fabry disease
Registered in Australia: 17 May 2002

Drug: Aimspro (Goat Serum Derived Product)
Sponsor: Daval Australia Pty Ltd
Date of designation: 22 August 2005
Indication: for treatment of Krabbe's Disease (Globoid Cell Leucodystrophy)

Drug: aldesleukin (PROLEUKIN)
Sponsor: CSL
Date of designation: 16 October 1998
Indication: for the treatment of metastatic renal cell carcinoma
Registered in Australia: 24 May 2000

Drug: aldesleukin (PROLEUKIN)
Sponsor: CSL
Date of designation: 16 October 1998
Indication: to treat metastatic melanoma

Drug: algucosidase alfa (MYOZYME)
Sponsor: Genzyme Australasia Pty Ltd
Date of designation: 8 September 2003
Indication: for the treatment of Pompe disease (glycogen storage disease type II)
Dose: powder for concentrate for solution for infusion
Registered in Australia: 4 March 2008

Drug: AMBRISENTAN
Sponsor: GlaxoSmithKline Australia Pty Ltd
Date of designation: 5 July 2007
Indication: for the treatment of patients with pulmonary arterial hypertension (PAH) to improve exercise capacity, delay clinical worsening and decrease the symptoms of PAH.
Dose: tablets 5 mg & 10 mg

Drug: anagrelide hydrochloride (AGRYLIN)
Sponsor: Orphan Australia
Date of designation: 16 October 1998
Indication: for the treatment of essential thrombocytosis
Registered in Australia: 23 November 1999

Drug: anagrelide hydrochloride (THROMBOREDUCTIN)
Sponsor: Max Pharma Pty Ltd
Date of designation: 26 May 2008
Indication: for the treatment of essential thrombocythaemia in patients with high or intermediate risk of thrombosis or bleeding
Dose: capsules

Drug: Arsenic Trioxide Injection
Sponsor: Pharmalab Pty Limited
Date of designation: 21 August 2003
Indication: for the treatment of patients with acute promyelocytic leukaemia, who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy.

Drug: Arsenic Trioxide Injection
Sponsor: Pharmalab Pty Limited
Date of designation: 8 June 2004
Indication: for the treatment of patients with acute promyelocytic leukaemia

Drug: artemether and lumefantrine (RIAMET)
Sponsor: Novartis Pharm
Date of designation: 27 October 1999
Indication: for the treatment of children and adults with infections due to Plasmodium falciparum or mixed infections including P falciparum
Registered in Australia: 5 July 2002

Drug: artesunate injection
Sponsor: Link Pharmaceuticals trading as Link Medical Products Pty Ltd
Date of designation: 17 January 2007
Indication: for the treatment of severe malaria including cerebral malaria and as a second line treatment in chloroquine resistant malaria cases
Dose: solution for injection

Drug: AZTREONAM LYSINE
Sponsor: Gilead Sciences Pty Ltd
Date of designation: 16 July 2007
Indication: control of gram-negative bacteria, particularly pseudomonas aeruginosa, in the respiratory tract of patients with cystic fibrosis.
Dose: single-use 1 mL vial containing 75 mg aztreonam and 52.5 mg lysine lyophilized powder, supplied with a single-use diluent ampoule containing 1 mL sterile water for injection with 0.17% NaCl.

Drug: 5-azacytidine
Sponsor: Pharmion Pty Ltd
Date of designation: 2 September 2004
Indication: for the treatment of myelodysplastic syndrome

Drug: azacytidine (VIDAZA)
Sponsor: Celgene Pty Ltd
Date of designation: 17 June 2008
Indication: for the treatment of myelodysplastic syndrome
Dose: powder for suspension for injection

Drug: bicaVera range of sterile peritoneal dialysis solutions
Sponsor: Fresenius Medical Care Australia Pty Ltd
Date of designation: 29 October 2002
Indication: for use as a peritoneal dialysis solution in the management of end stage renal disease and acute renal failure

Drug: bortezomib (VELCADE)
Sponsor: Janssen-Cilag
Date of designation: 22 October 2003
Indication: for the treatment of resistant or relapsed multiple myeloma
Dose: powder for injection
Registered in Australia: 8 February 2006

Drug: bosentan
Sponsor: Omnicare Clinical Research
Date of designation: 12 February 2001
Indication: for the treatment of primary pulmonary hypertension or pulmonary hypertension secondary to scleroderma, in patients meeting WHO criteria for inclusion in functional class III-IV pulmonary hypertension
Registered in Australia: 12 November 2002

Drug: busulfan (BUSULFEX) injection ampoule
Sponsor: Orphan Australia Pty Ltd
Date of designation: 12 October 2005
Indication: for use in combination with other chemotherapeutic agents and/or radiotherapy as a conditioning regimen prior to haematopoietic progenitor cell transplantation
Registered in Australia: 13 November 2007

Drug: C1 esterase inhibitor (Berinert P)
Sponsor: CSL Limited
Date of designation: 10 April 2007
Indication: for the treatment of hereditary angioedema
Dose: powder for injection

Drug: Caffeine Citrate injection
Sponsor: Ophtalmic Laboratories Ltd
Date of designation: 2 August 2007
Indication: for the short term treatment of apnoea of prematurity of gestational age 28 to less than 33 weeks
Dose: solution for injection 40 mg/2mL

Drug: Caffeine Citrate Oral Solution
Sponsor: Ophtalmic Laboratories Ltd
Date of designation: 2 August 2007
Indication: for the short term treatment of apnoea of prematurity of gestational age 28 to less than 33 weeks
Dose: 25mg/5mL

Drug: carboprost as the tropmethamine (HEMABATE)
Sponsor: Pharmacia & Upjohn
Date of designation: 16 October 1998
Indication: for the treatment of post-partum haemorrhage due to uterine atony
Registered in Australia: 7 June 2001

Drug: carmustine (GLIADEL)
Sponsor: Orphan Australia Pty Ltd
Date of designation: 25 September 2003
Indication: for use as an adjunct to surgery to prolong survival in patients with malignant glioma
Registered in Australia: 27 October 2004

Drug: carmustine implants (GLIADEL wafers)
Sponsor: Rhone-Poulenc Rorer Australia
Date of designation: 16 October 1998
Indication: for use as an adjunct to surgery to prolong survival in patients with recurrent glioblastoma multiforme for whom surgical resection is indicated
Registered in Australia: 29 December 2000

Drug: celecoxib (CELEBREX)
Sponsor: Searle
Date of designation: 10 April 2000
Indication: to reduce the number of adenomatous colorectal polyps in familial adenomatous polyposis (FAP), as an adjunct to usual care (eg endoscopic surveillance, surgery)
Registered in Australia: 17 August 1999

Drug: cladribine (LITAK)
Sponsor: Orphan Australia Pty Ltd
Date of designation: 23 January 2003
Indication: for the treatment of hairy cell leukaemia and lymphoplasmacytic lymphoma (Waldenstrom's macroglobulinaemia)
Registered in Australia: 22 June 2004

Drug: clofarabine (EVOLTRA)
Sponsor: Hospira Australia Pty Ltd
Date of designation: 2 October 2007
Indication: for the treatment of acute myeloid leukaemia in adults 60 years of age and over and for the treatment of acute lymphoblastic leukaemia in paediatric patients who have relapsed or are refractory after receiving at least two prior regimens.
Dose: concentrated solution for infusion

Drug: COMBIVIR
Sponsor: GlaxoSmithKline Australia
Date of designation: 10 July 2007
Indication: for antiretroviral combination therapy for the treatment of Human Immunodeficiency Virus (HIV) infected children under 12 years of age
Dose: tablets (containing lamivudine 150 mg & zidovudine 300 mg)

Drug: cyclosporin
Sponsor: Delpharm Consultants Pty Ltd
Date of designation: 17 November 2004
Indication: for the prevention of graft rejection of lung transplants

Drug: cytarabine intrathecal injection (DEPOCYT)
Sponsor: Orphan Australia Pty Ltd
Date of designation: 7 January 2004
Indication: for the treatment of symptomatic neoplastic meningitis
Dose: 50mg/5mL injection vial
Registered in Australia: 28 June 2006

Drug: dasatinib (SPRYCEL)
Sponsor: Bristol-Myers Squibb Pharmaceuticals
Date of designation: 12 October 2005
Indication: for the treatment of chronic myeloid leukaemia in patients who are resistant to or intolerant of imatinib
Date of designation: 18 January 2006
Indication: for Philadelphia-positive acute lymphoblastic leukaemia (Ph+ ALL)
Dose: tablets
Registered in Australia: 9 January 2007

Drug: deferasirox (EXJADE)
Sponsor: Novartis Pharmaceuticals Australia Pty Ltd
Date of designation: 24 September 2004
Indication: for the treatment of chronic iron overload in patients with transfusion-dependent anaemias Dose: dispersible tablets
Registered in Australia: 30 June 2006

Drug: deferiprone (FERRIPROX)
Sponsor: Orphan Australia Pty Ltd
Date of designation: 22 May 2001
Indication: for the treatment of iron overload in patients with thalassaemia major, unwilling or unable to take desferrioxamine therapy
Registered in Australia: 2 April 2003

Drug: dornase alpha (PULMOZYME)
Sponsor: Roche Products Pty Limited
Date of designation: 28 April 2004
Indication: for the management of demonstrated respiratory complications in cystic fibrosis in children under the age of five years

Drug: DUODOPA (Levodopa / Carbidopa)
Sponsor: Solvay Biosciences Pty Ltd
Date of designation: 18 April 2006
Indication: for the treatment of advanced idiopathic Parkinson's disease with severe motor fluctuations and not responding to oral treatment
Dose: 20mg/mL and 5mg/mL intestinal gel PVC Bag
Registered in Australia: 14 February 2008

Drug: eculizumab (SOLIRIS)
Sponsor: Cedarglen Investments Pty Limited
Date of designation: 10 April 2007
Indication: for the treatment of paroxysmal nocturnal haemoglobinuria

Dose: solution for injection Drug: efavirenz (STOCRIN)
Sponsor: Merck Sharp & Dohme
Date of designation: 31 October 2000
Indication: for the treatment of HIV-1 infection in children and adolescents over the age of 3 years
Registered in Australia: 16 August 2002

Drug: ELAPRASE idursulfase-rhu Solution for IV Infusion vial
Sponsor: Genzyme Australasia Pty Ltd
Date of designation: 30 June 2006
Indication: is indicated for the long term treatment of patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II)
Registered in Australia: 21 January 2008

Drug: epoprostenol sodium (FLOLAN)
Sponsor: Glaxo Wellcome
Date of designation: 6 December 1999
Indication: for the long term treatment of primary pulmonary hypertension in New York Heart Association (NYHA) Class III and IV patients
Registered in Australia: 19 September 2001

Drug: epoprostenol sodium (FLOLAN)
Sponsor: GlaxoSmithKline Pty Ltd
Date of designation: 5 April 2006
Indication: for the treatment of Pulmonary Arterial Hypertension (PAH) associated with collagen vascular diseases, in New York Heart Association (NYHA) functional class III and class IV patients

Drug: eptacog alfa (NovoSeven)
Sponsor: Novo Nordisk Pharmaceuticals Pty Ltd
Date of designation: 14 January 2005
Indication: for the prevention and treatment of bleeding episodes in patients with congenital Factor VII deficiency or Glanzmann's Thrombasthenia.

Drug: Ethyl Eicosapentaenoate
Sponsor: Orphan Australia Pty Ltd
Date of designation: 15 July 2004
Indication: for the treatment of Huntington's Disease

Drug: Extraneal brand of icodextrin 7.5% w/v peritoneal dialysis solution
Sponsor: Baxter Healthcare Pty Ltd
Date of designation: 26 September 2001
Indication: for use as an alternative osmotic agent in dialysis solutions for long dwell exchange in patients treated with peritoneal dialysis
Registered in Australia: 17 October 2002

Drug: FEIBA NF Factor Eight Inhibitor Bypassing Fraction 500U powder for injection vial with diluent vial
Sponsor: Baxter Healthcare Pty Ltd
Date of designation: 22 June 2004
Indication: is indicated as second line therapy for the control of spontaneous bleeding episodes and use in surgery in haemophilia A or B patients with inhibitors, for whom recombinant human factor VIIa has failed, is contraindicated or has been associated with unacceptable adverse effects.
Registered in Australia: 13 December 2006

Drug: Factor Eight Inhibitor Bypassing Activity (FEIBA VH)
Sponsor: Baxter Healthcare Pty Limited
Date of designation: 20 May 2004
Indication: for the treatment of haemophilia A patients with Factor VIII inhibitor and the treatment of hameophilia B patients with Factor IX inhibitor.

Drug: factor VIII[rDNA]; (KOGENATE FS, SF; HELIXATE FS, SF)
Sponsor: Bayer
Date of designation: 17 June 1999
Indication: for the treatment and prophylaxis of bleeding in haemophilia A (congenital factor VIII deficiency) in previously treated and untreated patients, and in patients with Factor VIII inhibitors (neutralising antibodies) who continue to respond to Kogenate FS (ie in whom haemostasis is achieved) [Kogenate FS does not contain von Willebrand's Factor and hence is not indicated in von Willebrand's disease]
Registered in Australia: 28 February 2001

Drug: Fluocinolone Acetonide Intravitreal Implant (RETISERT)
Sponsor: Bausch & Lomb
Date of designation: 12 December 2005
Indication: for treatment of chronic, non-infectious uveitis affecting the posterior segment of the eye

Drug: fomivirsen sodium (VITRAVENE)
Sponsor: Ciba Vision Australia Pty Ltd
Date of designation: 1 April 1999
Indication: for the local treatment of cytomegalovirus (CMV) retinitis in patients with acquired immunodeficiency (AIDS)
Registered in Australia: 2 May 2000

Drug: G17DT (IMMUNOGEN)
Sponsor: Orion Clinical Services (Australia) Pty Ltd
Date of designation: 29 November 2002
Indication: for the treatment of gastric and pancreatic cancers

Drug: G17DT (IMMUNOGEN)
Sponsor: Kendle R & D Pty Ltd
Date of designation: 20 November 2003
Indication: for the treatment of gastric and pancreatic cancers

Drug: Galsulfase rch (NAGLAZYME)
Sponsor: Cedarglen Investments Pty Ltd
Date of designation: 18 January 2006
Indication: for long-term enzyme replacement therapy in the treatment of patients with mucopolysaccharidosis type VI (MPS VI)
Dose: solution for injection
Registered in Australia: 16 February 2007

Drug: gemtuzumab ozogamicin (MYLOTARG)
Sponsor: Wyeth Australia Pty Ltd
Date of designation: 25 October 2000
Indication: for the treatment of acute myeloid leukaemia

Drug: GENOTROPIN brand of somatropin (rbe)
Sponsor: Pfizer Australia Pty Ltd
Date of designation: 09 February 2007
Indication: for the treatment of growth disturbance in children with chronic renal insufficiency
Dose: powder for injection

Drug: histamine dihydrochloride
Sponsor: FH Faulding and Co Limited
Date of designation: 25 October 2000
Indication: for the treatment of metatastic melanoma (stage IV)

Drug: human plasma-derived protein C (CEPROTIN)
Sponsor: Baxter Healthcare Pty Limited
Date of designation: 23 January 2003
Indication: for the treatment of severe congenital protein C deficiency

Drug: Humatrope brands of somatropin powder for subcutaneous or intramuscular injection
Sponsor: Eli Lilly Australia Pty Ltd
Date of designation: 13 June 2002
Indication: for the treatment of growth failure in children born small for gestational age

Drug: icodextrin 7.5% (EXTRANEAL PERITONEAL DIALYSIS SOLUTION)
Sponsor: Baxter Healthcare Pty Ltd
Date of designation: 16 October 1998
Indication: for the treatment of life threatening, End Stage Renal Disease, for those patients who have been identified as requiring the specialised treatment of peritoneal dialysis, and who have subsequently demonstrated significantly reduced ultrafiltration with intraperitoneal hyperosmolar glucose Registered in Australia: 16 December 1999

Drug: iloprost trometamol (ILOMEDIN)
Sponsor: Schering P/L
Date of designation: 13 October 2000
Indication: for the treatment of primary pulmonary hypertension

Drug: imatinib mesylate (GLIVEC)
Sponsor: Novartis Pharmaceuticals Australia Pty Ltd
Date of designation: 12 August 2002
Indication: for the treatment of chronic myeloid leukaemia
Registered in Australia: 28 February 2003

Drug: imatinib mesylate (GLIVEC)
Sponsor: Novartis Pharmaceuticals Australia Pty Ltd
Date of designation: 10 December 2001
Indication: for the treatment of adult patients with unresectable and/or metastatic malignant gastrointestinal stromal tumours
Registered in Australia: 21 June 2002

Drug: imatinib mesylate (GLIVEC)
Sponsor: Novartis Pharmaceuticals Australia Pty Ltd
Date of designation: 03 May 2006
Indications:
-- Philadelphia-positive acute lymphoblastic leukaemia (Ph+ ALL)
-- Dermatofibrosarcoma protuberans (DFSP)
-- Myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with platelet-derived growth factor receptor (PDGFR) gene rearrangements
-- Idiopathic hypereosinophilic syndrome including acute and chronic eosinophilic leukaemia
-- Systemic mastocytosis without the D816V c-kit mutation.

Drug: imiglucerase (CEREZYME)
Sponsor: Genzyme Australia
Date of designation: 16 October 1998
Indication: for enzyme replacement for patients with a confirmed diagnosis of Gaucher's disease
Registered in Australia: 17 May 1999

Drug: mecasermin [rhIGF-1] injection (Increlex) 10mg/ml in 40 mg vial
Sponsor: Ipsen Pty Ltd
Date of designation: 1 July 2008
Indication: for the long-term treatment of growth failure in children and adolescents with severe primary insulin-like growth factor-1 deficiency (severe primary IGFD).
Dose: solution for subcutaneous injection.

Drug: indinavir sulfate (CRIXIVAN)
Sponsor: Merck Sharp & Dohme
Date of designation: 3 October 2000
Indication: for the treatment of HIV-1 infection in children and adolescents over the age of 3 years
Registered in Australia: 18 September 2001

Drug: infliximab
Sponsor: Schering-Plough
Date of designation: 12 October 2005
Indication: for reducing the signs and symptoms in patients with moderately to severely active polyarticular-course juvenile rheumatoid arthritis (JRA) who have had an inadequate response to methotrexate

Drug: lamivudine (ZEFFIX)
Sponsor: Glaxosmithkline
Date of designation: 19 June 2002
Indication: for the treatment of children (2 years and above) and adolescents with chronic hepatitis B and evidence of hepatitis B virus (HBV) replication. This indication is based on changes in serological and histological markers in clinical studies up to 18 months duration in patients with compensated liver disease

Drug: lamivudine (ZEFFIX 3TCTM)
Sponsor: Glaxo Wellcome Australia Ltd
Date of designation: 11 January 2001
Indication: for use in combination with other antiretroviral agents for the treatment of HIV infection in children
Registered in Australia: 18 December 2001

Drug: lanreotide (SOMATULINE AUTOGEL)
Sponsor: Ipsen Pty Limited
Date of designation: 19 March 2004
Indication: for the treatment of the symptoms of carcinoid syndrome associated with carcinoid tumours and the symptoms of VIPomas
Registered in Australia: 17 December 2004

Drug: laronidase (ALDURAZYME)
Sponsor: Genzyme Australiasia Pty Ltd
Date of designation: 8 August 2001
Indication: for the treatment of mucopolysaccharidosis
Registered in Australia: 4 May 2004

Drug: Leflunomide
Sponsor: Aventis Pharma
Date of designation: 8 April 2004
Indication: for treatment of polyarticular course juvenile rheumatoid arthritis in patient 3 to 17 years of age

Drug: lenalidomide (REVLIMID)
Sponsor: Celgene Pty Ltd
Date of designation: 6 September 2006
Dose: capsules
Indication: for:
-- the treatment of previously treated multiple myeloma patients in combination with dexamethasone; and
-- the treatment of patients with transfusion-dependent anaemia due to low or intermediate-1 risk myelodysplastic syndromes associated with a deletion 5q cytogenetic abnormality, with or without additional cytogenetic abnormalities.
Registered in Australia: 13 December 2007

Drug: lepirudin (REFLUDAN)
Sponsor: Hoechst Marion Roussel
Date of designation: 16 October 1998
Indication: for the treatment of acute heparin associated thrombocytopenia (HAT) type II patients with thrombocytopenia or thromboembolic complications and for the prophylaxis of thrombocytopenia or new thromboembolic events in patients with a history of HAT type II and a need for effective antithrombotic measures (eg during open heart surgery)

Drug: linezolid (ZYVOX)
Sponsor: Pharmacia Australia Pty Ltd
Date of designation: 9 January 2003
Indication: for the treatment of children, from birth to less than 18 years, with suspected or proven infections due to organisms resistant to multiple classes of antibiotics, including methicillin resistant Staphylococcus species and vancomycin resistant Enterococcus species

Drug: melphalan (ALKERAN INJECTION)
Sponsor: Glaxo Wellcome
Date of designation: 22 March 1999
Indication: for the treatment of multiple myeloma in patients for whom oral therapy is inappropriate Registered in Australia: 4 September 2001

Drug: mepolizumab (BOSATRIA)
Sponsor: GlaxoSmithKline Australia Pty Ltd
Date of designation: 17 March 2008
Indication: for the treatment of patients with hypereosinophilic syndrome
Dose: powder for injection

Drug: miglustat (ZAVESCA)
Sponsor: Actelion Pharmaceuticals Australia Pty Ltd
Date of designation: 11 July 2005
Indication: for the treatment of mild to moderate type 1 Gaucher disease
Dose: capsules
Registered in Australia: 2 March 2007

Drug: miglustat (ZAVESCA)
Sponsor: Actelion Pharmaceuticals Australia Pty Ltd
Date of designation: 8 July 2008
Indication: for the treatment of Niemann-Pick disease Type C
Dose: capsules

Drug: m-Iodobenzyl [123I] guanidine injection (MIBGen)
Sponsor: Australian Nuclear Science and Technology Organisation (ANSTO)
Date of designation: 15 August 2002
Registered in Australia: 26 May 2004
Indication: for:
-- Diagnostic scintigraphic localisation of tumours originating in tissue that embryologically stems from the neural crest. These are neuroblastomas, pheochromocytomas, paragangliomas, chemodectomas and ganglioneuromas
-- Detection, staging and follow-up on therapy of neuroblastomas
-- For the diagnosis of neural crest tumours, including those arising from the adrenal medulla, by evaluation of the uptake of iobenguane

Drug: multiBic
Sponsor: Fresenius Medical Care Australia Pty Ltd
Date of designation: 16 August 2004
Indication: for the treatment of acute renal failure in intensive care units

Drug: nandrolone decanoate (DECA-DURABOLIN 100)
Sponsor: Organon
Date of designation: 30 December 1999
Indication: for patients who have proven HIV infection who have lost at least 5% of their usual body weight

Drug: nevirapine (VIRAMUNE)
Sponsor: Boehringer Ingelheim Pty Limited
Date of designation: 11 March 2003
Indication: for the prevention of mother to child transmission of HIV-1 in pregnant women who are not taking antiretroviral therapy at time of labour

Drug: nilotinib (TASIGNA)
Sponsor: Novartis Pharmaceuticals Australia Pty Ltd
Date of designation: 9 June 2006
Indication: for treatment of patients with chronic myeloid leukaemia
Dose: 200mg capsules blister packs
Registered in Australia: 14 January 2008

Drug: nitazoxanide (CRYPTAZ)
Sponsor: Tri-Med Distributors
Date of designation: 13 October 1999
Indication: for the treatment of patients with AIDS who are positive for a stool test for cryptosporidiosis

Drug: nitric oxide (INOmax)
Sponsor: Delpharm Consultants Pty Limited
Date of designation: 11 May 2006
Indication: for the treatment of Persistent Pulmonary Hypertension (PPH) in neonates
Dose: 800 ppm medicinal gas for inhalation cylinder
Registered in Australia: 16 November 2007

Drug: normal immunoglobulin (INTRAGLOBIN F)
Sponsor: Medical Dynamics Australia (MDA) Pharma
Date of designation: 27 February 2001
Registered in Australia: 11 April 2001
Indication: for the treatment of:
-- Primary Immune Deficiency Syndromes
-- Kawasaki disease
-- Paediatric HIV infection / Congenital AIDS
-- Allogeneic Bone Marrow Transplantation
-- Idiopathic Thrombocytopaenic Purpura

Drug: NOVOSEVEN brand of eptacog alfa (activated) bhk
Sponsor: Novo Nordisk Pharmaceuticals Pty Ltd
Date of designation: 30 January 2007
Indication: for the treatment of postpartum haemorrhage in patients unresponsive to standard obstetrical management, oxytocic drugs and standard blood component therapy prior to major invasive therapy
Dose: powder for injection

Drug: NSC-631570 (UKRAIN)
Sponsor: Mrs Aleksandra Harasemcuk
Date of designation: 8 June 2004
Indication: for the treatment of pancreatic cancer

Drug: oblimersen (GENASENSE)
Sponsor: ICON Clinical Research
Date of designation: 10 January 2008
Indication: for the treatment of patients with stage IV malignant melanoma
Dose: solution for infusion

Drug: oblimersen (GENASENSE)
Sponsor: Kendle R and D Pty Ltd
Date of designation: 24 November 2006
Indication: for the treatment of patients with stage IV malignant melanoma

Drug: oblimersen (GENASENSE)
Sponsor: Aventis Pharma Pty Ltd
Date of designation: 25 September 2003
Indication: for the treatment of patients with stage IV malignant melanoma

Drug: octocog alfa rch (ADVATE) intravenous injection
Sponsor: Baxter Healthcare Pty Ltd
Date of designation: 19 February 2007
Indication: is indicated for use in haemophilia A for prevention and control of haemorrhagic episodes.
Registered in Australia: 5 December 2007

Drug: octocog alfa rch (ADVATE) powder for injection vials with diluent
Sponsor: Baxter Healthcare Pty Ltd
Date of designation: 10 March 2004
Indication: is indicated for use in haemophilia A for prevention and control of haemorrhagic episodes.
Registered in Australia: 25 February 2005

Drug: oprelvekin (IL-11) (NEUMEGA)
Sponsor: Wyeth
Date of designation: 07 April 1999
Indication: for the prevention of severe chemotherapy-induced thrombocytopenia (SCIT) in patients with non-myeloid malignancies who are judged by their physician to be at risk of severe thrombocytopenia

Drug: PEDITRACE and ADDAMEL N brands of trace element solutions for intravenous injection
Sponsor: Fresenius Medical Care Australia Pty Ltd
Date of designation: 8 November 2001
Indication: for the supply of basal trace element requirements in infants/children and adults, respectively

Drug: pegaspargase (PEGASPAR)
Sponsor: Rhone-Poulenc Rorer
Date of designation: 20 January 1999
Indication: for the treatment of patients with acute lymphoblastic leukaemia who require L-asparaginase in their treatment regimen, but have developed hypersensitivity to the native forms of L-asparaginase

Drug: pegvisomant {rbe} (SOMAVERT)
Sponsor: Pharmacia Australia Pty Ltd.
Date of designation: 9 October 2003
Indication: for the treatment of patients with acromegaly
Dose: powder for injection with diluent
Registered in Australia: 7 December 2005

Drug: pegylated liposomal doxorubicin hydrochloride (CAELYX)
Sponsor: Schering-Plough
Date of designation: 28 February 2000
Indication: for the treatment of advanced ovarian cancer in women who have failed first-line platinum-based chemotherapy
Registered in Australia: 21 September 2001

Drug: pegylated liposomal doxorubicin (Caelyx)
Sponsor: Schering-Plough Pty Ltd
Date of designation: 5 July 2007
Indication: for use, in combination, for the treatment of progressive multiple myeloma in patients who have received at least one prior therapy and who have undergone or are unsuitable for bone marrow transplantation.
Dose: injection vial 20 ml/10mL and 50mg/25mL

Drug: peritoneal dialysis solution (GAMBROSOL BIO)
Sponsor: Gambro

Date of designation: 23 December 1999 Indication: for the treatment of patients receiving peritoneal dialysis who may need to change from peritoneal dialysis because of decreased ultrafiltration or infusion pain

Drug: peritoneal dialysis solution with 1.1% amino acids (NUTRINEAL-PD4)
Sponsor: Baxter Health Care Pty Ltd
Date of designation: 4 March 1999
Indication: for the treatment of protein malnutrition in patients with End Stage Renal Disease (ESRD) who are maintained by peritoneal dialysis

Drug: PHYSIONEAL 40 brand of peritoneal dialysis solutions containing 13.6mg/mL, 22.7mg/mL or 38.7mg/mL glucose
Sponsor: Baxter Healthcare Pty Ltd
Date of designation: 8 May 2002
Indication: for peritoneal dialysis
Registered in Australia: 11 May 2005

Drug: piracetam (NOOTROPIL)
Sponsor: UCB Pharma
Date of designation: 4 May 2000
Indication: as "add-on" treatment of cortical myoclonus

Drug: Poractant Alfa (Curosurf)
Sponsor: Douglas Pharmaceuticals Australia Ltd
Date of designation: 11 April 2003
Indication: for the treatment of Respiratory Distress Syndrome (RDS) in preterm infants and for prophylactic use in preterm infants at risk for RDS.
Registered in Australia: 13 January 2005

Drug: pralidoxime chloride
Sponsor: Ophthalmic Laboratories Pty Ltd (T/A Pharmalab)
Date of designation: 3 May 2006
Indication: for the treatment of poisoning due to those pesticides and chemicals of the organophosphate class which have anticholinesterase activity and the control of overdosage of the anticholinesterase drugs used in the treatment of myasthenia gravis

Drug: rabies immunoglobulin Pasteurised (IMOGAM RAGE)
Sponsor: CSL
Date of designation: 16 October 1998
Indication: to treat subjects who are thought to have been exposed to rabies virus, especially cases of major exposure, in accordance with WHO recommendations
Registered in Australia: 24 February 2000

Drug: ranpirnase (Onconase)
Sponsor: Delpharm Consultants Pty Ltd
Date of designation: 22 March 2005
Indication: for the treatment of malignant mesothelioma

Drug: rasburicase (FASTURTEC)
Sponsor: Sanofi-Synthelabo Aust Pty Ltd
Date of designation: 23 March 2001
Indication: for the treatment and prophylaxis of acute hyperuricaemia in patients with haematological malignancy at risk of a rapid tumour lysis
Registered in Australia: 15 July 2002

Drug: recombinant factor IX (BENEFIX)
Sponsor: Wyeth
Date of designation: 16 October 1998
Indication: for the control and prevention of haemorrhagic episodes and for routine and surgical prophylaxis in patients with haemophilia B (congenital factor IX deficiency or Christmas disease)
Registered in Australia: 12 October 2000

Drug: recombinant factor VIII (Advate)
Sponsor: Baxter Healthcare Pty Limited
Date of designation: 10 March 2003
Indication: for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)

Drug: recombinant factor VIII(rDNA)(cho) (REFACTO)
Sponsor: Wyeth
Date of designation: 18 January 1999
Indication: for the control and prevention of haemorrhagic episodes and for routine and surgical prophylaxis in patients with haemophilia A (congenital factor VIII deficiency or classical haemophilia)
Registered in Australia: 4 January 2001

Drug: Recombinant Human Iduronate-2-Sulfatase (IDURSULFASE)
Sponsor: Genzyme Australia Pty Ltd
Date of designation: 18 January 2006
Indication: for long-term treatment of patients with Hunter syndrome

Drug: retroviral γc cDNA-containing vector (ImmuGene)
Sponsor: AVAX Australia Pty Ltd
Date of designation: 21 August 2002
Indication: the treatment of severe combined immunodeficiency X1 (SCID X1)
Status: The orphan drug designation for this product was withdrawn on 2 October 2002.

Drug: riluzole (RILUTEK)
Sponsor: Aventis Pharma
Date of designation: 3 May 2001
Indication: for the treatment of amyotrophic lateral sclerosis (ALS)

Drug: sapropterin dihydrochloride
Sponsor: Serono Pty Ltd
Date of designation: 30 August 2006
Indication: for treatment of patients with hyperphenylalaninemia
Dose: oral tablet

Drug: Sirolimus
Sponsor: Wyeth Australia Pty Limited
Date of designation: 26 March 2007
Indication: for the prevention of coronary allograft vasculopathy
Dose: tablets 1 mg, 2 mg and 5 mg and oral solution (1 mg/mL, 60 mL)

Drug: somatropin (HUMATROPE PENFIL)
Sponsor: Aza Research
Date of designation: 10 June 1999
Indication: for the treatment of growth retardation in pre-pubertal children with chronic renal insufficiency
Registered in Australia: 1 March 2001

Drug: somatropin (rbe) (GENOTROPIN)
Sponsor: Pharmacia-Upjohn
Date of designation: 8 May 2000
Indication: for improvement of body composition and treatment of short stature in patients with Prader-Wili syndrome
Registered in Australia: 11 August 2003

Drug: somatropin (rbe) recombinant human growth hormone (GENOTROPIN)
Sponsor: Pharmacia Australia Pty Limited
Date of designation: 28 November 2001
Indication: for treatment of growth disturbance in pre-pubertal children with chronic renal insufficiency
Registered in Australia: 13 June 2003

Drug: somatropin (rbe) recombinant human growth hormone (GENOTROPIN)
Sponsor: Pharmacia Australia Pty Limited
Date of designation: 2 November 2001
Indication: for treatment of growth failure children born small for gestational age.

Drug: somatropin (rbe) recombinant human growth hormone (NORDITROPIN, NORDITROPIN PENSET 12 and 14, NORDITROPIN SIMPLEXX)
Sponsor: Novo Nordisk Pharmaceuticals P/L
Date of designation: 11 March 2002
Indication: for treatment of severe growth failure due to intrauterine growth retardation (i.e. children born small for gestational age without spontaneous catch up growth by 2 years of age and height below -3SD at start of treatment)
Registered in Australia: 20 January 2004

Drug: somatropin (rbe) recombinant human growth hormone (NORDITROPIN, NORDITROPIN SIMPLEXX)
Sponsor: Novo Nordisk Pharmaceuticals P/L
Date of designation: 22 May 2002
Indication: for treatment of severe growth failure due to intrauterine growth retardation (i.e. children born small for gestational age without spontaneous catch up growth by 2 years of age).

Drug: Stay Safe Balance and Sleep Safe Balance range of sterile peritoneal dialysis solutions
Sponsor: Fresenius Medical Care Australia Pty Ltd
Date of designation: 21 August 2002
Indication: for the management of end stage renal disease and acute renal failure
Registered in Australia: 13 June 2003

Drug: tacrolimus (PROGRAF)
Sponsor: Janssen-Cilag
Date of designation: 22 October 2003
Indication: for use as an immunosuppressant in lung transplantation

Drug: tacrolimus (PROGRAF)
Sponsor: Janssen-Cilag Pty Ltd
Date of designation: 6 September 2006
Indication: for use as an immunosuppressant in heart transplantation
Dose: 0.5, 1 and 5 mg cabsules and a 5 mg/mL concentrate for intravenous infusion

Drug: Temozolomide (Temodal)
Sponsor: Schering-Plough Pty Limited
Date of designation: 24 July 2003
Indication: for:
-- treatment of recurrence of anaplastic astrocytoma and glioblastoma multiforme in children, following standard therapy
-- treatment of metastatic malignant melanoma in children

Drug: Temozolomide (TEMODAL)
Sponsor: Schering-Plough Pty Limited
Date of designation: 21 September 2004
Indication: for the treatment of patients with newly diagnosed anaplastic astrocytoma and gioblastoma multiforme.

Drug: temsirolimus (TORISEL)
Sponsor: Wyeth Australia Pty Limited
Date of designation: 17 March 2008
Indication: for the treatment of patients with mantle cell lymphoma
Dose: concentrate for injection

Drug: Terlipressin
Sponsor: Ferring Pharmaceutical Pty Ltd
Date of designation: 21 January 2004
Indication: for the treatment of bleeding oesophageal varices and for the treatment of the hepatorenal syndrome

Drug: thalidomide (THALOMID)
Sponsor:Pharmion Pty Ltd
Date of designation: 13 March 2002
Indication: for the treatment of erythema nodosum leprosum or Type II lepra reactions; and treatment of relapsing or refractory multiple myeloma.
Registered in Australia: 3 October 2003

Drug: Thymoglobuline (rabbit anti-human thymcoyte immunoglobulin)
Sponsor: Genzyme Australasia Pty Ltd
Date of designation: 22 February 2007
Dose: powder for injection
Indication:
-- Prophylaxis and treatment of graft rejection in renal transplantation,
-- Prophylaxis of graft rejection in heart transplantation and;
-- Treatment of aplastic anaemia

Drug: thyrotropin alfa (THYROGEN)
Sponsor: Genzyme Aust
Date of designation: 7 October 1999
Indication: for use with radioactive iodine imaging and serum thyroglobulin (Tg) testing, undertaken for the detection of thyroid remnants and well-differentiated thyroid cancer in post-thyroidectomy patients maintained on hormone suppression therapy and at risk of recurrence
Registered in Australia: 17 June 2004

Drug: thyroptropin alfa-rch powder for injection
Sponsor: Genzyme Australasia Pty Ltd
Date of designation: 24 January 2005
Indication: for therapeutic use in post-thyroidectomy patients maintained on hormone suppression therapy in the ablation of thyroid remnant tissue in combination with radioactive iodine.

Drug: tipifarnib (Zarnestra)
Sponsor: Janssen-Cilag Pty Ltd
Date of designation: 17 November 2004
Indication: for the treatment of acute myeloid leukaemia

Drug: tobramycin solution for inhalation (TOBI)
Sponsor: Pathogenesis Corporation
Date of designation: 29 September 1998
Indication: for the treatment of cystic fibrosis patients infected with Pseudomonas aeruginosa
Registered in Australia: 15 February 2000

Drug: tobramycin sulfate
Sponsor: Ophthalmic Laboratories Pty Limited (T/A Pharmalab)
Date of designation: 28 April 2006
Indication: for treatment of patients with cystic fibrosis infected with Pseudomonas species

Drug: tobramycin sulfate solution for inhalation
Sponsor: Ophthalmic Laboratories Pty Limited
Date of designation: 18 June 2004
Indication: for the treatment of patients with cystic fibrosis infected with Pseudomonas species

Drug: Tracleer (BOSENTAN)
Sponsor: Actilion Pharmaceuticals Pty Ltd
Date of designation: 4 October 2005
Indication: for the treatment of Primary Pulmonary Arterial Hypertension (WHO Class PH I) in patients with WHO Functional Class III or IV symptoms

Drug: treprostinil (REMODULIN) (incorrectly gazetted as epoprostenol)
Sponsor: Orphan Australia Pty Ltd
Date of designation: 29 May 2001
Indication: for the treatment of pulmonary arterial hypertension
Registered in Australia: 20 May 2004

Drug: tumour necrosis factor alpha 1-a (BEROMUN)
Sponsor: Boehringer Ingelheim
Date of designation: 21 October 1998
Indication: as an adjunct to surgery for subsequent removal of the tumour (soft tissue sarcoma) so as to delay amputation, or in the palliative situation, for irresectable soft-tissue sarcoma of the limbs, used in conjunction with melphalan in hyperthermic isolated limb perfusion
Registered in Australia: 15 March 2001

Drug: zanolimumab
Sponsor: Merck Serono Australia Pty Ltd
Date of designation: 10 July 2007
Indication: for the treatment of patients with cutaneous T cell lymphoma
Dose: solution for infusion at 20 mg/mL

Drug: Zemaira Brand of Alpha Proteinase Inhibitor (Human)
Sponsor: CSL Ltd
Date of designation: 8 August 2007
Indication: for the treatment of chronic intravenous augmentation and maintenance therapy in individuals with alpha1-proteinase inhibitor deficiency and clinical evidence of emphysema
Dose: Powder for Injection

Department of Health and Ageing - Therapeutic Goods Administration
http://www.tga.gov.au