Orphan Drugs....continued

Orphan drug development

Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokinetics and pharmacodynamics, dosing, stability, safety and efficacy. However, some statistical burdens are lessened in an effort to maintain development momentum. The government bodies encourage orphan drug development by provifing :

• Tax incentives.
• Enhanced patent protection and marketing rights.
• Clinical research financial subsidization.
• Creating a government-run enterprise to engage in research and development (i.e Crown corporation).

Some Orphan Drugs in the Market :

European Union

USA

Generic Name: Albendazole
Trade Name: Albenza
Indication: Treatment of hydatid disease (cystic echinococcosis due to E. granulosus larvae or alveolar echinococcosis due to E. multilocularis larvae).
Generic Name: Albendazole
Trade Name: Albenza
Indication: Treatment of neurocysticercosis due to Taenia solium as: 1) chemotherapy of parenchymal, subarachnoidal and racemose (cysts in spinal fluid) neurocysticercosis in symptomatic cases and 2) prophylaxis of epilepsy and other sequelae in asymptomatic neurocysticercosis.
Generic Name: Aldesleukin
Trade Name: Proleukin
Indication: Treatment of metastatic renal cell carcinoma.
Generic Name: Aldesleukin
Trade Name: Proleukin
Indication: Treatment of metastatic melanoma.
Generic Name: Alglucerase injection
Trade Name: Ceredase
Indication: For replacement therapy in patients with Gaucher's disease type I.
Generic Name: Alitretinoin
Trade Name: Panretin
Indication: Topical treatment of cutaneous lesions in patients with AIDS-related Kaposi's sarcoma.
Generic Name: Allopurinol sodium
Trade Name: Zyloprim for Injection
Indication: Management of patients with leukemia, lymphoma, and solid tumor malignancies who are receiving cancer therapy which causes elevations of serum and urinary uric acid levels and who cannot tolerate oral therapy.
Generic Name: Alpha1-proteinase inhibitor (human)
Trade Name: Prolastin
Indication: For replacement therapy in the alpha-1-proteinase inhibitor congenital deficiency state.
Generic Name: Altretamine
Trade Name: Hexalen
Indication: Treatment of advanced adenocarcinoma of the ovary.
Generic Name: Amifostine
Trade Name: Ethyol
Indication: For use as a chemoprotective agent for cisplatin in the treatment of advanced ovarian carcinoma.
Generic Name: Amifostine
Trade Name: Ethyol
Indication: Reduction of the incidence of moderate to severe xerostomia in patients undergoing post-operative radiation treatment for head and neck cancer.
Generic Name: Aminosalicylic acid
Trade Name: Paser Granules
Indication: Treatment of tuberculosis infections.
Generic Name: Amiodarone HCl
Trade Name: Cordarone
Indication: For the acute treatment and prophylaxis of life-threatening ventricular tachycardia or ventricular fibrillation.
Generic Name: Amphotericin B lipid complex
Trade Name: Abelcet
Indication: Treatment of invasive fungal infections.
Generic Name: Anagrelide
Trade Name: Agrylin
Indication: Treatment of essential thrombocythemia.
Generic Name: Antihemophilic factor (recombinant)
Trade Name: ReFacto
Indication: For the control and prevention of hemorrhagic episodes and for surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).
Generic Name: Antihemophilic factor (recombinant)
Trade Name: Kogenate
Indication: Prophylaxis and treatment of bleeding in individuals with hemophilia A or for prophylaxis when surgery is required in individuals with hemophilia A.
Generic Name: Antihemophilic factor/von Willebrand factor complex (human), dried, pasteurized
Trade Name: Humate-P
Indication: Treatment and prevention of bleeding in hemophilia A (classical hemophilia) in adult patients; and treatment of spontaneous and trauma-induced bleeding episodes in severe von Willebrand disease, and in mild and moderate von Willebrand disease where use of desmopressin is known or suspected to be inadequate in adult and pediatric patients.
Generic Name: Antithrombin III (human)
Trade Name: ATnativ
Indication: For the treatment of patients with hereditary antithrombin III deficiency in connection with surgical or obstetrical procedures or when they suffer from thromboembolism.
Generic Name: Antithrombin III (human)
Trade Name: Thrombate III
Indication: For replacement therapy in congenital deficiency of AT-III for prevention and treatment of thrombosis and pulmonary emboli.
Generic Name: Aprotinin
Trade Name: Trasylol
Indication: For prophylactic use to reduce perioperative blood loss and the homologous blood transfusion requirement in patients undergoing cardiopulmonary bypass surgery in the course of repeat coronary artery bypass graft surgery, and in selected cases of primary coronary artery bypass graft surgery where the risk of bleeding is especially high (impaired hemostasis) or where transfusion is unavailable or unacceptable.
Generic Name: Atovaquone
Trade Name: Mepron
Indication: Treatment of AIDS associated Pneumocystis Carinii Pneumonia.

AUSTRALIA

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: vorinostat (ZOLINZA)
Sponsor: Merck Sharp & Dohme (Australia) Pty Ltd
Date of designation: 21 October 2008
Indication: for the treatment of patients with advanced cutaneous T-cell lymphoma (CTCL) who have progressive, persistent or recurrent disease subsequent to prior systemic therapy.
Dose: capsules

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

Drug: zoledronic acid (ZOMETA)
Sponsor: Novartis Pharmaceuticals Australia Pty Ltd
Date of designation: 8 May 2009
Indication: For the treatment of paediatric patients with severe osteogenesis imperfecta (OI)
Dose: 4 mg powder for injection and 4 mg/5 mL concentrated injection

Orphan Drug Economy :

• The global orphan drugs market reached $58.7 billion in 2006, growing by 8% from $54.5 billion in 2005. It is expected to grow at a compounded annual growth rate (CAGR) of 7% to reach $81.8 billion by 2011.
• Regionally, U.S. revenues in the market grew to $32.5 billion accounting for 55% of the market in 2006. The U.S. market is expected to grow at a CAGR of 8% to reach $47.8 billion by 2011.
• Biologic drugs account for over 60% of the orphan drug market with sales of $35.3 billion in 2006, up from $30.2 billion in 2005. The market size of biologic orphan drugs is projected to grow at a 9% CAGR to reach $53.4 billion by 2011.

References:

1)The department of health and ageing therapeutic goods administration,Government of Australia.

2)The US FDA

3)The European Medicines Agency.

4)The BCC reasearch.