NEW YORK, Jan. 3, 2013 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:
Orphan Drugs: Market Environment Products and Companieshttp://www.reportlinker.com/p01075850/Orphan-Drugs-Market-Environment-Products-and-Companies .html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Drug_and_Medication
For the pharma and diagnostic industries, rare diseases can provide significant commercial success. Examples of blockbuster orphan drugs include Novartis' leukaemia drug, Glivec/Gleevec (imatinib), with global sales of US$4,695 million in 2011; and Actelion's Tracleer (bosentan), the current gold standard in pulmonary arterial hypertension with sales of CHF1,522 million (US$1,598 million) in 2011.
The combination of government incentives to develop drugs for rare diseases and the promise of commercial opportunity will continue to fuel the industry's interest in orphan drugs; and provide hope to patients with debilitating conditions and high unmet medical needs. In addition, the fast track approval system allows companies to enter the market faster and more cost-effectively with the potential to extend indications.
Significant unmet clinical need
While orphan diseases are rare, patients with rare diseases are numerous. There are up to 7,000 identifiable rare diseases, affecting an estimated 622 million people around the world. There is a clear need to provide rare disease patients with the same quality of care as other patients, and governments have provided incentives to companies developing orphan drugs to address a significant public health need. As effective diagnostics play a greater role in accurately diagnosing orphan diseases, the addressable patient population will expand.
Companion diagnostics: a game changer?One of the biggest challenges for rare disease patients is obtaining the correct diagnosis, which can be extremely difficult and can take years, or even decades. An increasing number of companies developing drugs for rare diseases are working with diagnostic companies, or their own diagnostic divisions, to establish biomarkers and develop companion diagnostics for patient selection. Companion diagnostics can assist with identifying patients for clinical trials of a novel drug, and may be approved simultaneously as part of a conditional drug approval.
A comprehensive overview of orphan drugs and their strategic importance is provided in this valuable new report
• Market conditions – provides an analysis of the orphan drug market, detailing the drivers for recent growth, prevalence and scientific advances
• Regulatory landscape – provides an in-depth analysis of current regulations in major markets (US, EU, Australia and Japan) along with pricing and reimbursement information and orphan drug approvals
• Company activity – summarises leading and emerging companies and their established orphan drug products and pipelines
• Targeted diseases – summarises diseases being targeted such as Duchenne muscular dystrophy, amyotrophic lateral sclerosis, leukaemia and pulmonary hypertension
• Research – innovative areas of research such as antisense therapy, gene therapy, and cellular therapy in which orphan drugs are playing a key role.
FOREWORD 1 EXECUTIVE SUMMARY 3 MARKET ENVIRONMENT 5Rare Diseases 5Definitions and Prevalence 5Developing Drugs for Rare Diseases 6Scientific Advances 6Government Support & Regulation 7Legislation in the US7Clinical Data8Accelerating Approval 8Post-Marketing Approval Commitments 8Applying for Orphan Drug Designation 9Criticism of Orphan Drug Legislation in the US 9EU Orphan Drug Regulations 10Australian Orphan Drug Regulations 10Japanese Orphan Drug Regulations 11Harmonisation of US and EU regulation and advice 11Incentives for Orphan Drug Development 11Publicly Funded Projects 12Patient Advocacy Groups 13Orphan Drug Designations & Approvals 14Designations & Approvals in the US 14Designations and Approvals in the EU 20
CHALLENGES AND OPPORTUNITIES IN THE ORPHAN DRUGS MARKET 22
Challenges in the Discovery and Development of Drugs for Rare Diseases 22
Preclinical Development 22
Clinical Development 22
Innovation in Clinical Trial Design 23
Finding Patients: Organisations and Registries 24
Access to Investigational Products 24
Rare Disease Diagnosis and Diagnostics 25
Biomarkers and Companion Diagnostics 25
Targeted Diseases for Orphan Drug Development 26
Alpha-1 Antitrypsin Deficiency 26
Amyotrophic Lateral Sclerosis 26
Duchenne Muscular Dystrophy 27
Gastrointestinal Stromal Tumours (GIST) 28
Glioblastoma 28
Leukaemia 28
Lysosomal Storage Diseases 29
Pulmonary Hypertension 30
Innovative Technology Platforms 31
Antisense Therapy 31
Gene Therapy 32
Stem Cell Therapy 34
Therapeutic Vaccines 35
Pricing and Reimbursement 37
Pricing37
Reimbursement in the US 37
Reimbursement in the EU 38
COMPANY ACTIVITY 39Leading Pharma, Biotech & Specialty Companies 39AbbVie (Abbott) 39Actelion39Rare Diseases R&D40Bayer HealthCare 41Rare Diseases R&D 41Gastrointestinal Stromal Tumours41Pulmonary Hypertension 41Biogen Idec 42Rare Diseases R&D 42Amyotrophic Lateral Sclerosis 42Haemophilia 42Spinal Muscular Atrophy 43BioMarin 43Rare Diseases R&D 44Celgene 45Rare Diseases R&D 46Gilead Sciences 46Rare Disease R&D 46GlaxoSmithKline47Rare Diseases R&D48ADA-SCID; Fondazione Telethon, Fondazione San Raffaele del Monte Tabor 48Duchenne muscular dystrophy; Prosensa 48Lysosomal Storage Diseases; Collaborations with Angiochem and Amicus 48RNA Therapeutics for Rare and Infectious Diseases; Isis Pharmaceuticals 49Novartis 50Rare Diseases R&D 52Acute Myeloid Leukaemia (AML) 52Cushing Disease 52Pfizer 53Rare Diseases R&D 54Gaucher Disease; Protalix BioTherapeutics 54TTR Amyloid Polyneuropathy 55Vaso-Occlusive Crisis Associated With Sickle Cell Disease; GlycoMimetics 55Sanofi 55Rare Diseases R&D 56Gaucher disease 56Usher syndrome; Oxford BioMedica 56Shire 57Rare Diseases R&D 58Sigma-Tau 58Rare Diseases R&D 59Swedish Orphan Biovitrum 60Rare Diseases R&D 60ViroPharma 60Rare Diseases R&D 61Emerging Biotech Companies 62Acorda Therapeutics 62Alexion Pharmaceuticals 62Amicus Therapeutics 63Diffusion Pharmaceuticals 64Erytech Pharma 64Incyte 64InterMune 65Protalix Biotherapeutics 65Santhera Pharmaceuticals 65Seattle Genetics 66Vertex 66
SOURCES 68 List of TablesCompanies Receiving FDA Orphan Drug Designations, 2011-Q3 2012 18
Examples of Antisense Therapies in Development for Rare Diseases 32
Examples of Gene Therapies in Development for Rare Diseases 34
Examples of Stem Cell Therapies in Development for Rare Diseases 35
Example of Therapeutic Vaccines in Development for Rare Diseases 36
The High Cost of Orphan Drugs 37
Actelion: Approved Orphan Drugs 40
Bayer HealthCare: Key Orphan Drugs 41
BioMarin: Approved Orphan Drugs 44
Celgene: Approved Orphan Drugs 45
Gilead Sciences: Key Orphan Drugs 46
GSK: Key Orphan Drugs 47
Novartis: Key Orphan Drugs 51
Pfizer: Key Orphan Drugs 54
Genzyme (Sanofi): Key Orphan Drugs 56
Shire: Key Orphan Drugs 58
Sigma-Tau: Key Orphan Drugs 59
ViroPharma: Approved Orphan Drugs 61
List of Figures EuropeTotal Orphan Drug Designations and Approvals, 2000-2012 15
Big Pharma Orphan Drug Designations & Approvals, 2000-2012 16
Biotech/Specialty Orphan Drug Designations & Approvals, 2000-2012 17
COMP Opinions on Designation by Therapy Area, 2000-2010 20
Orphan Medicines Authorised in the EU, by Therapy Area 21
Attributes of Pivotal Clinical Trials for Orphan Drugs Approved by the FDA between 2007 and 2009 23
To order this report:Drug_and_Medication Industry: Orphan Drugs: Market Environment Products and Companies
Nicolas Bombourg
Reportlinker
Email: [email protected]
US: (805)652-2626
Intl: +1 805-652-2626
SOURCE Reportlinker
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