Orphan Drugs: Market Environment Products and Companies

  • December 2012
  • -
  • Espicom Business Intelligence
  • -
  • 68 pages

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.

Table Of Contents

Orphan Drugs: Market Environment Products and Companies

Rare Diseases 5
Definitions and Prevalence 5
Developing Drugs for Rare Diseases 6
Scientific Advances 6
Government Support and Regulation 7
Legislation in the US 7
Clinical Data 8
Accelerating Approval 8
Post-Marketing Approval Commitments 8
Applying for Orphan Drug Designation 9
Criticism of Orphan Drug Legislation in the US 9
EU Orphan Drug Regulations 10
Australian Orphan Drug Regulations 10
Japanese Orphan Drug Regulations 11
Harmonisation of US and EU regulation and advice 11
Incentives for Orphan Drug Development 11
Publicly Funded Projects 12
Patient Advocacy Groups 13
Orphan Drug Designations and Approvals 14
Designations and Approvals in the US 14
Designations and Approvals in the EU 20
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
Pricing 37
Reimbursement in the US 37
Reimbursement in the EU 38
Leading Pharma, Biotech and Specialty Companies 39
AbbVie (Abbott) 39
Actelion 39
Rare Diseases RandD 40
Bayer HealthCare 41
Rare Diseases RandD 41
Gastrointestinal Stromal Tumours 41
Pulmonary Hypertension 41
Biogen Idec 42
Rare Diseases RandD 42
Amyotrophic Lateral Sclerosis 42
Haemophilia 42
Spinal Muscular Atrophy 43
BioMarin 43
Rare Diseases RandD 44
Celgene 45
Rare Diseases RandD 46
Gilead Sciences 46
Rare Disease RandD 46
GlaxoSmithKline 47
Rare Diseases RandD 48
ADA-SCID; Fondazione Telethon, Fondazione San Raffaele del Monte Tabor 48
Duchenne muscular dystrophy; Prosensa 48
Lysosomal Storage Diseases; Collaborations with Angiochem and Amicus 48
RNA Therapeutics for Rare and Infectious Diseases; Isis Pharmaceuticals 49
Novartis 50
Rare Diseases RandD 52
Acute Myeloid Leukaemia (AML) 52
Cushing Disease 52
Pfizer 53
Rare Diseases RandD 54
Gaucher Disease; Protalix BioTherapeutics 54
TTR Amyloid Polyneuropathy 55
Vaso-Occlusive Crisis Associated With Sickle Cell Disease; GlycoMimetics 55
Sanofi 55
Rare Diseases RandD 56
Gaucher disease 56
Usher syndrome; Oxford BioMedica 56
Shire 57
Rare Diseases RandD 58
Sigma-Tau 58
Rare Diseases RandD 59
Swedish Orphan Biovitrum 60
Rare Diseases RandD 60
ViroPharma 60
Rare Diseases RandD 61
Emerging Biotech Companies 62
Acorda Therapeutics 62
Alexion Pharmaceuticals 62
Amicus Therapeutics 63
Diffusion Pharmaceuticals 64
Erytech Pharma 64
Incyte 64
InterMune 65
Protalix Biotherapeutics 65
Santhera Pharmaceuticals 65
Seattle Genetics 66
Vertex 66

List of Tables

Incentives for Orphan Drug Development in Selected Markets 11
Companies 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
Prevalence of Rare Diseases in Europe 6
Total Orphan Drug Designations and Approvals, 2000-2012 15
Big Pharma Orphan Drug Designations and Approvals, 2000-2012 16
Biotech/Specialty Orphan Drug Designations and 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

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