Orphan Drugs: Market Environment Products and Companies

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 Companies

http://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 5

Rare 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 39

Leading 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 Tables

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 Europe

Total 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]

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