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M&A in pharma

Explores the merits of mega-deal in contrast to a more measured portfolio growth strategy

The successful introduction of orphan products in Europe remains problematic for the developers, although they can seek to alleviate some of the issues by carefully developing a launch strategy.

Traditionally the development and marketing of orphan drugs has not been an area of interest for major pharmaceutical companies, reflecting the view that orphan drugs were specialty products with limited market potential thus offering a poor fit for companies based upon the blockbuster model. However, a number of major companies have reassessed their strategy with respect to orphan drugs and five of the top ten have made explicit commitments to the development of such drugs. This has seen a number of changes in the orphan drug landscape and further developments are likely, especially from some of those not yet active in the field.

The 302 drugs that are approved for the treatment of 373 orphan indications only over a fraction of the approximately 7,000 rare diseases categorised as rare by the National Institute for Health (US).

Orphan Drug Markets in Europe - Key developments and the strategies of major pharmaceutical companies is a unique report from URCH Publishing that offers the reader a comprehensive overview of the state of the orphan medicines market in Europe. The carefully authored study provides expert insight into the market to date, regulatory challenges and future opportunities concerning orphan drugs. Particular attention is paid to the differences in regulation and market between Europe Japan and the US.

• Within Europe sales of approved orphan products account for approximately $6bilion.
• Novartis’ Glivec (imatinib mesylate) sales are over $1.5 billion.
• Since January 2000 there has been a steady increase in applications for orphan designation with the Committee for Orphan Medicinal Products (COMP) averaging ten positive recommendations per month.
• Five companies generated major revenues from the marketing of orphan drug products in 2010, four primarily by supplying biological products while just Actelion supplied small molecule therapeutics.
• Big pharma has started to take orphan drugs seriously and a number have formed dedicated business units to explore opportunities.
• Alexion’s Soliris shows that small patient populations are not incompatible with commercial success.
• Although central approval covers 30 European countries, it does not necessarily provide for national availability as each national authority has to agree labelling and reimbursement.
• The increasing collaboration between the FDA and EMA has led to the adoption of a common application form should facilitate developers’ efforts to exploit orphan drugs on a more international basis.

• Obtain a complete understanding of the orphan drug regulations in Europe.
• Gain insight into the key companies operating in this sector.
• Understand the differences between the Europe and US markets.
• Evaluate which rare diseases offer the greatest commercial opportunity.
• Assess the performance of specialist orphan drug companies.
• Review how big pharma is starting to enter this market segment.
• Find the new areas of pharmaceutical market growth and key opportunities for delivering successful sales growth over the next five years.
• Support internal planning and decision-making with an external perspective founded on detailed analysis and transparent market forecasts.

This report is designed for anyone who needs a comprehensive overview of orphan medicines in Europe, including:

• Strategy managers and directors in large pharmaceutical companies
• Biotechnology companies with niche product pipelines
• Investors looking for opportunities
• US-based companies with an interest in expanding to Europe.

Companies & Organisations Mentioned in the Report Include

Actelion, Alexion, Alnara Pharmaceuticals, Amgen, Amicus Therapeutics, AmpliPhi Biosciences, Aventis, Bayer, BiogenIdec, Biovitrum, bluebird bio, Bristol-Myers Squibb, Celgene, Cephalon (Teva), COMP, Del Mar Pharmaceuticals, Edimer Pharmaceuticals, Eli Lilly, EURODIS, EUSA Pharma, FDA, Généthon, GlaxoSmithKline, Hyperion Therapeutics, Intermune, Isis Pharmaceuticals, JCR Pharmaceutical, Lotus Tissue Repair, Merck, National Institute for Health, NORD, Novartis, Novo Nordisk, Orfagen, Orphan Europe (Recordati), Orphan Therapeutics, Pfizer, Pharmamar, Prosena, Protalix Biotherapeutics, Roche, Sanofi, Santhera Pharmaceuticals,  
Shire, Sigma-Tau, SpePharm, Swedish Orphan Biovitrum, Sygnis Bioscience, Synageva BioPharma, Takeda, Teva, Trophos, United Therapeutics, Vivendy Therapeutics, Voisin Consulting, Zacharon Pharmaceuticals, Zymenex

Orphan medicines mentioned in this report include
Adcirca – Aldurazyme – Avonex – Campath -  Cerezyme - ClolarCopaxone - Elaprase - Epogen - Esbriet – Evoltra - ExJade – Fabrazyme - Gleevec / Glivec – Kogenate - Mozobil - Myozyme (and Lumizyme) – Neupogen – Nexavar – Novoseven - Provigil – Pulmozyme - Rebif – Revatio – Revlimid – Rituxan – Sensipar – Soliris - Somavert  - Sprycel – Temodar – Thyrogen – Tracleer – Velcade – Veletri – Ventavis – Xagrid – Yondelis - Zavesca

Outlook

Chapter 8: Outlook for orphan drugs
Targeting rare diseases more popular
High profile
Commercial opportunities
Potential pitfalls
Competition issues
European specific issues
Conclusions
Bibliography
Glossary

List of Figures

Figure 2.1 Global Time Line of Orphan Drug Legislation
Figure 2.2 FDA definitions of similarity
Figure 2.1 EMA key relationships relating to the designation and approval of orphan drugs
Figure 2.2 EU regulations pertaining to the orphan drug regulation (Regulation 141/2000)
Figure 2.3 The process of orphan drug designations by the COMP
Figure 2.4 The number of applications to the COMP for orphan drug designation, and their outcomes, to July 2011
Figure 3.1 Relative prevalence of 27 lysosomal storage disorders in Australia
Figure 4.1 Orphan drug authorizations in Europe by year (to July 2011)
Figure 4.2 Orphan drug approvals by marketing company
Figure 4.5 Approved orphan drugs by therapeutic area
Figure 6.4 Potential return from an orphan therapeutic in Europe for a prevalent indication
Figure 4.7 Applications for Orphan Drug Designation in Europe
Figure 4.8 Unsuccessful Applications for Orphan Drug Designation in Europe
Figure 4.9 COMP opinions by patient population
Figure 4.10 Geographic availability of 21 orphan drugs in Europe in 2007
Figure 4.11 Confirmed availability, and unavailability, of 60 orphan drugs in selected EU countries in 2007
Figure 4.12 Average price and reimbursement delays for generic products by country
Figure 4.13 Intermune's proposed EU launch strategy for Esbriet
Figure 5.14 European orphan designations by disease prevalence to 2010
Figure 5.15 New Drug Approvals by the FDA 2004-2010
Figure 7.1 The changing orphan drug landscape and major pharma
Figure 8.1 Competitive developments for treating Gaucher disease
Figure 8.2 Availability of 21 approved orphan drugs in Europe in 2007
Figure 8.3 Intermune's European launch strategy for Esbriet

List of Tables

Dr Peter Norman is a pharmaceutical consultant and analyst based in Burnham Beeches, near Windsor, England, with specialist knowledge of the respiratory disease and inflammation markets. He has written and presented widely on various aspects of respiratory disease, on the analysis of diverse therapeutic segments, generic opportunities and on Orphan Drugs. Dr. Norman has extensive experience of the pharmaceutical industry in both R&D and competitive intelligence. His publications include many reviews and management reports, sixteen original scientific papers and eleven patents. Dr. Norman holds science degrees from Cambridge University and Brunel University plus a M.B.A. degree from the Open University Business School.