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Orphan Drug Markets in Europe: Key Developments and the Strategies of Major Pharmaceutical Companies Product Image

Orphan Drug Markets in Europe: Key Developments and the Strategies of Major Pharmaceutical Companies

  • Published: November 2011
  • Region: Europe
  • 66 Pages
  • Spectra Intelligence


  • Actelion
  • Biovitrum
  • Lotus Tissue Repair
  • Pharmamar
  • Swedish Orphan Biovitrum
  • MORE

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.

The first marketing authorisations for orphan drugs in Europe took place a decade ago. Since then there have been over 60 designated orphan products. These products now account for annual sales of more than $6 billion.

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 READ MORE >

Executive Summary

Chapter 1: An introduction to orphan drugs in Europe

Historical Overview

Chapter 2: Orphan Drug Legislation
Global progress
The United States


Other markets

South Korea
A comparison

Chapter 3: Rare Diseases and Orphan Drugs

Rare Diseases
Genetic disorders
Cystic fibrosis
Muscular dystrophy
Lysosomal storage disorders
Very Rare Diseases

Patient registries

Chapter 4: Progress of orphan drugs in Europe
Revenues from orphan drugs in Europe
What is similar?
Designated orphan drugs

Leading applicants
Success stories

Soliris (Alexion)
Enzyme replacement therapies
Glivec (Novartis)
Fragmented market issues

Chapter 5: Orphan drugs in the US
25 years of approvals
Major successes

Chapter 6: Specialist Companies
Major players

Established specialist players

Orphan Europe (Recordati)
Sigma-Tau SpA
Swedish Orphan Biovitrum
EUSA Pharma
Emerging speciality companies

Amicus Therapeutics
AmpliPhi Biosciences
bluebird bio
Del Mar Pharmaceuticals
Edimer Pharmaceuticals
Hyperion Therapeutics
Lotus Tissue Repair
Orphan Therapeutics
Santhera Pharmaceuticals
Sygnis Bioscience
Synageva BioPharma
Vivendy Therapeutics

Chapter 7: Major pharmaceutical companies' involvement in orphan drugs

Eli Lilly
Other companies

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

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
Table 2.1 Comparison of Orphan Drug Criteria in the US, the EU and Japan
Table 4.1 European status of therapies for pulmonary hypertension
Table 5.2 Selected commercially successful biological orphan products in the US
Table 5.3 Selected commercially successful small molecule orphan products in the US
Table 6.4 Genzyme's orphan drug portfolio and 2010 sales
Table 6.5 Genzyme's orphan drug pipeline
Table 6.6 Actelion's orphan drug portfolio and 2010 sales
Table 6.7 Actelion's orphan drug pipeline
Table 6.8 Shire's orphan drug portfolio and 2010 sales
Table 9.6 Shire's orphan drug pipeline
Table 6.10 BioMarin's orphan drug portfolio and 2010 sales
Table 6.11 BioMarin's orphan drug pipeline
Table 6.12 Recordati's orphan drug portfolio
Table 6.13 Recordati's orphan drug pipeline
Table 6.11 Sigma-Tau's orphan drug pipeline
Table 6.12 Swedish Orphan Biovitrum's orphan drug portfolio and 2010 revenues
Table 6.13 PharmaMar's orphan drug pipeline
Table 6.14 Orfagen's orphan drug pipeline
Table 6.15 Amicus' orphan drug pipeline
Table 6.16 Prosensa's orphan drug pipeline
Table 6.17 Santhera's orphan drug pipeline
Table 6.18 Synagis' orphan drug pipeline
Table 6.14 Synageva's orphan drug pipeline

Number of orphan medicines to grow in Europe as ‘Big Pharma’ takes an interest says new report from URCH Publishing

Large drug companies starting to seriously consider rare disease markets

The number of medicines designated and marketed with an orphan status will grow steadily in Europe says a new report. Orphan Drug Markets in Europe - Key
developments and the strategies of major pharmaceutical companies published by URCH Publishing, a business information provider, says that the commercial success of many orphans over the last ten years is boosting interest from Big Pharma as their blockbuster model becomes unsustainable. Already 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.

“Developing treatments for some of these diseases offers pharmaceutical companies the chance of improving their corporate images, which have become tarnished in recent years, as well as offering commercial opportunities that can help sustain companies’ revenues,” says Dr Peter Norman, the analyst and author of the report. “Recently we have seen GSK, Pfizer and Sanofi beef-up their rare disease research though acquisitions and collaborations,” notes Dr Norman.

However, the 60 page market report points out that the European orphan drug market presents more challenges to developers than does the smaller US market. Although central approval covers 30 different countries, it does not automatically provide for national availability. Each national authority has to agree labelling and reimbursement which not only leads to delays in products reaching the market but also in orphan products becoming available in most member states. Two surveys by EURODIS have suggested that only in a few countries, including France and Germany, are all (or nearly all) approved orphan products available. Thus any European launch strategy needs careful planning addressing reimbursement issues, potential patient numbers and distribution of patients within the EU. However, for the more prevalent rare diseases, a strategy focusing on early launch in the five major markets remains the preferred option as it is for non-orphan drugs.

In addition the increasing collaboration between the FDA and EMA departments that deal with orphan drugs has led to the adoption of a common application form should facilitate developers’ efforts to exploit orphan drugs on a more international basis. But, what may cause a greater problem in future, will be trying to match the rising cost of orphan drug development against increasing cost-containment in health services which may pose challenges in marketing some new orphan products.

Orphan Drug Markets in Europe - Key developments and the strategies of major pharmaceutical companies is available from URCH Publishing. The 30,000 word report will provide the reader with a first-rate review of orphan drugs in Europe since the introduction of legislation a decade ago. 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.

- Actelion
- Alexion
- Alnara Pharmaceuticals
- Amgen
- Amicus Therapeutics
- AmpliPhi Biosciences
- Aventis
- Bayer
- BiogenIdec
- Biovitrum
- bluebird bio
- Bristol-Myers Squibb
- Celgene
- Cephalon (Teva)
- Del Mar Pharmaceuticals
- Edimer Pharmaceuticals
- Eli Lilly
- EUSA Pharma
- Généthon
- GlaxoSmithKline
- Hyperion Therapeutics
- Intermune
- Isis Pharmaceuticals
- JCR Pharmaceutical
- Lotus Tissue Repair
- Merck
- National Institute for Health
- 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

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