Home > Back to Category > Report Information

Orphan Drug Markets in Europe

Urch Publishing
November 1, 2011
66 Pages - SKU: URC6724618

Abstract Table of Contents Search inside this report Related Reports

Countries covered: Europe

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 diseases categorised as rare by the National Institute for Health (US).

The products Glivec and Tracleer show, it is possible for orphan drugs to generate substantial returns in Europe clearly demonstrating the commercial viability of Europe as a market for orphan drugs.

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.

Some key findings from the report include:

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.
Reasons to Buy
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.

Who should read this unique report

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.

Executive Summary
Chapter 1: An introduction to orphan drugs in Europe: Introduction; Historical Overview
Chapter 2: Orphan Drug Legislation: Global progress; The United States; Europe; COMP; Japan; Other markets; Singapore; Australia; Taiwan; Switzerland; South Korea; Brazil; A comparison
Chapter 3: Rare Diseases and Orphan Drugs: Overview; Rare Diseases; Genetic disorders; Cystic fibrosis; Muscular dystrophy; Thalassaemia; Lysosomal storage disorders; Cancer; Very Rare Diseases; Repurposing; Patient registries
Chapter 4: Progress of orphan drugs in Europe: Introduction; Revenues from orphan drugs in Europe; What is similar?; Designated orphan drugs; Leading applicants; Success stories; Soliris (Alexion); Enzyme replacement therapies; Tracleer; Glivec (Novartis); Fragmented market issues
Chapter 5: Orphan drugs in the US: Overview; 25 years of approvals; Major successes
Chapter 6: Specialist Companies: Introduction; Major players; Genzyme; Actelion; Shire; BioMarin; Alexion; Established specialist players; Orphan Europe (Recordati); Sigma-Tau SpA; Swedish Orphan Biovitrum; PharmaMar; EUSA Pharma; Orfagen; Emerging speciality companies; Amicus Therapeutics; AmpliPhi Biosciences; bluebird bio; Del Mar Pharmaceuticals; Edimer Pharmaceuticals; Généthon; Hyperion Therapeutics; Lotus Tissue Repair; Orphan Therapeutics; Prosena; Santhera Pharmaceuticals; SpePharm; Sygnis Bioscience; Synageva BioPharma; Trophos; Vivendy Therapeutics; Zacharon; Zymenex
Chapter 7: Major pharmaceutical companies' involvement in orphan drugs: Introduction; Eli Lilly; GlaxoSmithKline; Novartis; Pfizer; Sanofi; Other companies; 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: 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

More Europe Prescription Drugs Reports

Pharmaceuticals – Poland – a snapshot (2011) by Mintel - Snapshots
Pharmaceuticals in Poland by Mintel Global Market Navigator provides you with annual year-end market size data, most recently updated in 2011. This market covers prescription ...

Switzerland Pharmaceuticals and Healthcare Report Q2 2012 by Business Monitor International
BMI View: The expected growth of the Switzerland’s ageing population over the next 50 years will havean unprecedented impact on the demand for healthcare. The ...

Spain Pharmaceuticals and Healthcare Report Q2 2012 by Business Monitor International
BMI View: Companies with significant exposure to debt-ridden European markets will be hard hit bygovernment austerity measures across the region, particularly by pharmaceutical price erosionmechanisms. ...

Romania Pharmaceuticals and Healthcare Report Q2 2012 by Business Monitor International
BMI View: Following the resignation of Emil Boc and his government in February 2012, there is nowconsiderable uncertainty with regard to reform of the Romanian ...

Portugal Pharmaceuticals and Healthcare Report Q2 2012 by Business Monitor International
BMI View: Since the approval of an EU/ECB/IMF economic rescue package in May 2011, cost cutting measureshave taken their toll on all sectors – including ...

See all reports like this >>

More Europe Reports

A Comprehensive Study Of The UK Video Conferencing Equipment Market In 2010, Rating & Valuing Each Of The 30 Companies That Operate In Today's Market by Plimsoll Publishing Ltd.
The Plimsoll Analysis provides you with the most up to date and comprehensive analysis of the top 9 companies operating the market. It is ...

A Comprehensive Study Of The UK Tableware Market In 2010, Rating & Valuing Each Of The 166 Companies That Operate In Today's Market by Plimsoll Publishing Ltd.
The Plimsoll Analysis provides you with the most up to date and comprehensive analysis of the top 9 companies operating the market. It is ...

A Comprehensive Study Of The UK Surveyors & Valuers Market In 2010, Rating & Valuing Each Of The 943 Companies That Operate In Today's Market by Plimsoll Publishing Ltd.
The Plimsoll Analysis provides you with the most up to date and comprehensive analysis of the top 9 companies operating the market. It is ...

A Comprehensive Study Of The UK Subsea Engineering Market In 2010, Rating & Valuing Each Of The 142 Companies That Operate In Today's Market by Plimsoll Publishing Ltd.
The Plimsoll Analysis provides you with the most up to date and comprehensive analysis of the top 9 companies operating the market. It is ...

A Comprehensive Study Of The UK Structural Engineering Consultancy Market In 2010, Rating & Valuing Each Of The 300 Companies That Operate In Today's Market by Plimsoll Publishing Ltd.
The Plimsoll Analysis provides you with the most up to date and comprehensive analysis of the top 9 companies operating the market. It is ...

See all reports like this >>

Orphan Drug Markets in Europe

Share this report

Other tasks

Research Assistance

Related Markets

Prescription Drugs Reports

Free Alert Me service