Orphan Drugs to 2008

Understanding regulation & market opportunity in Europe

Publication Date	January 2005
Publisher	URCH Publishing
Product Type	Report
Pages	155
ISBN Number	954698126
Product Code	URC00009

Buy this product or for assistance call +44 20 7060 7474

PDF £995.00$1655 | €1114

PRINT £995.00$1655 | €1114

FAX Order above formats by FAX

Summary

Orphan Drugs to 2008- Understanding regulation & market opportunity in Europe is designed for executives to understand the market environment for orphan drugs. The content will help all global companies to better understand the opportunities presented by recent developments in Europe orphan drug legislation. Over the last four years the legislation has provided opportunities for big and small companies, employing deliberate or add-on orphan drug development programmes. As has been shown through the success of the more mature market environment for US orphan medicines, the impact of European measures is likely to be significant and is only just beginning to materialise.

The report uses detailed regulatory, company and primary source information in order to provide a detailed guide to employing orphan drug development and marketing strategies in Europe. The report looks first at the global opportunity for orphan medicines, before outlining the European regulations and procedures in full detail. In order to assess the impact of the legislation both designated and subsequently launched orphan drugs are analysed and profiled. Detailed case studies lead to a set of key conclusions and recommendations for all pharmaceutical and biotechnology companies.

The report contains detailed analysis of major orphan drugs:
Aldurazyme, Busilvex, Carbaglu,Fabrazyme, Glivec, Litak, PhotoBarr, Onsenal, Replagal, Somavert, Tracleer, Trisenox,Ventavis and Zavesca with details of:

Disease description
Prevalence and incidence of disease
Development trials
Pivotal clinical trials
Development and regulatory timeline
CPMP conclusions
CPMP decision
Post-marketing programme
Steps taken after granting the Marketing Authorization
Commercial impact

Content

Executive Summary
Chapter 1: An Introduction to Orphan Drugs
- Report purpose
- Approach and methodology
- Report outline
Chapter 2: Orphan Drug Activity Outside Europe
- The impact of more than 20 years of US orphan drug legislation
- Regulations and procedures
- The US
- The Orphan Drug Act
- Orphan drug designation
- Orphan drug incentives
- Japan
- Definition of an orphan product
- The role of the OPSR
- The role of the MHLW
- Australia
- Key incentives
- Characteristics of the orphan drug programme
- Singapore
- Definition
- Legal framework for imports
- Impact and uptake
- Orphan drug designations
- Orphan drug approvals
- Leading orphan drug companies
- Leading orphan drugs
Chapter 3: Case Studies of Leading Orphan Drugs
- Epogen (Amgen)
- Anaemia associated with end-stage renal disease
- Anaemia in Zidovudine-treated HIV-infected patients
- Sales trends
- IntronA (Schering-Plough)
- AIDS-related Kaposi's sarcoma
- Chronic hepatitis C in paediatric patients
- Sales trends
- Remicade (Johnson & Johnson)
- Moderately to severely active Crohn's disease
- Sales trends
- Rituxan (Genentech)
- Non-Hodgkin's B-cell lymphoma
- Sales trends
- Enbrel (Amgen)
- Moderately to severe active juvenile rheumatoid arthritis
- Sales trends
- Neupogen (Amgen)
- Neutropenia in cancer patients receiving bone marrow transplant
- Severe chronic neutropenia
- Peripheral blood progenitor cell collection and therapy
- Acute myeloid leukaemia receiving induction or consolidation chemotherapy
- Sales trends
- Avonex (Biogen IDEC)
- Multiple sclerosis
- Sales trends
- Gleevec/Glivec (Novartis)
- Chronic myeloid leukaemia
- Gastrointestinal stromal tumours
- Sales trends
- Topamax (Johnson & Johnson)
- Lennox-Gastaut syndrome
- Sales trends
- Taxol (BMS)
- AIDS-related Kaposi's sarcoma
- Sales trends
Chapter 4: Orphan Drug Regulations in Europe
- Summary
- Current legislation and regulations
- History of European orphan drug legislation
- Key purposes of European orphan drug legislation
- Defining an orphan drug
- The Committee for Orphan Medicinal Products
- The designation procedure
- Key incentives
- Protocol assistance
- Community marketing authorisation
- Market exclusivity
- Other incentives
- Subsequent regulations
- Orphan drug designation applications
- Orphan drug designation procedures
- Orphan drug designation fee reductions
- Orphan drug designation protocol assistance
- Orphan drug designation inventory of incentives
- Key protagonists
- The Committee for Orphan Medicinal Products
- The European Agency for the Evaluation of Medicinal Products
- The European Commission/European Parliament
- Patient groups
- European Organisation for Rare Diseases (Eurordis)
- European Alliance of Patient and Parent Organisations for Innovation in Genetic Services (EAGS)
- Industry representatives
- Learned societies
- Key benefits and limitations
- Key benefits
- Key limitations
- Cross-regional comparison
- Key ambiguities
- 'Significant benefit'
- 'Similar product'
- 'Medical plausibility''
- 'Prevalence'
- Trade name
- Future legislation and regulations
- Budget
- Coverage
- US model
Chapter 5: European Orphan Drug Activity
- Orphan drug designations
- Key orphan drug indications
- Key orphan drug companies
- Key orphan drug timelines
- Key orphan drug strategies
- Extended indications/formulations of non-orphan drugs
- Global orphan drug designations
- Size of target indication prevalence
- Stage of development at designation
- Special licences for pre-approval use
Chapter 6: Fabrazyme - Genzyme Europe
- Overview
- Clinical pharmacology
- Fabry disease
- Development trials
- Pivotal clinical trials
- Development and regulatory timelines
- CPMP conclusions
- CPMP Decision
- Post-marketing programme
- Steps taken after granting the marketing authorisation
- Commercial impact
Chapter 7: Replagal - Transkaryotic Therapies
- Overview
- Fabry disease
- Incidence/prevalence of Fabry disease
- Development trials
- Pivotal clinical trial
- Other clinical trials
- Fabry Outcome Survey
- Development and regulatory timelines
- CPMP conclusions
- CPMP Decision
- Post-marketing programme
- Steps taken after granting the marketing authorisation
- Commercial impact
Chapter 8: Trisenox - Cell Therapeutics (UK)
- Overview
- Acute promyelocytic leukaemia (APL)
- Myelodysplastic syndromes
- Development trials
- Pivotal clinical trials
- Other clinical trials
- Development and regulatory timelines
- CPMP conclusions
- CPMP Decision
- Post-marketing programme
- Steps taken after granting the marketing authorisation
- Commercial impact
Chapter 9: Zavesca - Actelion Registration
- Overview
- Gaucher disease description
- Affected individuals
- Prevalence and incidence of Gaucher disease
- Development trials
- Pivotal clinical trials
- Development and regulatory timelines
- CPMP conclusions
- CPMP Decision
- Post-marketing programme
- Steps taken after granting the marketing authorisation
- Commercial impact
Chapter 10: Carbaglu - Orphan Europe
- Overview
- N-Acetylglutamate synthase (NAGS) deficiency
- Development trials
- Pivotal clinical trials
- Development and regulatory timelines
- CPMP conclusions
- CPMP Decision
- Steps taken after granting the marketing authorisation
- Commercial impact
Chapter 11: Busilvex - Pierre Fabre Mdicament
- Overview
- Haematopoietic progenitor cell transplantation
- Conditioning regimen
- Rationale for intravenous high-dose busulfan
- Development trials
- Pivotal clinical trials
- Other clinical trials
- Development and regulatory timelines
- CPMP conclusions
- CPMP Decision
- Post-marketing programme
- Steps taken after granting the marketing authorisation
- Commercial impact
Chapter 12: Aldurazyme - Genzyme Europe
- Overview
- Mucopolysaccharidosis, type I
- Signs and symptoms
- Disease progression
- Development trials
- Pivotal clinical trials
- Other clinical trials
- Development and regulatory timelines
- CPMP conclusions
- CPMP Decision
- Post-marketing programme
- Steps taken after granting the marketing authorisation
- Commercial impact
Chapter 13: Glivec - Novartis Europharm
- Overview
- Indications and usage
- Drivers
- Barriers
- Chronic myeloid leukaemia
- Development trials
- Pivotal clinical trials
- Other clinical trials
- Development and regulatory timelines
- CPMP conclusions
- CPMP Decision
- Post-marketing programme
- Steps taken after granting the marketing authorisation
- Commercial impact
Chapter 14: Somavert - Pharmacia Enterprises
- Overview
- Acromegaly description
- Prevalence and incidence of acromegaly
- Development trials
- Pivotal clinical trials
- Adverse events
- Other clinical trials
- Development and regulatory timelines
- CPMP conclusions
- CPMP Decision
- Post-marketing programme
- Steps taken after granting the marketing authorisation
- Commercial impact
Chapter 15: Tracleer - Actelion Registration
- Overview
- Pulmonary arterial hypertension description
- Development trials
- Pivotal clinical trials
- Other clinical trials
- Development and regulatory timelines
- CPMP conclusions
- CPMP Decision
- Post-marketing programme
- Steps taken after granting the marketing authorisation
- Commercial impact
Chapter 16: Four Other Key Orphan Drugs
- Ventavis
- Litak
- Onsenal
- PhotoBarr
Chapter 17: Key Conclusions and Recommendations
- Key orphan drug strategies
- Elements in the design of an orphan drug strategy
- Trial design and initial indication approved
- Speed to market
- Pricing
- Profile raising
- Licensing and marketing agreements
- Key success stories and bottlenecks
- Glivec: a success story
- Somavert: an example of bottlenecks
- Key recommendations
List of Figures
- Figure 2.2 - US orphan drug approvals, 1983-2004
- Figure 2.3 - Leading companies by US orphan drug designations, 1983-2004
- Figure 3.1 - Epogen global sales, 1999-2003
- Figure 3.2 - IntronA global sales, 1999-2003
- Figure 3.3 - Remicade global sales, 1999-2003
- Figure 3.4 - Rituxan global sales, 1999-2003
- Figure 3.5 - Enbrel global sales, 1999-2003
- Figure 3.6 - Neupogen global sales, 1999-2003
- Figure 3.7 - Avonex global sales, 1999-2003
- Figure 3.8 - Gleevec/Glivec global sales, 2001-03
- Figure 3.9 - Topamax global sales, 1999-2003
- Figure 3.10 - Taxol global sales, 1999-2003
- Figure 4.1 - The orphan designation procedure
- Figure 6.1 - Fabrazyme: key development and regulatory timelines
- Figure 6.2 - Fabrazyme sales, 2001-04
- Figure 7.1 - Replagal: key development and regulatory timelines
- Figure 7.2 - Replagal sales, 2002-04
- Figure 8.1 - Trisenox: key development and regulatory timelines
- Figure 8.2 - Trisenox sales, 2002-04
- Figure 9.1 - Zavesca: key development and regulatory timelines
- Figure 9.2 - Zavesca sales, 2003/04
- Figure 10.1 - Carbaglu: key development and regulatory timelines
- Figure 11.1 - Busilvex: key development and regulatory timelines
- Figure 12.1 - Aldurazyme: key development and regulatory timelines
- Figure 12.2 - Aldurazyme sales, 2003/04
- Figure 13.1 - Glivec: key development and regulatory timelines
- Figure 13.2 - Glivec sales, 2001-04
- Figure 14.1 - Somavert: key development and regulatory timelines
- Figure 15.1 - Tracleer: key development and regulatory timelines
- Figure 15.2 - Tracleer sales, 2001-04
- Figure 17.1 - Components in a successful orphan drug strategy
- Figure 17.2 - Key business strategies for orphan drugs
List of Tables
- Table 2.1 - Leading orphan drugs, 2002/03
- Table 3.1 - Epogen (erythropoietin alfa) - drug monograph
- Table 3.2 - IntronA/Rebetol (interferon alfa-2b/ribavirin) - drug monograph
- Table 3.3 - Remicade (infliximab) - drug monograph
- Table 3.4 - Rituxan (rituximab) - drug monograph
- Table 3.5 - Enbrel (etanercept) - drug monograph
- Table 3.6 - Neupogen (filgrastim) - drug monograph
- Table 3.7 - Avonex (interferon beta-1a) - drug monograph
- Table 3.8 - Gleevec/Glivec (imatinib mesilate) - drug monograph
- Table 3.9 - Topamax (topiramate) - drug monograph
- Table 3.10 - Taxol (paclitaxel) - drug monograph
- Table 4.1 - Cross-regional comparison of orphan drug programmes
- Table 5.1 - Orphan drug activity, 2000-04
- Table 5.2 - Key orphan drug indications
- Table 5.3 - Orphan drugs with multiple indications
- Table 5.4 - Key orphan drug companies
- Table 5.5 - Orphan drugs with multiple sponsors
- Table 5.6 - Orphan drugs receiving market authorisation
- Table 5.7 - Global orphan drugs
- Table 6.1 - Fabrazyme (agalsidase beta) - drug monograph
- Table 7.1 - Replagal (agalsidase alfa) - drug monograph
- Table 7.2 - Overview of clinical trials programme
- Table 8.1 - Trisenox (arsenic trioxide) - drug monograph
- Table 9.1 - Zavesca (miglustat) - drug monograph
- Table 10.1 - Carbaglu (carglumic acid) - drug monograph
- Table 11.1 - Busilvex (busulfan) - drug monograph
- Table 12.1 - - Aldurazyme (laronidase) - drug monograph
- Table 13.1 - Glivec (imatinib mesilate) - drug monograph
- Table 13.2 - EMEA (EU) approved indications for Glivec
- Table 14.2 - Somavert (pegvisomant) - drug monograph
- Table 15.1 - Tracleer (bosentan) - drug monograph
- Table 17.1 - Marketing submission to approval for orphan drugs in Europe