Biologics: Specific Drug Safety Challenges. Violetta B. Kyburz

Save this PDF as:
 WORD  PNG  TXT  JPG

Size: px
Start display at page:

Download "Biologics: Specific Drug Safety Challenges. Violetta B. Kyburz"

Transcription

1 Biologics: Specific Drug Safety Challenges Violetta B. Kyburz

2 Biologics: Specific Drug Safety Challenges Topics for discussion Particular issues in the preclinical development of biologics Challenges for biologics in clinical development Are there differences between small molecules and biologics post marketing? Biosimilars: implications for pharmacovigilance

3 Biologics: Increasing Role in the Treatment of Cancer and Autoimmune Diseases Biologics: Recombinant human proteins (enzymes, cytokines, growth factors, hormones), monoclonal antibodies (mabs), fusion proteins Large, highly complex molecules with a very high target affinity and a much higher species specificity than small molecules First biomolecular drug launched in 1982: Humulin (human insulin), first mab launched 1986: OKT 3 For many years one of the fastest growing drug classes

4 Differences Between Small Molecules and Proteins Molecular weight Complex secondary, tertiary, and quaternary structures Biologics: targeted therapy to interact or modulate human cellular targets Source: Amouzadeh and Vargas, 2013

5 Significant Rise of Biologics/Orphan Drugs are Changing Drug Development and Drug Safety Landscape Source: FDA, Office of Orphan Products Development, Mathew T. Thomas

6 Significant Rise of Biologics/Orphan Drugs are Changing Drug Development and Drug Safety Landscape Source: Jim Kling, Nature Biotechnology; Vol. 32; , 2014

7 Number of Patients Studied Prior to Approval Orphan Source: Duijnhoven et al. PLoS March 2013

8 Top Selling Drugs in 2013: 7 of the Top 10 were Biologics Product INN Company Global Sales 2013 (in bio $) Biosimilars 1. Humira adalimumab AbbVie/Eisai 11.0 Phase III 2. Enbrel etenercept Amgen/Pfizer/ Takeda 3. Remicade infliximab J&J/ Merck & Co 8.8 Phase III 8.4 EU approved, Advair fluticasone GSK 8.25 NA 5. Lantus Insuline glargine Sanofi 7.6 EU approved, Rituxan/ MabThera rituximab Roche/BiogenIdec 7.5 Phase III 7. Avastin bevacizumab Roche 6.8 Phase III 8. Herceptin trastuzumab Roche 6.6 Phase III 9. Crestor Rosuvastatin AstraZeneca 6.0 NA 10 Abilify Aripiprazole BMS/Otsuka 5.5 NA Source: First Word and Fierce Pharma, March 2014

9 Specifics of Biologics in Preclinical Development Selection of relevant animal model is often a challenge For mabs: usually immunogenic in animals Prolonged half-life and lag time for pharmacodynamic effect MoA for new targets often not entirely clear ADRs for biologics are typically «on-target» effect but off-target toxicities cannot be excluded Toxicology profile for a biologic can be unrevealing: Predictability of adverse drugs reactions based on animal trials is low Additional in-vitro, in-vivo and ex-vivo tests necessary

10 Developing and Manufacturing Biologics vs Small Molecules Biologics Manufactured in a living system such as a microorganism, plant or animal cells Complex to manufacture Higher GMP requirements Difficult to characterize Small Molecules Manufacturing process is chemical synthesis, combining specific chemical ingredients in an ordered process Well-defined chemical structures Finished drug can usually be analyzed to determine all its various components

11 Developing and Manufacturing Biologics vs Small Molecules Biologics Manufactured in a living system such as a microorganism, plant- or animal cells Complex to manufacture Higher GMP requirements Difficult to characterize Small Molecules Manufacturing process is chemical synthesis, combining specific chemical ingredients in an ordered process Well-defined chemical structures, Finished drug can usually be analyzed to determine all its various components Picture from

12 Developing and Manufacturing Biologics The Process is the Product Even minor alterations in the manufacturing process or in the formulation may produce functional changes (cell behaviour, differences in structure, stability or other quality aspects) Batch-to-batch variability regarding purity and aggregation is the norm Any of these alterations may affect the safety and efficacy of the biologic and can increase the risk of immunogenicity and ADRs

13 Challenges in Clinical Development of Biologics Pre-clinical trials are often not predictive for acute and long-term toxicity of biologics ADRs of biologics are often related to immune function, may be more complex and rare; late onset is possible Number of patients treated in pivotal clinical trials often smaller (biologics often developed for orphan/rare diseases or as third-line therapy) and longer-term usage even less, i.e. too small to detect more rare ADRs

14 Lack of Preclinical Toxicity Finding does not Preclude that Things Can Go Terribly Wrong: TGN 1412 (TeGenero) In Phase I trial, 6/8 patients developed serious adverse events: organ failure, respiratory distress, DIC Transferred to ICU (part of same facility) Initial signs appeared min after dosing Produced by Cytokine Release Storm

15 Adverse Drug Reactions Specific to Biologics Immunotoxicity: immune-response mediated reactions such as immunogenicity, hypersensitivity, and acute phase reactions Usually ADRs are «on-target» and thus often affect the immune system. However there are differences between biologics for the same biologic in different indications ADRs of biologics typically include: infections reactivation of viruses and bacteria malignancies

16 Immune Related Safety Warnings of Top Biologics Biologic Type Indications Immune Related Safety Warning Adalimumab Human mab RA, CD, AS Serious infections, malignancies, HSTCL, immunogenicity Etanercept Fusion protein RA, AS, psoriasis Serious infections (TB, fungal), malignancy PML, neutropenia, infections Infliximab Chimeric mab RA, CD, AS, UC Serious infections, malignancy, PML, HSTCL, immunogenicity CRS/SIRS, immunogenicity, Rituximab Chimeric mab NHL, CLL, RA, Wegener s granulomatosis Acute infusion reactions, CRS, TLS, severe mucocutaneous reactions PML Source: Satish et al, Nature Reviews, April 2013, Vol 12;

17 Approval Process for Small Molecule Generics, New Biologic Agents, and Biosimilars Quality Small Molecule Generic Individual quality assessment Comparison with reference product New Biological Agent Individual quality assessment Preclinical No data required Full pre-clinical program Clinical Bioequivalence study Phase I Phase II Phase III in all indications Risk Management Plan Biosimilar Individual quality assessment Comprehensive comparison with reference product Abbreviated pre-clinical program (tolerance, PK/PD) Phase I PK/PD study Phase III study in one sensitive, representative indication Risk Management Plan Source: Mellstedt, Clinical considerations for biosimilar antibodies, EJC, Vol.11 (3); 1 11, Dec 2013

18 Biosimilars and Implications for Pharmacovigilance Since 2005/2007, relatively simple biosimilars such as growth hormones, erythropoietin and GCSF available With the upcoming patent expiry of several mab biologics, biosimilar mabs are close to market entry Pre-clinical/clinical path already established by reference biologic BUT Although similar to the reference biologic, the biosimilar may be different with respect to molecular structure, glycosylation etc. This may affect both efficacy and safety.

19 Biosimilars and Implications for Pharmacovigilance Even fewer patients studied during clinical development of biosimilars Unlikely to detect rare ADRs risk minimization strategies of safety and monitoring of efficacy (especially in indications where the biosimilar drug has not been studied) will be important All currently approved biosimilars in the EU have additional requirements regarding surveillance (patient questionnaires, registries, post-marketing surveillance)

20 Remsima: First Biosimilar mab Approved in 2013 (RA, Crohn s Disease, UC, Ankylosing Arthritis, Psoriasis) A total of 440 patients treated with Remsima for at least 30 weeks: Several PAES studies and registries for Crohn s disease and IB requested by EMA to further clarify long-term safety profile of patients Source: Assessment Report Remsima EMA, June 27, 2013

21 Conclusions Biologics currently represent 30% of licensed products and are among the world s best-selling pharmaceuticals Due to high affinity for targets, biologics usually have fewer unwanted effects compared to small molecules ADRs fall into two groups: «on-target» ADRs triggered by exaggerated pharmacology and «off-target» ADRs Developing biologics is complex and requires a thorough understanding of disease, immune system, target of immunomodulation and of the biologic itself Preclinical studies, particularly animal studies (due to the species specificity) can be unrevealing Patient numbers studied prior to approval are smaller for biologics compared to small molecules and even smaller for biosimilars Pharmacovigilance of biologics needs (even more) expertise and thorough understanding of biologics and diseases

22 Thank you Violetta Kyburz, Commercial Director Fishawack Archimed Tel: LinkedIn: Fishawack Group of Companies

Overview of Phase 1 Oncology Trials of Biologic Therapeutics

Overview of Phase 1 Oncology Trials of Biologic Therapeutics Overview of Phase 1 Oncology Trials of Biologic Therapeutics Susan Jerian, MD ONCORD, Inc. February 28, 2008 February 28, 2008 Phase 1 1 Assumptions and Ground Rules The goal is regulatory approval of

More information

Monoclonal antibody (mab) products are currently a fast

Monoclonal antibody (mab) products are currently a fast Antibody Monoclonal Therapeutics Janice M. Reichert, Ph.D. Monoclonal antibody (mab) products are currently a fast growing category of drugs. Like antibodies produced naturally by the human immune system,

More information

Company Presentation June 2011 Biotest AG 0

Company Presentation June 2011 Biotest AG 0 Company Presentation June 2011 0 Disclaimer This document contains forward-looking statements on overall economic development as well as on the business, earnings, financial and asset situation of and

More information

Biologic Treatments for Rheumatoid Arthritis

Biologic Treatments for Rheumatoid Arthritis Biologic Treatments Rheumatoid Arthritis (also known as cytokine inhibitors, TNF inhibitors, IL 1 inhibitor, or Biologic Response Modifiers) Description Biologics are new class of drugs that have been

More information

Biotherapeutic Medicines

Biotherapeutic Medicines International Federation of Pharmaceutical Manufacturers & Associations Biotherapeutic Medicines Grasping the New Generation of Treatments Biotherapeutic medicines are an integral and valuable part of

More information

Biologics Biosimilars

Biologics Biosimilars Biologics Biosimilars Q u e st i o n s Po l i c y S a fe t y What are biosimilars? Biosimilars are sometimes incorrectly and inappropriately called generic versions of original biological medicines. But

More information

TGN 1412 Welche Änderungen haben sich für die Erstanwendung am Menschen aus Sicht des BfArM ergeben?

TGN 1412 Welche Änderungen haben sich für die Erstanwendung am Menschen aus Sicht des BfArM ergeben? TGN 1412 Welche Änderungen haben sich für die Erstanwendung am Menschen aus Sicht des BfArM ergeben? PD Dr. med. Thomas Sudhop Bundesinstitut für Arzneimittel, Bonn Bundesinstitut für Arzneimittel IMP

More information

Non-clinical development of biologics

Non-clinical development of biologics Aurigon Life Science GmbH Non-clinical development of biologics Requirements, challenges and case studies Committed to Life. Sigrid Messemer vet. med. M4 Seminar March 10 th 2014 Aurigon - your full service

More information

Company Presentation

Company Presentation Company Presentation DISCLAIMER This document contains forward-looking statements on overall economic development as well as on the business, earnings, financial and asset situation of amp biosimilars

More information

Immune modulation in rheumatology. Geoff McColl University of Melbourne/Australian Rheumatology Association

Immune modulation in rheumatology. Geoff McColl University of Melbourne/Australian Rheumatology Association Immune modulation in rheumatology Geoff McColl University of Melbourne/Australian Rheumatology Association A traditional start to a presentation on biological agents in rheumatic disease is Plasma cell

More information

Welcome to the Launch of Biocon s Breakthrough Innovation. For Psoriasis. Treatment

Welcome to the Launch of Biocon s Breakthrough Innovation. For Psoriasis. Treatment Welcome to the Launch of Biocon s Breakthrough Innovation For Psoriasis Treatment PRESS MEET Bangalore, August 10, 2013 Autoimmune Diseases are on the Rise Globally Autoimmune Disease Segment includes

More information

Biosimilar Monoclonal Antibodies in the Pipeline: Major Players and Strategies

Biosimilar Monoclonal Antibodies in the Pipeline: Major Players and Strategies Biosimilar Monoclonal Antibodies in the Pipeline: Major Players and Strategies Fern Barkalow, Ph.D, Senior Analyst, Oncology Citeline One of the biggest challenges facing biosimilar drug developers is

More information

PRODUCT INN REFERENCE AUTHORIZATION Omnitrope

PRODUCT INN REFERENCE AUTHORIZATION Omnitrope PRODUCT INN REFERENCE AUTHORIZATION Omnitrope Genotropin 12/04/2006 somatropin Valtropin (W/D) Humatrope 24/04/2006 Abseamed Binocrit Epoetina alfa hexal Retacrit Silapo Biograstim Filgrastim ratiopharm

More information

Clinical trials preclinical requirements. Clinical trials - legislation

Clinical trials preclinical requirements. Clinical trials - legislation Clinical trials preclinical requirements Mikael Andersson, PhD Senior Expert Medical Products Agency, Sweden Tallinn 9/10 2009 Clinical trials - legislation Directive 2001/20/EC Clinical trial directive

More information

The Future of Monoclonal Antibodies Therapeutics: Innovation in Antibody Engineering, Key Growth Strategies and Forecasts to 2011

The Future of Monoclonal Antibodies Therapeutics: Innovation in Antibody Engineering, Key Growth Strategies and Forecasts to 2011 Brochure More information from http://www.researchandmarkets.com/reports/344881/ The Future of Monoclonal Antibodies Therapeutics: Innovation in Antibody Engineering, Key Growth Strategies and Forecasts

More information

EMA and Progressive Multifocal Leukoencephalopathy.

EMA and Progressive Multifocal Leukoencephalopathy. EMA and Progressive Multifocal Leukoencephalopathy. ENCePP Plenary, London 23 November 2011 Presented by: Henry Fitt Head of Coordination & Networking, Pharmacovigilance & Risk Management An agency of

More information

NATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE. Health Technology Appraisal

NATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE. Health Technology Appraisal NATIONAL INSTITUTE FOR HEALTH AND CLINICAL EXCELLENCE Health Technology Appraisal Adalimumab, etanercept, infliximab, rituximab and abatacept for the treatment of rheumatoid arthritis after the failure

More information

Biological. Medicines. A Focus on Biosimilar. Medicines

Biological. Medicines. A Focus on Biosimilar. Medicines TM Biological Medicines TM EuropaBio is the voice of the European biotech Industry. It represents the interests of the industry towards the European institutions so that legislation encourages and enables

More information

A Comparison of US and EU Biosimilars Regimes

A Comparison of US and EU Biosimilars Regimes A Comparison of US and EU Biosimilars Regimes summary statement Economic barriers, along with regulatory complexity and uncertainty, are shaping the biosimilars industry into something entirely different

More information

The case for biosimilar monoclonal antibodies: Ask the experts - biosimilar speaker tour

The case for biosimilar monoclonal antibodies: Ask the experts - biosimilar speaker tour The introduction of biosimilars to the New Zealand health system The case for biosimilar monoclonal antibodies: Ask the experts - biosimilar speaker tour July 2012 1 The case for biosimilar monoclonal

More information

Lessons for the United States: Biosimilar Market Development Worldwide

Lessons for the United States: Biosimilar Market Development Worldwide Lessons for the United States: Biosimilar Market Development Worldwide Sumant Ramachandra, MD, PhD, MBA Senior Vice President, Chief Scientific Officer Hospira $67B+ of 2012 LMV is expected to face biosimilar

More information

Scientific Challenges for Development of Biosimilar Monoclonal Antibodies. Rafiqul Islam Director, Global Bioanalytical Services Celerion

Scientific Challenges for Development of Biosimilar Monoclonal Antibodies. Rafiqul Islam Director, Global Bioanalytical Services Celerion Scientific Challenges for Development of Biosimilar Monoclonal Antibodies Rafiqul Islam Director, Global Bioanalytical Services Celerion Presentation outline Biosimilars Definitions and Concepts Regulatory

More information

Winter 2013. Changing landscapes, pipeline products and plan sponsor impact

Winter 2013. Changing landscapes, pipeline products and plan sponsor impact Winter 2013 Changing landscapes, pipeline products and plan sponsor impact Changing landscapes, pipeline products and plan sponsor impact The pharmaceutical landscape is changing as is the profile of blockbuster

More information

PlantForm Corporation

PlantForm Corporation PlantForm Corporation Professor J. Christopher Hall CSO PlantForm Corporation Canada s Top10 Winner Life Sciences 2008/9 Innovator of the Year Guelph, Canada, 2010 Opportunity The introduction of more

More information

Biopharmaceuticals and Products Liability Litigation

Biopharmaceuticals and Products Liability Litigation Biopharmaceuticals and Products Liability Litigation Steven Weisman, Ph.D. Head, Global Healthcare Products Innovative Science Solutions ABA Roundtable April 17, 2013 Krista L. Cosner Counsel Drinker Biddle

More information

Briefing Paper on Biological and Biosimilar Medicines

Briefing Paper on Biological and Biosimilar Medicines Briefing Paper on Biological and Biosimilar Medicines Contents With our thanks IAPO would like to thank all those involved in planning this Toolkit and supporting its development. A full list of acknowledgements

More information

Rheumatoid Arthritis. Outline. Treatment Goal 4/10/2013. Clinical evaluation New treatment options Future research Discussion

Rheumatoid Arthritis. Outline. Treatment Goal 4/10/2013. Clinical evaluation New treatment options Future research Discussion Rheumatoid Arthritis Robert L. Talbert, Pharm.D., FCCP, BCPS University of Texas at Austin College of Pharmacy University of Texas Health Science Center at San Antonio Outline Clinical evaluation New treatment

More information

An Industry Perspective

An Industry Perspective AIPPI World IP Congress 2014 Workshop Pharma 2 Biosimilar pharmaceutical products An Industry Perspective September 16, 2014 Masahisa Yamaguchi, PhD Department Manager Intellectual Property Dept. Chugai

More information

ENDORSED BY THE GOVERNANCE COMMITTEE

ENDORSED BY THE GOVERNANCE COMMITTEE Guideline for the Preparation or Manipulation of Monoclonal Antibodies (MABs) and related compounds such as Fusion Proteins, used in the Treatment of Cancer Date Approved by Network Governance July 2012

More information

RADIOPHARMACEUTICALS BASED ON MONOCLONAL ANTIBODIES

RADIOPHARMACEUTICALS BASED ON MONOCLONAL ANTIBODIES RADIOPHARMACEUTICALS BASED ON MONOCLONAL ANTIBODIES Guideline Title Radiopharmaceuticals based on Monoclonal Antibodies Legislative basis Directives 65/65/EEC, 75/318/EEC as amended, Directive 89/343/EEC

More information

Biologics and biosimilars. An overview

Biologics and biosimilars. An overview Biologics and biosimilars An overview Contents An introduction to biotechnology... 3 A brief history of medicine development... 4 What are biologic medicines?... 5 How are biologic medicines developed?...

More information

Read the Questions Carefully

Read the Questions Carefully PHT. 463- (Quality Control of Pharmaceutical Dosage Forms) Midterm Exam I Dr. Ibrahim A. Alsarra s Section Part I Read the Questions Carefully TRUE OR FALSE: Indicate whether the statement is true or false

More information

Biosimilars Demystified

Biosimilars Demystified Biosimilars Demystified Thijs J. Giezen, PhD, PharmD, MSc Hospital Pharmacist, Foundation Pharmacy for Hospitals in Haarlem Member Biosimilar Working Party, European Medicines Agency I attend this conference

More information

Challenges and Opportunities of Monoclonal Antibody Manufacturing in China

Challenges and Opportunities of Monoclonal Antibody Manufacturing in China BioPharma Antibody Therapeutics News Digest Challenges and Opportunities of Monoclonal Antibody Manufacturing in China Chris Chen Introduction About the Author: Dr. Chris Chen is currently Chief Operating

More information

EBE Position paper on labelling of biosimilars Summary of Product Characteristics (SmPC) and Patient Information Leaflet (PIL)- draft April 2013

EBE Position paper on labelling of biosimilars Summary of Product Characteristics (SmPC) and Patient Information Leaflet (PIL)- draft April 2013 FINAL EBE 21 August 2013 EBE Position paper on labelling of biosimilars Summary of Product Characteristics (SmPC) and Patient Information Leaflet (PIL)- draft April 2013 Introduction This document seeks

More information

biologics for the treatment of psoriasis

biologics for the treatment of psoriasis How to contact us The Psoriasis Association Dick Coles House 2 Queensbridge Northampton NN4 7BF tel: 08456 760 076 (01604) 251 620 fax: (01604) 251 621 email: mail@psoriasis-association.org.uk www.psoriasis-association.org.uk

More information

GUIDELINES FOR THE REGISTRATION OF BIOLOGICAL PEST CONTROL AGENTS FOOD AND AGRICULTURE ORGANIZATION OF THE UNITED NATIONS

GUIDELINES FOR THE REGISTRATION OF BIOLOGICAL PEST CONTROL AGENTS FOOD AND AGRICULTURE ORGANIZATION OF THE UNITED NATIONS GUIDELINES FOR THE REGISTRATION OF BIOLOGICAL PEST CONTROL AGENTS FOOD AND AGRICULTURE ORGANIZATION OF THE UNITED NATIONS -ii- GUIDELINES ON THE REGISTRATION OF BIOLOGICAL PEST CONTROL AGENTS FOOD AND

More information

Testing Services for Large Molecule Drug Development

Testing Services for Large Molecule Drug Development Testing Services for Large Molecule Drug Development Our mission is to extend our clients capabilities by combining scientific knowledge, capacity, regulatory expertise and flexibility to provide the trusted,

More information

REFERENCE CODE GDHC503DFR PUBLICAT ION DATE DECEMBER 2014 METHOTREXATE (RHEUMATOID ARTHRITIS) - FORECAST AND MARKET ANALYSIS TO 2023

REFERENCE CODE GDHC503DFR PUBLICAT ION DATE DECEMBER 2014 METHOTREXATE (RHEUMATOID ARTHRITIS) - FORECAST AND MARKET ANALYSIS TO 2023 REFERENCE CODE GDHC503DFR PUBLICAT ION DATE DECEMBER 2014 METHOTREXATE (RHEUMATOID ARTHRITIS) - Executive Summary The table below provides the key metrics for Methotrexate in the 10MM (US, France, Germany,

More information

Chugai Pharmaceutical Co., Ltd. Oncology Area, Scientific Group 2, Breast Cancer Group Takehiro Yamaguchi, Herceptin Product Manager

Chugai Pharmaceutical Co., Ltd. Oncology Area, Scientific Group 2, Breast Cancer Group Takehiro Yamaguchi, Herceptin Product Manager Anti-malignant Tumor Agent with Anti-HER2 Humanized Monoclonal Antibody: Biologics, Designated drug, Prescription drug HERCEPTIN Injection 60 HERCEPTIN Injection 150 Trastuzumab (genetical recombination)

More information

Implementation of Advanced Chromatography techniques to Mitigate Purification Concerns in Bispecific Monoclonal Antibody Manufacturing

Implementation of Advanced Chromatography techniques to Mitigate Purification Concerns in Bispecific Monoclonal Antibody Manufacturing www.beroe- WHITEPAPER December 2012 inc.com Implementation of Advanced Chromatography techniques to Multi Column Solvent Gradient Purification or MCSGP chromatography process is a better solution to Bispecific

More information

Original Policy Date

Original Policy Date MP 5.01.20 Tysabri (natalizumab) Medical Policy Section Prescription Drug Issue 12:2013 Original Policy Date 12:2013 Last Review Status/Date Local Policy/12:2013 Return to Medical Policy Index Disclaimer

More information

Basic Overview of Preclinical Toxicology Animal Models

Basic Overview of Preclinical Toxicology Animal Models Basic Overview of Preclinical Toxicology Animal Models Charles D. Hebert, Ph.D., D.A.B.T. December 5, 2013 Outline Background In Vitro Toxicology In Vivo Toxicology Animal Models What is Toxicology? Background

More information

IMPROVEMENT OF RECOMBINANT PROTEIN S COST OF GOODS & AFFORDABILITY

IMPROVEMENT OF RECOMBINANT PROTEIN S COST OF GOODS & AFFORDABILITY IMPROVEMENT OF RECOMBINANT PROTEIN S COST OF GOODS & AFFORDABILITY Li How Chen PhD Senior Director, revo Biologics Inc. USA Sami CHTOUROU PhD Senior Vice President Innovation & Scientific Affairs LFB Biotechnologies

More information

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) GUIDELINE ON SIMILAR BIOLOGICAL MEDICINAL PRODUCTS

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) GUIDELINE ON SIMILAR BIOLOGICAL MEDICINAL PRODUCTS European Medicines Agency Evaluation of Medicines for Human Use CHMP/437/04 London, 30 October 2005 COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) GUIDELINE ON SIMILAR BIOLOGICAL MEDICINAL PRODUCTS

More information

Quality and Safety Evaluation of Gene Therapy Products in Japan

Quality and Safety Evaluation of Gene Therapy Products in Japan Quality and Safety Evaluation of Gene Therapy Products in Japan Review Mechanism for Gene Therapy in Japan The review mechanism for gene therapy in Japan was partially amended to simplify the necessary

More information

Goals & Objectives. Drug Development & the FDA Pharmacy 309. Outline. An History of Disasters. Be able to describe

Goals & Objectives. Drug Development & the FDA Pharmacy 309. Outline. An History of Disasters. Be able to describe Drug Development & the FDA Pharmacy 309 Tom Hazlet, Pharm.D., Dr.P.H. 616-2732 thazlet@u... Goals & Objectives Be able to describe the major regulatory events in the drug development process the concepts

More information

Challenges in early clinical development of biologics

Challenges in early clinical development of biologics Challenges in early clinical development of biologics Peter Lloyd March 2011 Acknowledgements: Jennifer Sims, Phil Lowe, Sebastian Spindeldreher, Andrew Warren Protein therapeutics biologics Current generation

More information

WHITE PAPER. Developing Biosimilars in Emerging Markets: Regulatory and Clinical Considerations

WHITE PAPER. Developing Biosimilars in Emerging Markets: Regulatory and Clinical Considerations WHITE PAPER Developing Biosimilars in Emerging Markets: Regulatory and Clinical Considerations ppdi.com March 2013 EXECUTIVE SUMMARY Emerging markets in Asia Pacific, Latin America and Eastern Europe are

More information

Commercial Insight: Cancer Targeted Therapies and Immunotherapies -Top monoclonal antibody brands will resist competitive pressures through to 2019

Commercial Insight: Cancer Targeted Therapies and Immunotherapies -Top monoclonal antibody brands will resist competitive pressures through to 2019 Brochure More information from http://www.researchandmarkets.com/reports/1314569/ Commercial Insight: Cancer Targeted Therapies and Immunotherapies -Top monoclonal antibody brands will resist competitive

More information

Rheumatic Diseases, Psoriasis, and Crohn s Disease

Rheumatic Diseases, Psoriasis, and Crohn s Disease Rheumatic Diseases, Psoriasis, and Crohn s Disease What does this handout cover? This handout has information about rheumatic disease, psoriasis, and Crohn s disease. It also has information on how these

More information

From The Lab To Your Medicine Cabinet:

From The Lab To Your Medicine Cabinet: From The Lab To Your Medicine Cabinet: A Pharmaceutical Drug s Journey Presented by: Dr. Steven Hansel Sr. Director; Pfizer Moderated by: Sam Gilchrist Ph.D. Candidate; UBC The Drug Development Process

More information

Achieving Regulatory Success: Areas of focus for biotechnology companies. Michael J. Schlosser, PhD, DABT April 21, 2013

Achieving Regulatory Success: Areas of focus for biotechnology companies. Michael J. Schlosser, PhD, DABT April 21, 2013 Achieving Regulatory Success: Areas of focus for biotechnology companies Michael J. Schlosser, PhD, DABT April 21, 2013 Regulatory Success Outline Regulatory Initiatives Regulatory Science Pre-Regulatory

More information

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP)

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) European Medicines Agency Evaluation of Medicines for Human Use London, 22 February 2006 EMEA/CHMP/BMWP/31329/2005 COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) ANNEX TO GUIDELINE ON SIMILAR BIOLOGICAL

More information

Eisen en Verplichtingen

Eisen en Verplichtingen Registratie ti van Biosimilars: i il Eisen en Verplichtingen Dr. Thijs J. Giezen Ziekenhuisapotheker, Stichting Apotheek Haarlemse Ziekenhuizen Lid, CHMP Biosimilar Working Party (BMWP), European Medicines

More information

RESEARCH DIAGNOSTICS PHARMACEUTICALS

RESEARCH DIAGNOSTICS PHARMACEUTICALS RESEARCH DIAGNOSTICS PHARMACEUTICALS Therapeutic drug monitoring is vitally important to optimize therapy for all patients treated with biologicals. Routine measurement of serum drug levels brings both

More information

Luca Romagnoli, Ph.D. Business Development Manager

Luca Romagnoli, Ph.D. Business Development Manager Modelli innovativi di produzione per lo sviluppo di un processo altamente qualitativo di farmaci biologici Luca Romagnoli, Ph.D. Business Development Manager BIOLOGICAL DRUGS - SOURCES Monoclonal antibodies

More information

Understanding specialty drugs

Understanding specialty drugs Group Benefits Understanding specialty drugs What your pharmaceutical benefits plan may look like over the next decade Between 2011 and 2014, the percentage of total drug spend represented by specialty

More information

General toxicity study designs

General toxicity study designs General toxicity study designs Jan Willem van der Laan Section on Safety of Medicines and Teratology Centre for Biological Medicines and Medical Technology National Institute for Public Health and the

More information

INTERNATIONAL CONFERENCE ON HARMONISATION OF TECHNICAL REQUIREMENTS FOR REGISTRATION OF PHARMACEUTICALS FOR HUMAN USE S1A. Current Step 4 version

INTERNATIONAL CONFERENCE ON HARMONISATION OF TECHNICAL REQUIREMENTS FOR REGISTRATION OF PHARMACEUTICALS FOR HUMAN USE S1A. Current Step 4 version INTERNATIONAL CONFERENCE ON HARMONISATION OF TECHNICAL REQUIREMENTS FOR REGISTRATION OF PHARMACEUTICALS FOR HUMAN USE ICH HARMONISED TRIPARTITE GUIDELINE GUIDELINE ON THE NEED FOR CARCINOGENICITY STUDIES

More information

Objectives: Immunity Gone Wrong: Autoimmune Diseases in Dental Hygiene Practice

Objectives: Immunity Gone Wrong: Autoimmune Diseases in Dental Hygiene Practice Objectives: 1) Understand the concept of self- tolerance versus non- self- tolerance. 2) Recognize systemic and oral indicators of autoimmune diseases. 3) Identify various autoimmune diseases and their

More information

Regulatory Pathways for Licensure and Use of Ebola Virus Vaccines During the Current Outbreak FDA Perspective

Regulatory Pathways for Licensure and Use of Ebola Virus Vaccines During the Current Outbreak FDA Perspective Regulatory Pathways for Licensure and Use of Ebola Virus Vaccines During the Current Outbreak FDA Perspective Office of Vaccines Research and Review Center for Biologics Evaluation and Research U.S. Food

More information

Guidance for Industry

Guidance for Industry Guidance for Industry Scientific Considerations in Demonstrating Biosimilarity to a Reference Product DRAFT GUIDANCE This guidance document is being distributed for comment purposes only. Comments and

More information

PX Therapeutics : the partner for early stage biotherapeutics development Biotuesday, May 5 2009

PX Therapeutics : the partner for early stage biotherapeutics development Biotuesday, May 5 2009 PX Therapeutics : the partner for early stage biotherapeutics development Biotuesday, May 5 2009 Christelle Dagoneau, PhD Business Development Director Company Profile Protein expert incorporated in 2000

More information

ICH Topic S 1 A The Need for Carcinogenicity Studies of Pharmaceuticals. Step 5

ICH Topic S 1 A The Need for Carcinogenicity Studies of Pharmaceuticals. Step 5 European Medicines Agency July 1996 CPMP/ICH/140/95 ICH Topic S 1 A The Need for Carcinogenicity Studies of Pharmaceuticals Step 5 NOTE FOR GUIDANCE ON THE NEED FOR CARCINOGENICITY STUDIES OF PHARMACEUTICALS

More information

Not All Clinical Trials Are Created Equal Understanding the Different Phases

Not All Clinical Trials Are Created Equal Understanding the Different Phases Not All Clinical Trials Are Created Equal Understanding the Different Phases This chapter will help you understand the differences between the various clinical trial phases and how these differences impact

More information

MRC Technology Centre for Therapeutics Discovery

MRC Technology Centre for Therapeutics Discovery MRC Technology Centre for Therapeutics Discovery fast-tracking discovery and development of novel drugs from academia Dr Duncan Young Business Development Manager UbiFrance Symposium, October 2012 established

More information

Summary of the risk management plan (RMP) for Accofil (filgrastim)

Summary of the risk management plan (RMP) for Accofil (filgrastim) EMA/475472/2014 Summary of the risk management plan (RMP) for Accofil (filgrastim) This is a summary of the risk management plan (RMP) for Accofil, which details the measures to be taken in order to ensure

More information

FDA approves Rituxan/MabThera for first-line maintenance use in follicular lymphoma

FDA approves Rituxan/MabThera for first-line maintenance use in follicular lymphoma Media Release Basel, 31 January 2011 FDA approves Rituxan/MabThera for first-line maintenance use in follicular lymphoma Approval provides option that improves the length of time people with incurable

More information

Guideline on similar biological medicinal products containing biotechnology-derived proteins as active substance: non-clinical and clinical issues

Guideline on similar biological medicinal products containing biotechnology-derived proteins as active substance: non-clinical and clinical issues 18 December 2014 EMEA/CHMP/BMWP/42832/2005 Rev1 Committee for Medicinal Products for Human Use (CHMP) Guideline on similar biological medicinal products containing biotechnology-derived proteins as active

More information

Roles & Responsibilities of the Sponsor

Roles & Responsibilities of the Sponsor Roles & Responsibilities of the Sponsor Developed by Center for Cancer Research, National Cancer Institute, NIH Endorsed by the CTN SIG Leadership Group Objectives Funding for clinical research comes from

More information

Medicines for Psoriatic Arthritis. A Review of the Research for Adults

Medicines for Psoriatic Arthritis. A Review of the Research for Adults Medicines for Psoriatic Arthritis A Review of the Research for Adults Is This Information Right for Me? Yes, this information is right for you if: Your doctor* has told you that you have psoriatic (pronounced

More information

Global Monoclonal Antibodies Pipeline Insight 2015

Global Monoclonal Antibodies Pipeline Insight 2015 Brochure More information from http://www.researchandmarkets.com/reports/3113388/ Global Monoclonal Antibodies Pipeline Insight 2015 Description: Ever since the Nobel Prize was bestowed on the person who

More information

PharmaceuticalDevelopment

PharmaceuticalDevelopment PharmaceuticalDevelopment Activities & Timelines Agenda Factors driving development Types of product Timelines for development Factors affecting length of development Regulation of Pharma environment Cost

More information

BioBusiness Biopharmaceutical Ingredients Recombinant albumins & albumin fusion Dermot Pearson, Strategic Product Director, NZ Biopharma

BioBusiness Biopharmaceutical Ingredients Recombinant albumins & albumin fusion Dermot Pearson, Strategic Product Director, NZ Biopharma BioBusiness Biopharmaceutical Ingredients Recombinant albumins & albumin fusion Dermot Pearson, Strategic Product Director, NZ Biopharma 2 Novozymes solutions dedicated to the biopharmaceutical industry

More information

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) REFLECTION PAPER

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) REFLECTION PAPER European Medicines Agency London 23 April 2009 EMEA/CHMP/BMWP/102046/2006 COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) REFLECTION PAPER NON-CLINICAL AND CLINICAL DEVELOPMENT OF SIMILAR MEDICINAL

More information

Cancer Monoclonal Antibodies Market Forecast to 2017

Cancer Monoclonal Antibodies Market Forecast to 2017 RNCOS Page 1 Table of Contents 1. Analyst View 2. Research Methodology 3. The Global Burden of Cancer 3.1 Cancer - A Leading Cause of Death 3.2 Current and Future Cancer Cases: By Type and Geography 3.3

More information

BIOSIMILARS A COMPLETE DEVELOPMENT PLATFORM

BIOSIMILARS A COMPLETE DEVELOPMENT PLATFORM BIOSIMILARS A COMPLETE DEVELOPMENT PLATFORM FROM ORIGINATOR THROUGH COMPARABILITY TO MARKET STREAMLINE YOUR R&D, REGULATORY, ANALYTICAL, AND CLINICAL NEEDS WITH ONE PROVIDER The development pathway of

More information

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP)

COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) European Medicines Agency Evaluation of Medicines for Human Use London, 10 October 2007 Doc. Ref. COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP) GUIDELINE ON POTENCY TESTING OF CELL BASED IMMUNOTHERAPY

More information

Subject: Tecfidera (dimethyl fumarate): new measures to minimise the risk of PML enhanced monitoring and stopping rules

Subject: Tecfidera (dimethyl fumarate): new measures to minimise the risk of PML enhanced monitoring and stopping rules Date: 25 th November 2015 Subject: Tecfidera (dimethyl fumarate): new measures to minimise the risk of PML enhanced monitoring and stopping rules Dear Healthcare Professional In agreement with the European

More information

Manufacturer of drug substance

Manufacturer of drug substance Original s and Biosimilars (marketing authorizations in the EU) Date: January 2016 1) Somatropin s ORIGINAL PRODUCTS: Authorization Genentech Genotropin Somatropin Pharmacia/ Pfizer E. coli Humatrope Somatropin

More information

Overview of Drug Development: the Regulatory Process

Overview of Drug Development: the Regulatory Process Overview of Drug Development: the Regulatory Process Roger D. Nolan, PhD Director, Project Operations Calvert Research Institute November, 2006 Adapted from course taught by Cato Research Background: Roger

More information

As featured in. Orphan disease specialists find home with Big Pharma.

As featured in. Orphan disease specialists find home with Big Pharma. Orphan disease specialists find home with Big Pharma. By Maura Musciacco Senior Analyst January 2013 Tumbling over the patent cliff, many big pharma companies are being forced to find new strategies for

More information

THE BIOTECH & PHARMACEUTICAL INDUSTRY

THE BIOTECH & PHARMACEUTICAL INDUSTRY THE BIOTECH & PHARMACEUTICAL INDUSTRY ESSENTIAL CAREERS INFORMATION CALUM LECKIE KATIE BISARO CAREERS CONSULTANTS What we will cover Sector overview Types of role Graduate recruitment trends and issues

More information

specialty pharmacy summit 2016

specialty pharmacy summit 2016 Specialty Pharmaceuticals: What s In? What s Out & What s Ahead? Doug Long VP Industry Relations IMS Health Asembia May 4, 2016 0 US Spending increased 12.2% to $424.8Bn in 2015 Spending increased $46.2Bn

More information

From the Text. Definitions. Definitions. Definitions. Definitions. RSPT 2217 Basic Concepts & Principles. RSPT 2217-01 Basic Concepts

From the Text. Definitions. Definitions. Definitions. Definitions. RSPT 2217 Basic Concepts & Principles. RSPT 2217-01 Basic Concepts RSPT 2217 & Principles Gardenhire Chapter 1; p. 1-10 From the Text Key Terms and page 2 Orphan Drugs Table 1-1; page 6 Abbreviations Table 1-2; page 8 Aerosolized Agents Table 1-3; page 9 Drug: Pharmacology:

More information

Draft Agreed by BMWP October Adoption by CHMP for release for consultation 17 November 2011

Draft Agreed by BMWP October Adoption by CHMP for release for consultation 17 November 2011 21 February 2013 EMA/CHMP/BMWP/671292/2010 Committee for Medicinal Products for Human Use (CHMP) Guideline on non-clinical and clinical development of similar biological medicinal products containing recombinant

More information

Biotechnology Forum 2015

Biotechnology Forum 2015 Biotechnology Forum 2015 WHO Guidelines on scientific principles for regulatory risk assessment of biotherapeutic products Mexico, 23 rd February 2015 Dr Ivana Knezevic, WHO/HIS/EMP 1 Outline WHO International

More information

MAB Solut. MABSolys Génopole Campus 1 5 rue Henri Desbruères 91030 Evry Cedex. www.mabsolut.com. is involved at each stage of your project

MAB Solut. MABSolys Génopole Campus 1 5 rue Henri Desbruères 91030 Evry Cedex. www.mabsolut.com. is involved at each stage of your project Mabsolus-2015-UK:Mise en page 1 03/07/15 14:13 Page1 Services provider Department of MABSolys from conception to validation MAB Solut is involved at each stage of your project Creation of antibodies Production

More information

WHO guideline for abbreviated licensing pathways for certain biological therapeutic products

WHO guideline for abbreviated licensing pathways for certain biological therapeutic products WHO guideline for abbreviated licensing pathways for certain biological therapeutic products - Clinical evaluation - Martina Weise, MD Federal Institute for Drugs and Medical Devices, Germany General considerations

More information

LFB GROUP & SANOFI combine their bioproduction capabilities to provide integrated CMO services for biopharmaceuticals

LFB GROUP & SANOFI combine their bioproduction capabilities to provide integrated CMO services for biopharmaceuticals LFB GROUP & SANOFI combine their bioproduction capabilities to provide integrated CMO services for biopharmaceuticals MAbLaunch TM is a joint bioproduction platform combining LFB Biomanufacturing (LFB

More information

Guideline on similar biological medicinal products containing biotechnology-derived proteins as active substance: non-clinical and clinical issues

Guideline on similar biological medicinal products containing biotechnology-derived proteins as active substance: non-clinical and clinical issues 1 2 3 03 June May 2013 EMEA/CHMP/BMWP/42832/2005 Rev. 1 Committee for Medicinal Products for Human Use (CHMP) 4 5 6 7 Guideline on similar biological medicinal products containing biotechnology-derived

More information

MEDICATION GUIDE. ACTEMRA (AC-TEM-RA) (tocilizumab) Solution for Intravenous Infusion

MEDICATION GUIDE. ACTEMRA (AC-TEM-RA) (tocilizumab) Solution for Intravenous Infusion MEDICATION GUIDE ACTEMRA (AC-TEM-RA) (tocilizumab) Solution for Intravenous Infusion ACTEMRA (AC-TEM-RA) (tocilizumab) Injection, Solution for Subcutaneous Administration Read this Medication Guide before

More information

The Clinical Trials Process an educated patient s guide

The Clinical Trials Process an educated patient s guide The Clinical Trials Process an educated patient s guide Gwen L. Nichols, MD Site Head, Oncology Roche TCRC, Translational and Clinical Research Center New York DISCLAIMER I am an employee of Hoffmann-

More information

Methotrexate reduces immunogenicity in adalimumab treated rheumatoid arthritis patients in a dose-dependent manner

Methotrexate reduces immunogenicity in adalimumab treated rheumatoid arthritis patients in a dose-dependent manner 8b Methotrexate reduces immunogenicity in adalimumab treated rheumatoid arthritis patients in a dose-dependent manner Charlotte Krieckaert Mike Nurmohamed Gertjan Wolbink ANNALS OF RHEUMATIC DISEASES Vol.

More information

A Generic Kit-Based Approach for Quantifying Monoclonal Antibody Drugs Through Direct Digestion of Discovery Study Samples

A Generic Kit-Based Approach for Quantifying Monoclonal Antibody Drugs Through Direct Digestion of Discovery Study Samples A Generic Kit-Based Approach for Quantifying Monoclonal Antibody Drugs Through Direct Digestion of Discovery Study Samples Mary Lame, Hua Yang, Sherri Naughton, and Erin Chambers Waters Corporation, Milford,

More information

BIOTECHNOLOGY OPERATIONS

BIOTECHNOLOGY OPERATIONS BIOTECHNOLOGY OPERATIONS Principles and Practices Michael J. Roy TECHNISCHE INFORMATION SBIBLIOTHEK UNIVERSITATSBIBLIOTHEK HANNOVER CRC Press TaylorStFrancis Croup Boca Raton London New York CRC Press

More information

Rheumatoid Arthritis

Rheumatoid Arthritis Rheumatoid Arthritis While rheumatoid arthritis (RA) has long been feared as one of the most disabling types of arthritis, the outlook has dramatically improved for many newly diagnosed patients. Certainly

More information

A Case for Cure: Drug Portfolio Analysis. Team: PharmaKings Anirvan Chaudhuri Neha Madan Xiao Huang Jason Park Anjai Lal

A Case for Cure: Drug Portfolio Analysis. Team: PharmaKings Anirvan Chaudhuri Neha Madan Xiao Huang Jason Park Anjai Lal A Case for Cure: Drug Portfolio Analysis Team: PharmaKings Anirvan Chaudhuri Neha Madan Xiao Huang Jason Park Anjai Lal Executive Summary Our team recommends. A Recombinant Protein for treatment of Multiple

More information

Remicade (infliximab)

Remicade (infliximab) Criteria for approval Commercial Only Remicade (infliximab) Background: Tumor Necrosis Factor (TNF) alpha is one of two proinflammatory cytokines that appear to play a dominant role in the inflammatory

More information