Global Orphan Diseases Partnering 2010-2023: Deal trends, players and financials

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Global Orphan Diseases Partnering 2010 to 2023 provides the full collection of orphan diseases disease deals signed between the world’s pharmaceutical and biotechnology companies since 2010.


Trends in orphan diseases partnering deals
Financial deal terms for headline, upfront and royalty by stage of development
Orphan diseases partnering agreement structure
Orphan diseases partnering contract documents
Top orphan diseases deals by value
Most active orphan diseases dealmakers


Most of the deals included within the report occur when a licensee obtains a right or an option right to license a licensor’s product or technology. More often these days these deals tend to be multi-component including both a collaborative R&D and a commercialization of outcomes element.

The report takes readers through the comprehensive orphan diseases disease deal trends, key players and top deal values allowing the understanding of how, why and under what terms companies are currently entering orphan diseases deals.

The report presents financial deal terms values for orphan diseases deals, where available listing by overall headline values, upfront payments, milestones and royalties enabling readers to analyse and benchmark the value of current deals.

The initial chapters of this report provide an orientation of orphan diseases dealmaking trends.

Chapter 1 provides an introduction to the report.

Chapter 2 provides an overview of the trends in orphan diseases dealmaking since 2010 covering trends by year, deal type, stage of development, technology type and therapeutic indication.

Chapter 3 includes an analysis of financial deal terms covering headline value, upfront payment, milestone payments and royalty rates.

Chapter 4 provides a review of the leading orphan diseases deals since 2010. Deals are listed by headline value. The chapter includes the top 25 most active orphan diseases dealmakers, together with a full listing of deals to which they are a party. Where the deal has an agreement contract published at the SEC a link provides online access to the contract.

Chapter 5 provides comprehensive access to orphan diseases deals since 2010 where a deal contract is available, providing the user with direct access to contracts as filed with the SEC regulatory authorities. Each deal title links via Weblink to an online version of the deal record contract document, providing easy access to each contract document on demand.

Chapter 6 provides a comprehensive directory of all orphan diseases partnering deals by specific orphan diseases target announced since 2010. The chapter is organized by specific orphan diseases therapeutic target. Each deal title links via Weblink to an online version of the deal record and where available, the contract document, providing easy access to each contract document on demand.

In addition, a comprehensive appendix is provided with each report of all orphan diseases partnering deals signed and announced since 2010. The appendices are organized by company A-Z, stage of development at signing, deal type (collaborative R&D, co-promotion, licensing etc) and technology type. Each deal title links via Weblink to an online version of the deal record and where available, the contract document, providing easy access to each contract document on demand.

The report also includes numerous tables and figures that illustrate the trends and activities in orphan diseases partnering and dealmaking since 2010.

In conclusion, this report provides everything a prospective dealmaker needs to know about partnering in the research, development and commercialization of orphan diseases technologies and products.


Report scope

Global Orphan Diseases Partnering 2010 to 2023 is intended to provide the reader with an in-depth understanding and access to orphan diseases trends and structure of deals entered into by leading companies worldwide.

Global Orphan Diseases Partnering 2010 to 2023 includes:

Trends in orphan diseases dealmaking in the biopharma industry since 2010
Access to headline, upfront, milestone and royalty data
Access to hundreds of orphan diseases deal contract documents
Comprehensive access to over 300 orphan diseases deal records
The leading orphan diseases deals by value since 2010
Most active orphan diseases dealmakers since 2010


In Global Orphan Diseases Partnering 2010 to 2023, available deals and contracts are listed by:

Headline value
Upfront payment value
Royalty rate value
Stage of development at signing
Deal component type
Technology type
Specific therapy indication


Each deal title links via Weblink to an online version of the deal record and where available, the contract document, providing easy access to each contract document on demand.

The Global Orphan Diseases Partnering 2010-2023 report provides comprehensive access to available deals and contract documents for over 300 orphan diseases deals. Analyzing actual contract agreements allows assessment of the following:

What are the precise rights granted or optioned?
What is actually granted by the agreement to the partner company?
What exclusivity is granted?
What is the payment structure for the deal?
How are sales and payments audited?
What is the deal term?
How are the key terms of the agreement defined?
How are IPRs handled and owned?
Who is responsible for commercialization?
Who is responsible for development, supply, and manufacture?
How is confidentiality and publication managed?
How are disputes to be resolved?
Under what conditions can the deal be terminated?
What happens when there is a change of ownership?
What sublicensing and subcontracting provisions have been agreed?
Which boilerplate clauses does the company insist upon?
Which boilerplate clauses appear to differ from partner to partner or deal type to deal type?
Which jurisdiction does the company insist upon for agreement law?

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Executive Summary

Chapter 1 – Introduction

Chapter 2 – Trends in orphan diseases dealmaking
2.1. Introduction
2.2. Orphan diseases partnering over the years
2.3. Orphan diseases partnering by deal type
2.4. Orphan diseases partnering by industry sector
2.5. Orphan diseases partnering by stage of development
2.6. Orphan diseases partnering by technology type

Chapter 3 – Financial deal terms for orphan diseases partnering
3.1. Introduction
3.2. Disclosed financials terms for orphan diseases partnering
3.3. Orphan diseases partnering headline values
3.4. Orphan diseases deal upfront payments
3.5. Orphan diseases deal milestone payments
3.6. Orphan diseases royalty rates

Chapter 4 – Leading orphan diseases deals and dealmakers

4.1. Introduction
4.2. Most active in orphan diseases partnering
4.3. List of most active dealmakers in orphan diseases
4.4. Top orphan diseases deals by value

Chapter 5 – Orphan diseases contract document directory

5.1. Introduction
5.2. Orphan diseases partnering deals where contract document available

Chapter 6 – Orphan diseases dealmaking by therapeutic target
6.1. Introduction
6.2. Deals by orphan diseases therapeutic target

Appendices
Appendix 1 – Directory of orphan diseases deals by company A-Z since 2010

Appendix 2 – Directory of orphan diseases deals by deal type since 2010

Appendix 3 – Directory of orphan diseases deals by stage of development since 2010

Appendix 4 – Directory of orphan diseases deals by technology type since 2010

Further reading on dealmaking

Deal type definitions

About Wildwood Ventures

Current Partnering
Current Agreements
Recent report titles from CurrentPartnering


Table of figures
Figure 1: Orphan diseases partnering since 2010
Figure 2: Orphan diseases partnering by deal type since 2010
Figure 3: Orphan diseases partnering by industry sector since 2010
Figure 4: Orphan diseases partnering by stage of development since 2010
Figure 5: Orphan diseases partnering by technology type since 2010
Figure 6: Orphan diseases partnering by indication since 2010
Figure 7: Orphan diseases deals with a headline value
Figure 8: Orphan diseases deals with upfront payment values
Figure 9: Orphan diseases deals with milestone payment
Figure 10: Orphan diseases deals with royalty rates
Figure 11: Active orphan diseases dealmaking activity since 2010
Figure 12: Top orphan diseases deals by value since 2010

AAVLife, Abbvie, Abiogen Pharma, AbMed, Abpro, Acceleron Pharma, Accelovance, Aceragen, ACMG Foundation for Genetic and Genomic Medicine, Acorda Therapeutics, Actelion, Adalvo, Adamas Pharmaceuticals, Advanced Accelerator Applications, Advaxis, Aegerion Pharmaceutical, Aevi Genomic Medicine, AFT Pharmaceuticals, Agility Clinical, Aires Pharmaceuticals, Akari Therapeutics, Akcea Therapeutics, Alcyone Lifesciences, Alexion Pharmaceuticals, Alitair Pharmaceuticals, Allergan, Almirall, Alnylam Pharmaceuticals, Amarantus BioSciences, American National Multiple Sclerosis Society, American Red Cross, AmerisourceBergen, Amicus Therapeutics, Amneal Pharmaceuticals, AmorChem, Amryt Pharma, Andromeda Biotech, AndroScience, ANI Pharmaceuticals, AOP Orphan Pharmaceuticals, Aperion Biologics, Apic Bio, Applied Genetic Technologies, Aprecia Pharmaceuticals, Aptuit, Aradigm, Arbutus, Arctic Therapeutics, Arcturus Therapeutics, Arecor, Aridis Pharmaceuticals, ArmaGen, Arphio, Array Biopharma, Asceneuron, Aslan Pharma, Astellas Pharma, AstraZeneca, Atlas Venture, Audentes Therapeutics, Auven Therapeutics, Avanzanite Bioscience, Baxalta, Beijing Double-Crane Pharmaceutical, Bellus Health, Benuvia Therapeutics, BioAlliance Pharma, BioCryst Pharmaceuticals, BioMarin Pharmaceutical, BioNTech, Bionure, BioPontis Alliance, BioSante Pharmaceuticals, BioSoteria, BioTie Therapies, BioXcel, Blood Systems, Bpifrance, BTG, Buck Institute for Age Research, California Institute for Biomedical Research, Calliditas Therapeutics, Camurus, Canadian Organization for Rare Disorders, Cancer Prevention Pharmaceuticals, Castle Creek Biosciences, Catabasis Pharmaceuticals, Catalent, Catalyst Pharmaceutical Partners, CBR International, CellAct, Cell Therapeutics, Celsion, Celsus Therapeutics, Censa Pharmaceuticals, Centocor, Centogene, Cerenis Therapeutics, Cerus, ChemoCentryx, Chiesi Farmaceutici, Children's Hospital Boston, Children's Hospital of Philadelphia, Chord Therapeutics, Chroma Therapeutics, Chugai Pharma Marketing, Clal Biotech, Clalit Health Fund, Clinigen, Cloud Pharmaceuticals, CMC Biologics, CMIC, CNS Pharmaceuticals, Confo Therapeutics, Corino Therapeutics, Cortendo, Crealta Pharmaceuticals, Critical Path Institute (C-Path), CSL Behring, Cumberland Pharmaceuticals, Cutaneous Lymphoma Foundation, Cycle Pharmaceuticals, Cyclica, Cystic Fibrosis Foundation, Cystinosis Research Foundation, Cytokinetics, D&A Pharma, DanDrit Biotech, DBV Technologies, Delta-Vir, Department of Defense, Dicerna Pharmaceuticals, Diurnal, DNAtrix, Dohmen, Drugs for Neglected Diseases Initiative, DuoCort Pharma, Edico Genome, Edimer Pharmaceuticals, Edison Pharmaceuticals, Edmond Pharma, EffRx, Eiger BioPharmaceuticals, Elan, Elixirgen, ElsaLys Biotech, Eluminex Biosciences, EMD Serono, Enable Injections, Engrail Therapeutics, EpiCept, EpiDestiny, Epirium Bio, Epivax, Erytech, Eureka Eurostars, European Commission, EUSA Pharma, Evotec, ExCEEd Orphan, Eyevance, Fast Forward, Fibrocell Science, Florida Biologix, Food and Drug Administration (FDA), Fortress Biotech, Foundation Fighting Blindness, Foundation For Angelman Syndrome Therapeutics, Fujifilm Pharma, G-treeBNT, Galena Biopharma, GBS-CIDP Foundation, Genethon, Genevant Sciences, Genomics England, Gentium, Genzyme, Gilead Sciences, Ginkgo BioWorks, GlaxoSmithKline, Global Genes, GlycoMimetics, Great Ormond Street Hospital (GOSH), Grifols, Grupo Ferrer, GtreeBNT, Halozyme Therapeutics, Harvard Medical School, Harvard University, Healthcare Royalty Partners, Hematech, HLB Therapeutics, HLS Therapeutics, Horizon Pharma plc, Houston Pharmaceuticals, Hy BioPharma, Hyperion Therapeutics, iBio, Icahn School of Medicine at Mount Sinai, Idera Pharmaceuticals, Ikano Therapeutics, Ikaria, Imaxio, ImmuneWorks, Imperial College London, In-Depth Genomics, InformedDNA, Inhibrx, Institute for the Promotion of Innovation by Science and Technology in Flanders, Intellect Neurosciences, InterMune, Invida Pharmaceuticals, Ionis Pharmaceuticals, Ipsen, Isomerase Therapeutics, Israeli National Authority for Technological Innovation, Ivax, Jackson Laboratory, Janssen Sciences, Jazz Pharmaceuticals, JCR Pharmaceuticals, John P. Hussman Foundation, Johnson & Johnson, Jupiter Orphan Therapeutics, Kadmon Pharmaceuticals, Kalon Biotherapeutics, Kalytera Therapeutics, Kamada, Kashiv BioSciences, Kentucky Bioprocessing, Kings College London, Kyowa Hakko Kirin, La Jolla Labs, Lee's Pharmaceutical, Leiden University, Lenus Therapeutics, Leukaemia & Lymphoma Research, Lexicon Pharmaceuticals, LFB Biomedicaments, LifeArc, LifeMax Laboratories, Ligand Pharmaceuticals, Longitude Capital, Lotus Tissue Repair, Lumena Pharmaceuticals, Lumos Pharma, Lumos Pharma (acquired), Lung Biotechnology, Mallinckrodt Pharmaceuticals, Marnier-Lapostolle Foundation, Massachusetts General Hospital, Mast Therapeutics, Meda, Medgenics, Medicenna Therapeutics, MedImmune, Medipal Holdings, MediWound, Medosome Biotec, Medunik Canada, Merck and Co, Mereo BioPharma, Mesoblast, Microsoft, Military Pharmaceutical Laboratory, Mills Pharmaceuticals, Minoryx Therapeutics, Mirum Pharmaceuticals, Mitsubishi Tanabe Pharma, Moulder Center for Drug Discovery Research, MSD Action Foundation (MSDAF), Mundipharma, Mustang Bio, n-Lorem Foundation, Nacuity Pharmaceuticals, NanoSonic Products, National Cancer Institute, National Health Research Institutes, National Institute of Diabetes and Digestive and Kidney Diseases, National Institute of Neurological Disorders and Stroke, National Institutes of Health, National Organization for Rare Disorders, Nestle Health Science, Neuraxpharm, Neurotrope BioScience, NeuroVive Pharmaceutical, Newron Pharmaceuticals, NIH Undiagnosed Diseases Program, Nimbus Apollo, Nippon Chemiphar, NMI TT Pharmaservices, Novartis, Novasep, NovelMed, Noventia Pharma, NovImmune, Novo Nordisk, OcuNexus Therapeutics, OncoPep, OncoSec Medical, Oncosynergy, Onxeo, Ophthotech, OPKO Health, Oraxion Therapeutics, Orchard Therapeutics, OrphanDev, Orphan Europe, OrphanPacific, Orphan Therapeutics, Orphatec Pharmaceuticals, Orphazyme, Orphic Therapeutics, Oryzon, OSI Pharmaceuticals, Oxford BioMedica, Parent Project Muscular Dystrophy, PellePharm, Penn State Research Foundation, PerkinElmer, Perlara, Pfizer, PharmaMar, Pharming Group, Pharnext, Piramal, Plexcera, PoC Capital, Population Diagnostics, Presidio Pharmaceuticals, Progeria Research Foundation, Prometic Life Sciences, ProQR Therapeutics, Proteostasis Therapeutics, ProThera, PSR Orphan Drug Experts, PTC Therapeutics, Qiagen, Queen Mary University of London, R-Tech Ueno, Radius Health, Ramot at Tel Aviv University, Rare Disease Company Coalition, Rare Expertise, RCC Pharma, Receptos, Recipharm, Recursion, Regenerx, Regenxbio, ReGenX Biosciences, Regulus Therapeutics, Replica Analytics, Repligen, Resverlogix, Retinagenix, Retrophin, RetroSense Therapeutics, Rett Syndrome Research Trust, Rezolute, Rhythm Pharmaceuticals, Rigi Healthcare, Rigi Orphan, Roche, Rocket Pharmaceuticals, S*Bio, Sagent Pharmaceuticals, Saint Louis University, Sanofi, Savara Pharma, Savient Pharmaceuticals, Scioderm, SCOUT, Selecta Biosciences, Selexis, Sentinel Oncology, Seres Therapeutics, Shenzhen Hepalink Pharmaceutical, Shire Human Genetic Therapies, Shire Laboratories, Shire Pharmaceuticals, SIFI (Societa Industria Farmaceutica Italiana), Sirenas, Sirtex Medical, SK Biopharmaceuticals, Sol-Gel, Soleno Therapeutics, Soligenix, SOM Biotech, Spark Therapeutics, SpePharm, Sperogenix Therapeutics, Spooner Girls Foundation, St. Jude Children's Research Hospital, STADA Arzneimittel, Stanford University, Stealth BioTherapeutics, Stealth Therapeutics, Sucampo AG, Sucampo Pharmaceuticals, Sumitomo Dainippon Pharma, Swedish Orphan, Swedish Orphan Biovitrum, Swixx Biopharma, SymbioTec, Synageva BioPharma, SynCo Bio Partners, Taisho Pharmaceutical, Takeda America Holdings, Takeda Pharmaceutical, Tamid Bio, Taysha Gene Therapies, Teva Pharmaceutical Industries, Texas A&M University, Therapyx, Theratechnologies, Tillotts Pharma, Timber Pharmaceuticals, TNI BioTech, TopoTarget, Translational Medicine Accelerator, Turgut Ilaclari, TWi Biotechnology, twoXAR, Tyris Therapeutics, Ucyclyd Pharma, UH Harrington Discovery Institute, Ultragenyx Pharmaceuticals, UniQure, Universite Laval, University College Dublin, University College London, University of Birmingham, University of California Irvine, University of California Los Angeles, University of Cambridge, University of Florida, University of Manchester, University of North Carolina, University of Oxford, University of Pennsylvania, University of Pennsylvania Center for Orphan Disease Research and Therapy, University of Quebec, University of Southern California, Upsher-Smith, UT Southwestern Medical Center, Valeant Pharmaceuticals, Vanderbilt University, Vaxil BioTherapeutics, Veeva Systems, Vericel, VIB, Videregen, Viela Bio, Vifor Pharma, Viking Therapeutics, Viropharma, Vivalis, Vivet Therapeutics, Volution Immuno Pharma, Vtesse, Wellstat Therapeutics, Winhealth Pharma Group, World Orphan Drug Alliance, WuXi Biologics, X-chem, Xenetic Biosciences, Xenon Pharmaceuticals, XERIS Pharmaceuticals, Xoma, Yungjin Pharmaceutical, YuYang DNU, Zacharon Pharmaceuticals, Zhejiang Hisun Pharmaceutical

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