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


The Global Orphan Diseases Partnering 2010-2023: Deal trends, players and financials report provides comprehensive understanding and unprecedented access to the orphan diseases partnering deals and agreements entered into by the worlds leading healthcare companies.

Publication date
May 2023
Number of pages
Product type
Research report
Available formats
PDF document
Report edition

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?

Global Orphan Diseases Partnering 2010 to 2023 provides the reader with the following key benefits:


  • In-depth understanding of orphan diseases deal trends since 2010
  • Access orphan diseases deal headline, upfront, milestone and royalty data
  • Research hundreds of actual contracts between orphan diseases partner companies
  • Comprehensive access to over 300 links to actual orphan diseases deals entered into by the world’s biopharma companies
  • Indepth review of orphan diseases deals entered into by the top 25 most active dealmakers
  • Benchmark the key deal terms companies have agreed in previous deals
  • Identify key terms under which companies partner orphan diseases opportunities
  • Uncover companies actively partnering orphan diseases opportunities

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

2.7. Orphan diseases partnering by therapeutic indication


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




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


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Current Partnering is part of Wildwood Ventures Ltd., based in York, United Kingdom. We have been trading successfully online since 2009 and work with some of the most famous brands in the business. We carefully select our partners to ensure that the publications meet expectations of demanding customers.

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

Current Partnering reports provide insight into the trends and terms of partnering deals in the global life sciences sector.

Current Partnering reports are updated every six months to ensure the user has access to the latest announcements and trends in the topic focus of the report.

Current Partnering analysts review the deal data to provide an overview and analysis of deal trends, including example deals and terms.

Current Partnering reports source deal data from our proprietary deals and alliances database, Current Agreements. The database is updated daily by our analysts with new deals as they are announced globally by the company’s party to the deal. In addition, deal records are updated with new data as it becomes available.

The data in the Current Agreements deals and alliances database is obtained from secondary sources such as publicly available industry sources including press releases, company presentations, investor presentations, company SEC filings, other company filings, company websites, conference presentations. Sources are identified to allow for user verification.

Current Partnering provides comprehensive coverage of the following partnering or deal types:
• Asset purchase
• Assignment
• Co-development
• Co-market
• Co-promotion
• Collaborative R&D
• Contract service
• Cross-licensing
• Development
• Distribution
• Equity purchase
• Evaluation
• Grant
• Joint venture
• Licensing
• Loan
• Manufacturing
• Marketing
• Option
• Promotion
• Research
• Royalty financing
• Settlement
• Spin out
• Sub license
• Supply
• Termination
• Warrant

Every deal record is fully categorized and includes the following data, where available:
• Industry sector
• Therapy areas
• Technology type
• Deal components
• Financial terms
• Stage of development
• Exclusivity
• Asset type
• Geographic focus
• Excluded geography
• Company press release
• SEC filing data including contract document

All financial amounts are converted to US$ using the exchange rate available on the date of deal announcement, enabling direct comparison of deal terms across international territories.

Financial data and contract documents displayed in deal records is obtained from public sources, where disclosed by the parties to the deal.