Company lists · Data refreshed September 2026
Top 25 Orphan drug companies worldwide
RevenueBase identified 1,083 company matches for orphan drug companies worldwide as of September 2026 and tracks 219,743 professional contacts across them, including 83,769 with verified work emails. The United States is the largest market and London the largest hub.
This list covers company matches for orphan drug companies worldwide, found by RevenueBase SmartSearch using the meaning of business descriptions in its company graph at Exact. Ranked by professionals tracked, largest first. Rows show recorded descriptions where available, Exact search membership and the snapshot date. Data refreshed September 20, 2026. Country and city lists for orphan drug companies follow the table.
Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase SmartSearch, the natural-language company search RevenueBase customers use to build their own targeted lists and unlock verified emails and direct dials at the companies on them. How these lists are built and judged.
The 25 largest orphan drug companies worldwide
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 29,414 contacts → | ||||
Exact match “We all want a healthier future.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 2 | 26,637 contacts → | ||||
Exact match “At Johnson & Johnson Innovative Medicine, we innovate with purpose, to lead where medicine is going.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 3 | 18,228 contacts → | ||||
Exact match “Syneos Health® is a leading fully integrated biopharmaceutical solutions organization built to accelerate customer success.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 4 | 17,217 contacts → | ||||
Exact match “About Genentech We're passionate about finding solutions for people facing the world's most difficult-to-treat conditions.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 5 | 16,043 contacts → | ||||
Exact match “At Regeneron we believe that when the right idea finds the right team, powerful change is possible.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 6 | 10,958 contacts → | ||||
Exact match “Servier is an independent international pharmaceutical group governed by a foundation.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 7 | 6,136 contacts → | ||||
Exact match “Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 8 | 5,943 contacts → | ||||
Exact match “We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 9 | 3,943 contacts → | ||||
Exact match “Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 10 | 3,644 contacts → | ||||
Exact match “Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 11 | 3,593 contacts → | ||||
Exact match “Jazz Pharmaceuticals plc (Nasdaq: JAZZ), is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 12 | 2,789 contacts → | ||||
Exact match “Recordati has been at the forefront of life-enhancing and life-changing medicines for 100 years.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 13 | 2,570 contacts → | ||||
Exact match “At Eisai Inc, human health care (hhc) is our goal.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 14 | 1,781 contacts → | ||||
Exact match “Precision for Medicine is an industry-leading global clinical research services organization, purpose-built to improve the clinical research and development process for new…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 15 | 1,778 contacts → | ||||
Exact match “Novotech is a globally recognized full-service clinical research organization (CRO) and scientific advisory company that provides biotech and small- to mid-sized pharma companies…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 16 | 1,601 contacts → | ||||
Exact match “Hetero Healthcare Ltd is a leading Innovative Pharmaceutical Company in India, headquartered at Hyderabad.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 17 | 1,578 contacts → | ||||
Exact match “We aspire to create, improve and prolong life for people living with difficult-to-treat conditions like infertility, multiple sclerosis and cancer.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 18 | 1,561 contacts → | ||||
Exact match “Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 19 | 1,556 contacts → | ||||
Exact match “Leading with purpose.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 20 | 1,512 contacts → | ||||
Exact match “Insmed is a people-rst global biopharmaceutical company striving to deliver rst- and best-in-class therapies to transform the lives of patients facing serious diseases.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 21 | 1,212 contacts → | ||||
Exact match “PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 22 | 1,054 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 23 | 1,043 contacts → | ||||
Exact match “CTI Clinical Trial and Consulting Services is a global, privately held, research service organization, delivering a complete spectrum of clinical trial and consulting services…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 24 | 1,019 contacts → | ||||
Exact match “Bial is a family-owned company with a rich history of over 100 years, dedicated to patient welfare, with products available in more than 50 countries.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 25 | 885 contacts → | ||||
Exact match “Purdue and its subsidiaries develop, manufacture and market medications to meet the evolving needs of healthcare professionals, patients, and caregivers.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
RevenueBase SmartSearch
Describe a niche business in your own words. RevenueBase SmartSearch compares the meaning of your description with company business descriptions compiled in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit.
This list uses Exact for “orphan drug companies”. Each Exact match badge shows membership in that saved search; it is not an individual similarity score or a service-quality rating. Recorded descriptions are shown where available.
Showing 25 of 1,083 orphan drug companies worldwide
See the full list, filter by size, metro and specialty, and access 83,769 tracked professional contacts with verified work emails. Direct dials are available where recorded.
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Orphan drug companies worldwide at a glance
As of Sep 20, 2026Companies by employee count
Email recency is reported on each country list linked below.
How this list was built
The recorded descriptions help you assess these company matches. Check a company’s website for its current services and locations.
- Source
- The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase SmartSearch, the natural-language company search available to every RevenueBase user.
- Coverage
- Every country. Country and city lists for orphan drug companies are linked below.
- What qualifies
- Company records worldwide whose business descriptions match “orphan drug companies” by meaning at the Exact setting. Descriptions are compiled in the RevenueBase company graph from public websites, profiles and company information where available; they are not guaranteed quotations from the company's website.
- RevenueBase SmartSearch
- Describe a niche company in your own words to find businesses by meaning. Exact, Similar, Related and Broad control the breadth of RevenueBase SmartSearch. This saved list uses Exact. Each row's Exact match badge shows inclusion in that search, not an individual score or a service-quality judgment.
- Public preview
- The public preview omits records without a verified work email. Dataset totals and geographic summaries describe the full matched records before these preview filters.
- Match quality
- RevenueBase's August 25, 2026 study assessed 4,198 description/company pairs across matching settings. The publisher reports 86% good or better at Exact. This evaluates the method across queries; it does not audit this individual list. Recorded company descriptions, where available, help you judge each match yourself. Read the full methodology: how the lists are built and what the study measures.
- Ranking
- Team size, largest first: companies are ordered by the number of professionals RevenueBase tracks at each one. Employee ranges shown are reported bands, for context.
- Verification
- Every displayed company carries at least one verified work email. RevenueBase tracks 83,769 professional contacts with verified work emails across the full matched dataset. Contacts re-verify on a rolling cadence, and each contact's own verification date is shown inside RevenueBase.
- Refresh
- This page's figures were pulled from the company graph on September 20, 2026. Lists refresh periodically as the graph is re-checked.
- Record context
- Company records and descriptions can change. Check the company's website for its current services and locations.
Orphan drug companies by location
6 lists · largest firstEvery Orphan drug companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
Frequently asked
What is RevenueBase SmartSearch?
RevenueBase SmartSearch finds niche companies by the meaning of their business descriptions in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit your description. This list uses Exact for “orphan drug companies”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many orphan drug companies are there worldwide?
RevenueBase identified 1,083 company matches for orphan drug companies worldwide as of September 2026, using the meaning of business descriptions at Exact. This is the recorded matched set in the RevenueBase company graph.
What is the largest company on this list?
Takeda tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.
Which country has the most orphan drug companies?
The United States leads with 329 companies, followed by India (88) and the United Kingdom (65). Every orphan drug companies list by country and city is linked on this page.
Which city has the most orphan drug companies?
London leads with 28 company records, followed by Boston (14) and New York (13).
How do I get contact information for these companies?
A free RevenueBase account lets you run this exact search and work limited free results with 500 credits to start. Upgrading provides access to the 83,769 professional contacts with verified work emails tracked across these 1,083 companies; direct dials are available where recorded.
How current is this list?
The list refreshes periodically as the company graph is re-checked. This page's figures were pulled on September 20, 2026.
Ready to reach these companies? Verified emails and direct dials for all 1,083 orphan drug companies worldwide. Start with a free account, no credit card.
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About this data
RevenueBase is a B2B data infrastructure company. Its company graph covers 60 million companies and 398.9 million professional contacts, with emails verified on a rolling cadence rather than scraped once and left to decay. These company lists are generated directly from that graph with RevenueBase SmartSearch, the same natural-language company search every RevenueBase user has: describe a market in plain words, get the companies that match, and add verified emails and direct dials for the people at them. It is the same data that powers RevenueBase's B2B data products, and a free account includes 500 credits to start. How these lists are built and judged.
More company lists
Company intelligence · public preview
Top 25 orphan drug companies worldwide
Find companies in any niche.
Describe the businesses you want in your own words. RevenueBase SmartSearch matches company descriptions by meaning.
RevenueBase records 1,083 matching company records for orphan drug companies worldwide, with 219,743 tracked contacts and 83,769 verified work emails.
This public preview shows 25 selected records ordered by tracked contacts. RevenueBase SmartSearch finds company descriptions that match by meaning at Exact. Snapshot: September 20, 2026.
RevenueBase compiles business descriptions for niche-company discovery; each row shows a recorded excerpt where available. Adapt this search to find the companies and contacts you want.
Top 25 orphan drug companies worldwide
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 |  Takedatakeda.comHQ: Tokyo, IL, JapanReported employees: 10001+ | Tokyo, IL, Japan | 10001+ | Exact match | 29,414 contacts → |
“We all want a healthier future.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | Johnson & Johnson Innovative Medicineinnovativemedicine.jnj.com HQ: Titusville, NJ, United StatesReported employees: 10001+ | Titusville, NJ, United States | 10001+ | Exact match | 26,637 contacts → |
“At Johnson & Johnson Innovative Medicine, we innovate with purpose, to lead where medicine is going.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 |  Syneos Healthsyneoshealth.comHQ: Morrisville, NC, United StatesReported employees: 10001+ | Morrisville, NC, United States | 10001+ | Exact match | 18,228 contacts → |
“Syneos Health® is a leading fully integrated biopharmaceutical solutions organization built to accelerate customer success.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 |  Genentechgene.comHQ: South San Francisco, CA, United StatesReported employees: 10001+ | South San Francisco, CA, United States | 10001+ | Exact match | 17,217 contacts → |
“About Genentech We're passionate about finding solutions for people facing the world's most difficult-to-treat conditions.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 |  Regeneronregeneron.comHQ: Tarrytown, NY, United StatesReported employees: 10001+ | Tarrytown, NY, United States | 10001+ | Exact match | 16,043 contacts → |
“At Regeneron we believe that when the right idea finds the right team, powerful change is possible.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 |  Servierservier.comHQ: Suresnes, FranceReported employees: 10001+ | Suresnes, France | 10001+ | Exact match | 10,958 contacts → |
“Servier is an independent international pharmaceutical group governed by a foundation.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 |  Alexion Pharmaceuticals, INCalexion.comHQ: Boston, MA, United StatesReported employees: 1,001-5,000 | Boston, MA, United States | 1,001-5,000 | Exact match | 6,136 contacts → |
“Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 |  IPSENipsen.comHQ: Paris, FranceReported employees: 5,001-10,000 | Paris, France | 5,001-10,000 | Exact match | 5,943 contacts → |
“We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 |  Biomarin Pharmaceutical INCbiomarin.comHQ: San Rafael, CA, United StatesReported employees: 1,001-5,000 | San Rafael, CA, United States | 1,001-5,000 | Exact match | 3,943 contacts → |
“Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 |  Chiesi Groupchiesi.comHQ: Parma, ItalyReported employees: 5,001-10,000 | Parma, Italy | 5,001-10,000 | Exact match | 3,644 contacts → |
“Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 |  Jazz Pharmaceuticalsjazzpharma.comHQ: Dublin, GermanyReported employees: 1,001-5,000 | Dublin, Germany | 1,001-5,000 | Exact match | 3,593 contacts → |
“Jazz Pharmaceuticals plc (Nasdaq: JAZZ), is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 |  Recordatirecordati.comHQ: Milan, ItalyReported employees: 1,001-5,000 | Milan, Italy | 1,001-5,000 | Exact match | 2,789 contacts → |
“Recordati has been at the forefront of life-enhancing and life-changing medicines for 100 years.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 |  Eisai USeisai.comHQ: Nutley, NJ, United StatesReported employees: 1,001-5,000 | Nutley, NJ, United States | 1,001-5,000 | Exact match | 2,570 contacts → |
“At Eisai Inc, human health care (hhc) is our goal.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 |  Precision For Medicineprecisionformedicine.comHQ: Bethesda, MD, United StatesReported employees: 1,001-5,000 | Bethesda, MD, United States | 1,001-5,000 | Exact match | 1,781 contacts → |
“Precision for Medicine is an industry-leading global clinical research services organization, purpose-built to improve the clinical research and development process for new…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 |  Novotechnovotech-cro.comHQ: Singapore, AustraliaReported employees: 1,001-5,000 | Singapore, Australia | 1,001-5,000 | Exact match | 1,778 contacts → |
“Novotech is a globally recognized full-service clinical research organization (CRO) and scientific advisory company that provides biotech and small- to mid-sized pharma companies…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Hetero Healthcare Limitedheterohealthcare.com HQ: Hyderabad, IndiaReported employees: 1,001-5,000 | Hyderabad, India | 1,001-5,000 | Exact match | 1,601 contacts → |
“Hetero Healthcare Ltd is a leading Innovative Pharmaceutical Company in India, headquartered at Hyderabad.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 |  EMD Serono, INCemdserono.comHQ: Boston, MA, United StatesReported employees: 1,001-5,000 | Boston, MA, United States | 1,001-5,000 | Exact match | 1,578 contacts → |
“We aspire to create, improve and prolong life for people living with difficult-to-treat conditions like infertility, multiple sclerosis and cancer.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 |  SOBIsobi.comHQ: Stockholm, SwedenReported employees: 1,001-5,000 | Stockholm, Sweden | 1,001-5,000 | Exact match | 1,561 contacts → |
“Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Ultragenyxultragenyx.com HQ: Novato, CA, United StatesReported employees: 1,001-5,000 | Novato, CA, United States | 1,001-5,000 | Exact match | 1,556 contacts → |
“Leading with purpose.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 |  Insmed Incorporatedinsmed.comHQ: Bridgewater Township, NJ, United StatesReported employees: 1,001-5,000 | Bridgewater Township, NJ, United States | 1,001-5,000 | Exact match | 1,512 contacts → |
“Insmed is a people-rst global biopharmaceutical company striving to deliver rst- and best-in-class therapies to transform the lives of patients facing serious diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 |  PTC Therapeutics, INCptcbio.comHQ: Warren, NJ, United StatesReported employees: 501-1,000 | Warren, NJ, United States | 501-1,000 | Exact match | 1,212 contacts → |
“PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 |  Neutec İlacneutec.com.trHQ: Istanbul, TürkiyeReported employees: 5,001-10,000 | Istanbul, Türkiye | 5,001-10,000 | Exact match | 1,054 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 23 |  CTI Clinical Trial And Consulting Servicesctifacts.comHQ: Covington, KY, United StatesReported employees: 1,001-5,000 | Covington, KY, United States | 1,001-5,000 | Exact match | 1,043 contacts → |
“CTI Clinical Trial and Consulting Services is a global, privately held, research service organization, delivering a complete spectrum of clinical trial and consulting services…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 |  BIALbial.comHQ: Trofa, PortugalReported employees: 501-1,000 | Trofa, Portugal | 501-1,000 | Exact match | 1,019 contacts → |
“Bial is a family-owned company with a rich history of over 100 years, dedicated to patient welfare, with products available in more than 50 countries.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 |  Purdue Pharma L.Ppurduepharma.comHQ: Stamford, CT, United StatesReported employees: 501-1,000 | Stamford, CT, United States | 501-1,000 | Exact match | 885 contacts → |
“Purdue and its subsidiaries develop, manufacture and market medications to meet the evolving needs of healthcare professionals, patients, and caregivers.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 1,083 company matches
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Orphan drug companies: dataset insights
September 20, 2026These summaries describe recorded coverage in this saved dataset. This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Reported company sizes
1,080 records in the displayed employee bands.
3 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
Email recency for this list is measured across the whole country list rather than for Worldwide, so it is reported there. Company counts and contact totals on this page are exact for Worldwide, over 1,083 companies.
How this company list was built
Describe a niche in your own words: companies installing commercial solar, or manufacturers of sustainable packaging. RevenueBase SmartSearch finds companies whose compiled business descriptions match by meaning. This list is one saved example to adapt.
Where the company descriptions come from
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
RevenueBase SmartSearch matching settings
- Exact
- Closest fits to the business described.
- Similar
- Similar businesses with a wider range of fits.
- Related
- Related businesses in and around the category.
- Broad
- A broader set of businesses connected to the description.
RevenueBase SmartSearch · Exact match identifies inclusion in this search's closest semantic matching setting. It is not a per-company confidence score, a literal keyword match, or a quality endorsement.
Selection, ordering and scope
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Recorded company descriptions support research; check company websites for current services. The selected preview is ordered by tracked contacts and is not a list of the best service providers.
Snapshot: September 20, 2026.
RevenueBase is a B2B data infrastructure company. Its September 3, 2026 published methodology describes a graph of 60 million companies and 398.9 million professional contacts; these platform figures differ from this page's totals. Published methodology ↗
Orphan drug companies by location
6 lists · largest firstEvery Orphan drug companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
Questions about this company list
What does Exact match mean for orphan drug companies?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many orphan drug companies did RevenueBase find worldwide?
The saved snapshot records 1,083 matching company records, 219,743 tracked contacts and 83,769 verified emails. The public preview displays 25 selected records.
Where do the company descriptions come from?
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
How are the public companies ordered?
The selected public records are ordered by contacts tracked in RevenueBase, highest first. Contact coverage is a prospecting measure, not an assessment of the company's services or quality.
How can I get contact information?
Select a company's contact count to open its filtered contacts in RevenueBase. Free signup is required and includes 500 credits and limited free results. Verified emails, direct dials and mobile numbers depend on available coverage.
How current is this list?
Snapshot: September 20, 2026. Review each company's website for current services and locations. A recorded business description does not establish that every company offers every service named in the search.
What do headquarters and employee bands mean?
These are recorded company-level fields, rather than a map of every branch or a count of specialists. Missing headquarters or employee bands are labeled; older records may show a location separately from recorded headquarters.
Build your next target company list
Adapt this niche search and work with available contacts in RevenueBase.
Related lists
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Cite this list
RevenueBase Data Team. Top 25 orphan drug companies worldwide. Snapshot: September 20, 2026. https://revenuebase.ai/companies/browse/orphan-drug-companies.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
More company lists
Company intelligence · public preview
Top 25 orphan drug companies worldwide
RevenueBase records 1,083 matching company records for orphan drug companies worldwide, with 219,743 tracked contacts and 83,769 verified work emails.
This public preview shows 25 selected records ordered by tracked contacts. RevenueBase SmartSearch finds company descriptions that match by meaning at Exact. Snapshot: September 20, 2026.
RevenueBase compiles business descriptions for niche-company discovery; each row shows a recorded excerpt where available. Adapt this search to find the companies and contacts you want.
Top 25 orphan drug companies worldwide
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 | Takedatakeda.com HQ: Tokyo, IL, JapanReported employees: 10001+ | Tokyo, IL, Japan | 10001+ | Exact match | 29,414 contacts → |
“We all want a healthier future.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | Johnson & Johnson Innovative Medicineinnovativemedicine.jnj.com HQ: Titusville, NJ, United StatesReported employees: 10001+ | Titusville, NJ, United States | 10001+ | Exact match | 26,637 contacts → |
“At Johnson & Johnson Innovative Medicine, we innovate with purpose, to lead where medicine is going.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 | Syneos Healthsyneoshealth.com HQ: Morrisville, NC, United StatesReported employees: 10001+ | Morrisville, NC, United States | 10001+ | Exact match | 18,228 contacts → |
“Syneos Health® is a leading fully integrated biopharmaceutical solutions organization built to accelerate customer success.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | Genentechgene.com HQ: South San Francisco, CA, United StatesReported employees: 10001+ | South San Francisco, CA, United States | 10001+ | Exact match | 17,217 contacts → |
“About Genentech We're passionate about finding solutions for people facing the world's most difficult-to-treat conditions.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | Regeneronregeneron.com HQ: Tarrytown, NY, United StatesReported employees: 10001+ | Tarrytown, NY, United States | 10001+ | Exact match | 16,043 contacts → |
“At Regeneron we believe that when the right idea finds the right team, powerful change is possible.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Servierservier.com HQ: Suresnes, FranceReported employees: 10001+ | Suresnes, France | 10001+ | Exact match | 10,958 contacts → |
“Servier is an independent international pharmaceutical group governed by a foundation.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | Alexion Pharmaceuticals, INCalexion.com HQ: Boston, MA, United StatesReported employees: 1,001-5,000 | Boston, MA, United States | 1,001-5,000 | Exact match | 6,136 contacts → |
“Alexion, AstraZeneca Rare Disease is focused on pioneering new possibilities for the rare disease community.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | IPSENipsen.com HQ: Paris, FranceReported employees: 5,001-10,000 | Paris, France | 5,001-10,000 | Exact match | 5,943 contacts → |
“We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | Biomarin Pharmaceutical INCbiomarin.com HQ: San Rafael, CA, United StatesReported employees: 1,001-5,000 | San Rafael, CA, United States | 1,001-5,000 | Exact match | 3,943 contacts → |
“Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | Chiesi Groupchiesi.com HQ: Parma, ItalyReported employees: 5,001-10,000 | Parma, Italy | 5,001-10,000 | Exact match | 3,644 contacts → |
“Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | Jazz Pharmaceuticalsjazzpharma.com HQ: Dublin, GermanyReported employees: 1,001-5,000 | Dublin, Germany | 1,001-5,000 | Exact match | 3,593 contacts → |
“Jazz Pharmaceuticals plc (Nasdaq: JAZZ), is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | Recordatirecordati.com HQ: Milan, ItalyReported employees: 1,001-5,000 | Milan, Italy | 1,001-5,000 | Exact match | 2,789 contacts → |
“Recordati has been at the forefront of life-enhancing and life-changing medicines for 100 years.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | Eisai USeisai.com HQ: Nutley, NJ, United StatesReported employees: 1,001-5,000 | Nutley, NJ, United States | 1,001-5,000 | Exact match | 2,570 contacts → |
“At Eisai Inc, human health care (hhc) is our goal.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Precision For Medicineprecisionformedicine.com HQ: Bethesda, MD, United StatesReported employees: 1,001-5,000 | Bethesda, MD, United States | 1,001-5,000 | Exact match | 1,781 contacts → |
“Precision for Medicine is an industry-leading global clinical research services organization, purpose-built to improve the clinical research and development process for new…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | Novotechnovotech-cro.com HQ: Singapore, AustraliaReported employees: 1,001-5,000 | Singapore, Australia | 1,001-5,000 | Exact match | 1,778 contacts → |
“Novotech is a globally recognized full-service clinical research organization (CRO) and scientific advisory company that provides biotech and small- to mid-sized pharma companies…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Hetero Healthcare Limitedheterohealthcare.com HQ: Hyderabad, IndiaReported employees: 1,001-5,000 | Hyderabad, India | 1,001-5,000 | Exact match | 1,601 contacts → |
“Hetero Healthcare Ltd is a leading Innovative Pharmaceutical Company in India, headquartered at Hyderabad.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | EMD Serono, INCemdserono.com HQ: Boston, MA, United StatesReported employees: 1,001-5,000 | Boston, MA, United States | 1,001-5,000 | Exact match | 1,578 contacts → |
“We aspire to create, improve and prolong life for people living with difficult-to-treat conditions like infertility, multiple sclerosis and cancer.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | SOBIsobi.com HQ: Stockholm, SwedenReported employees: 1,001-5,000 | Stockholm, Sweden | 1,001-5,000 | Exact match | 1,561 contacts → |
“Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Ultragenyxultragenyx.com HQ: Novato, CA, United StatesReported employees: 1,001-5,000 | Novato, CA, United States | 1,001-5,000 | Exact match | 1,556 contacts → |
“Leading with purpose.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Insmed Incorporatedinsmed.com HQ: Bridgewater Township, NJ, United StatesReported employees: 1,001-5,000 | Bridgewater Township, NJ, United States | 1,001-5,000 | Exact match | 1,512 contacts → |
“Insmed is a people-rst global biopharmaceutical company striving to deliver rst- and best-in-class therapies to transform the lives of patients facing serious diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | PTC Therapeutics, INCptcbio.com HQ: Warren, NJ, United StatesReported employees: 501-1,000 | Warren, NJ, United States | 501-1,000 | Exact match | 1,212 contacts → |
“PTC is a patient-centered biopharmaceutical company focused on discovering, developing & commercializing medicine for children and adults living with rare and serious disease.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | Neutec İlacneutec.com.tr HQ: Istanbul, TürkiyeReported employees: 5,001-10,000 | Istanbul, Türkiye | 5,001-10,000 | Exact match | 1,054 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 23 | CTI Clinical Trial And Consulting Servicesctifacts.com HQ: Covington, KY, United StatesReported employees: 1,001-5,000 | Covington, KY, United States | 1,001-5,000 | Exact match | 1,043 contacts → |
“CTI Clinical Trial and Consulting Services is a global, privately held, research service organization, delivering a complete spectrum of clinical trial and consulting services…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | BIALbial.com HQ: Trofa, PortugalReported employees: 501-1,000 | Trofa, Portugal | 501-1,000 | Exact match | 1,019 contacts → |
“Bial is a family-owned company with a rich history of over 100 years, dedicated to patient welfare, with products available in more than 50 countries.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Purdue Pharma L.Ppurduepharma.com HQ: Stamford, CT, United StatesReported employees: 501-1,000 | Stamford, CT, United States | 501-1,000 | Exact match | 885 contacts → |
“Purdue and its subsidiaries develop, manufacture and market medications to meet the evolving needs of healthcare professionals, patients, and caregivers.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 1,083 company matches
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Orphan drug companies: dataset insights
September 20, 2026These summaries describe recorded coverage in this saved dataset. This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Reported company sizes
1,080 records in the displayed employee bands.
3 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
Email recency for this list is measured across the whole country list rather than for Worldwide, so it is reported there. Company counts and contact totals on this page are exact for Worldwide, over 1,083 companies.
How this company list was built
Describe a niche in your own words: companies installing commercial solar, or manufacturers of sustainable packaging. RevenueBase SmartSearch finds companies whose compiled business descriptions match by meaning. This list is one saved example to adapt.
Where the company descriptions come from
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
RevenueBase SmartSearch matching settings
- Exact
- Closest fits to the business described.
- Similar
- Similar businesses with a wider range of fits.
- Related
- Related businesses in and around the category.
- Broad
- A broader set of businesses connected to the description.
RevenueBase SmartSearch · Exact match identifies inclusion in this search's closest semantic matching setting. It is not a per-company confidence score, a literal keyword match, or a quality endorsement.
Selection, ordering and scope
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
This page is derived from saved source records; per-contact mobile coverage and email-recency results are not established by this snapshot.
Recorded company descriptions support research; check company websites for current services. The selected preview is ordered by tracked contacts and is not a list of the best service providers.
Snapshot: September 20, 2026.
RevenueBase is a B2B data infrastructure company. Its September 3, 2026 published methodology describes a graph of 60 million companies and 398.9 million professional contacts; these platform figures differ from this page's totals. Published methodology ↗
Orphan drug companies by location
6 lists · largest firstEvery Orphan drug companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
Questions about this company list
What does Exact match mean for orphan drug companies?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many orphan drug companies did RevenueBase find worldwide?
The saved snapshot records 1,083 matching company records, 219,743 tracked contacts and 83,769 verified emails. The public preview displays 25 selected records.
Where do the company descriptions come from?
RevenueBase compiles company information from public sources, including company websites and public professional profiles such as LinkedIn, alongside information supplied by companies. The short recorded excerpt shown here is separate from the complete business description used for matching; individual source URLs and collection dates are not provided in this public snapshot.
How are the public companies ordered?
The selected public records are ordered by contacts tracked in RevenueBase, highest first. Contact coverage is a prospecting measure, not an assessment of the company's services or quality.
How can I get contact information?
Select a company's contact count to open its filtered contacts in RevenueBase. Free signup is required and includes 500 credits and limited free results. Verified emails, direct dials and mobile numbers depend on available coverage.
How current is this list?
Snapshot: September 20, 2026. Review each company's website for current services and locations. A recorded business description does not establish that every company offers every service named in the search.
What do headquarters and employee bands mean?
These are recorded company-level fields, rather than a map of every branch or a count of specialists. Missing headquarters or employee bands are labeled; older records may show a location separately from recorded headquarters.
Build your next target company list
Adapt this niche search and work with available contacts in RevenueBase.
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Cite this list
RevenueBase Data Team. Top 25 orphan drug companies worldwide. Snapshot: September 20, 2026. https://revenuebase.ai/companies/browse/orphan-drug-companies.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗