Company lists · Data refreshed September 2026
Top 25 Orphan drug developers worldwide
RevenueBase identified 252 company matches for orphan drug developers worldwide as of September 2026 and tracks 23,330 professional contacts across them, including 10,884 with verified work emails. The United States is the largest market and Cambridge the largest hub.
This list covers company matches for orphan drug developers 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 developers 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 developers worldwide
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 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 | |||||
| 2 | 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 | |||||
| 3 | 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 | |||||
| 4 | 1,556 contacts → | ||||
Exact match “Leading with purpose.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 5 | 657 contacts → | ||||
Exact match “Arrowhead Pharmaceuticals is a clinical stage, Nasdaq listed (ticker ARWR) company developing medicines that treat intractable diseases by silencing the genes that cause them.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 6 | 548 contacts → | ||||
Exact match “At AMAG, aiming higher is in our DNA.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 7 | 491 contacts → | ||||
Exact match “Our mission is to identify, develop and deliver life-changing therapies to people living with rare disease.#InRareForLife Community Guidelines…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 8 | 395 contacts → | ||||
Exact match “Upsher‑Smith is the commercial arm of the Bora Group, focused on developing, marketing, and distributing pharmaceutical products that make a real difference for patients.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 9 | 374 contacts → | ||||
Exact match “Mirum Pharmaceuticals is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 10 | 259 contacts → | ||||
Exact match “Summit Mission Statement: To build a viable, long-lasting health care organization that assumes full responsibility for designing, developing, trial execution and enrollment…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 11 | 221 contacts → | ||||
Exact match “Nordic Pharma is a privately owned, medium-size, fully integrated Pharma company with a history of internal product development and acquisitions.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 12 | 217 contacts → | ||||
Exact match “Focused on complex therapies and orphan diseases, Varifarma is a company which is devoted to improving the patients' quality of life.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 13 | 189 contacts → | ||||
Exact match “Founded in 2003, CG Life is a specialized pharma agency built for the unique challenges of bringing therapies for rare and hard-to-treat diseases to market, where traditional…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 14 | 185 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 15 | 175 contacts → | ||||
Exact match “GENESIS Pharma is a European biopharmaceutical company specializing in the commercialization of innovative pharmaceutical products for serious and rare diseases.” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 16 | 137 contacts → | ||||
Exact match “Centessa Pharmaceuticals plc, a wholly owned subsidiary of Eli Lilly and Company, is developing a portfolio of orexin receptor 2 (OX2R) agonists for the potential treatment of…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 17 | 118 contacts → | ||||
Exact match “Transforming Rare Disease Treatment At Orphalan we identify, develop, and provide access to innovative treatments for patients with rare diseases to make a positive and meaningful…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 18 | 116 contacts → | ||||
Exact match “Pioneering Medicines, Flagship Pioneering’s in house drug discovery and development unit, is dedicated to conceiving and developing a broad portfolio of life-changing treatments…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 19 | 114 contacts → | ||||
Exact match “With demonstrated clinical development and commercialization expertise, VIVUS is dedicated to addressing the therapeutic needs of patients with serious medical conditions and…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 20 | 98 contacts → | ||||
Exact match “QOL Medical is a specialty biopharmaceutical company dedicated to improving the quality of life for patients with rare diseases, for their families, and for the physicians and…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 21 | 84 contacts → | ||||
Exact match “We are a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 22 | 80 contacts → | ||||
Exact match “ADIENNE is an integrated biopharmaceutical group of companies based in Lugano, Switzerland, with a proprietary portfolio of orphan-designated marketed medicinal product, clinical…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 23 | 78 contacts → | ||||
Exact match “We are a purpose-driven, commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 24 | 74 contacts → | ||||
Exact match “Valentech is a specialized Latin American healthcare company focused on enabling patient access to innovative therapies for high‑burden rare diseases across Central and South…” Recorded in the RevenueBase company graph · Sep 20, 2026 · View company records in RevenueBase | |||||
| 25 | 70 contacts → | ||||
Exact match “We are committed to transforming the lives of people affected by cancer or rare disease, globally.” 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 developers”. 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 252 orphan drug developers worldwide
See the full list, filter by size, metro and specialty, and access 10,884 tracked professional contacts with verified work emails. Direct dials are available where recorded.
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Orphan drug developers 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 developers are linked below.
- What qualifies
- Company records worldwide whose business descriptions match “orphan drug developers” 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 10,884 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 developers by location
1 lists · largest firstEvery Orphan drug developers 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 developers”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many orphan drug developers are there worldwide?
RevenueBase identified 252 company matches for orphan drug developers 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?
Alexion Pharmaceuticals, INC tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.
Which country has the most orphan drug developers?
The United States leads with 95 companies. Every orphan drug developers list by country and city is linked on this page.
Which city has the most orphan drug developers?
Cambridge leads with 7 company records, followed by London (7) and New York (6).
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 10,884 professional contacts with verified work emails tracked across these 252 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.
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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 developers worldwide
Find companies in any niche.
Describe the businesses you want in your own words. RevenueBase SmartSearch matches company descriptions by meaning.
RevenueBase records 252 matching company records for orphan drug developers worldwide, with 23,330 tracked contacts and 10,884 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 developers 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 |  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. | |||||
| 2 |  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. | |||||
| 3 |  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. | |||||
| 4 | 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. | |||||
| 5 | Arrowhead Pharmaceuticalsarrowheadpharma.com HQ: Pasadena, CA, United StatesReported employees: 501-1,000 | Pasadena, CA, United States | 501-1,000 | Exact match | 657 contacts → |
“Arrowhead Pharmaceuticals is a clinical stage, Nasdaq listed (ticker ARWR) company developing medicines that treat intractable diseases by silencing the genes that cause them.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 |  Amag Pharmaceuticalsamagpharma.comHQ: Waltham, MA, United StatesReported employees: 201-500 | Waltham, MA, United States | 201-500 | Exact match | 548 contacts → |
“At AMAG, aiming higher is in our DNA.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | Travere Therapeuticstravere.com HQ: San Diego, CA, United StatesReported employees: 201-500 | San Diego, CA, United States | 201-500 | Exact match | 491 contacts → |
“Our mission is to identify, develop and deliver life-changing therapies to people living with rare disease.#InRareForLife Community Guidelines…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | Upsher-Smithupsher-smith.com HQ: Maple Grove, MN, United StatesReported employees: 51-200 | Maple Grove, MN, United States | 51-200 | Exact match | 395 contacts → |
“Upsher‑Smith is the commercial arm of the Bora Group, focused on developing, marketing, and distributing pharmaceutical products that make a real difference for patients.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | Mirum Pharmaceuticals, INCmirumpharma.com HQ: Foster City, CA, United StatesReported employees: 201-500 | Foster City, CA, United States | 201-500 | Exact match | 374 contacts → |
“Mirum Pharmaceuticals is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 |  Summit Therapeutics, INCsmmttx.comHQ: Miami, FL, United StatesReported employees: 201-500 | Miami, FL, United States | 201-500 | Exact match | 259 contacts → |
“Summit Mission Statement: To build a viable, long-lasting health care organization that assumes full responsibility for designing, developing, trial execution and enrollment…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 |  Nordic Pharmanordicpharma.comHQ: Hoofddorp, NetherlandsReported employees: 201-500 | Hoofddorp, Netherlands | 201-500 | Exact match | 221 contacts → |
“Nordic Pharma is a privately owned, medium-size, fully integrated Pharma company with a history of internal product development and acquisitions.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | Laboratorio Varifarmavarifarma.com HQ: Beccar, ArgentinaReported employees: 201-500 | Beccar, Argentina | 201-500 | Exact match | 217 contacts → |
“Focused on complex therapies and orphan diseases, Varifarma is a company which is devoted to improving the patients' quality of life.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 |  CG Lifecglife.comHQ: Chicago, IL, United StatesReported employees: 51-200 | Chicago, IL, United States | 51-200 | Exact match | 189 contacts → |
“Founded in 2003, CG Life is a specialized pharma agency built for the unique challenges of bringing therapies for rare and hard-to-treat diseases to market, where traditional…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Catalyst Pharmaceuticals, INCcatalystpharma.com HQ: Coral Gables, FL, United StatesReported employees: 51-200 | Coral Gables, FL, United States | 51-200 | Exact match | 185 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 15 |  Genesis Pharmagenesispharma.comHQ: Athens, GreeceReported employees: 51-200 | Athens, Greece | 51-200 | Exact match | 175 contacts → |
“GENESIS Pharma is a European biopharmaceutical company specializing in the commercialization of innovative pharmaceutical products for serious and rare diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Centessa Pharmaceuticalscentessa.com HQ: United StatesReported employees: 51-200 | United States | 51-200 | Exact match | 137 contacts → |
“Centessa Pharmaceuticals plc, a wholly owned subsidiary of Eli Lilly and Company, is developing a portfolio of orexin receptor 2 (OX2R) agonists for the potential treatment of…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Orphalanorphalan.com HQ: Paris, FranceReported employees: 51-200 | Paris, France | 51-200 | Exact match | 118 contacts → |
“Transforming Rare Disease Treatment At Orphalan we identify, develop, and provide access to innovative treatments for patients with rare diseases to make a positive and meaningful…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 |  Pioneering Medicinesflagshippioneering.comHQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 116 contacts → |
“Pioneering Medicines, Flagship Pioneering’s in house drug discovery and development unit, is dedicated to conceiving and developing a broad portfolio of life-changing treatments…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Vivus LLCvivus.com HQ: Campbell, CA, United StatesReported employees: 11-50 | Campbell, CA, United States | 11-50 | Exact match | 114 contacts → |
“With demonstrated clinical development and commercialization expertise, VIVUS is dedicated to addressing the therapeutic needs of patients with serious medical conditions and…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | QOL Medical LLCqolmed.com HQ: Vero Beach, FL, United StatesReported employees: 51-200 | Vero Beach, FL, United States | 51-200 | Exact match | 98 contacts → |
“QOL Medical is a specialty biopharmaceutical company dedicated to improving the quality of life for patients with rare diseases, for their families, and for the physicians and…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | Fulcrum Therapeuticsfulcrumtx.com HQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 84 contacts → |
“We are a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 |  Adienne Pharma & Biotechadienne.comHQ: Lugano, SwitzerlandReported employees: 51-200 | Lugano, Switzerland | 51-200 | Exact match | 80 contacts → |
“ADIENNE is an integrated biopharmaceutical group of companies based in Lugano, Switzerland, with a proprietary portfolio of orphan-designated marketed medicinal product, clinical…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 |  Zevra Therapeuticszevra.comHQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 78 contacts → |
“We are a purpose-driven, commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | Valentech Pharmavalentechforlife.com HQ: Bogotá, ColombiaReported employees: 51-200 | Bogotá, Colombia | 51-200 | Exact match | 74 contacts → |
“Valentech is a specialized Latin American healthcare company focused on enabling patient access to innovative therapies for high‑burden rare diseases across Central and South…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Eusa Pharmaeusapharma.com HQ: Hemel Hempstead, United KingdomReported employees: 201-500 | Hemel Hempstead, United Kingdom | 201-500 | Exact match | 70 contacts → |
“We are committed to transforming the lives of people affected by cancer or rare disease, globally.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 252 company matches
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Orphan drug developers: 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
251 records in the displayed employee bands.
1 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 252 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 developers by location
1 lists · largest firstEvery Orphan drug developers 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 developers?
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 developers did RevenueBase find worldwide?
The saved snapshot records 252 matching company records, 23,330 tracked contacts and 10,884 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 developers worldwide. Snapshot: September 20, 2026. https://revenuebase.ai/companies/browse/orphan-drug-developers.
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 developers worldwide
RevenueBase records 252 matching company records for orphan drug developers worldwide, with 23,330 tracked contacts and 10,884 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 developers 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 | 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. | |||||
| 2 | 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. | |||||
| 3 | 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. | |||||
| 4 | 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. | |||||
| 5 | Arrowhead Pharmaceuticalsarrowheadpharma.com HQ: Pasadena, CA, United StatesReported employees: 501-1,000 | Pasadena, CA, United States | 501-1,000 | Exact match | 657 contacts → |
“Arrowhead Pharmaceuticals is a clinical stage, Nasdaq listed (ticker ARWR) company developing medicines that treat intractable diseases by silencing the genes that cause them.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | Amag Pharmaceuticalsamagpharma.com HQ: Waltham, MA, United StatesReported employees: 201-500 | Waltham, MA, United States | 201-500 | Exact match | 548 contacts → |
“At AMAG, aiming higher is in our DNA.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | Travere Therapeuticstravere.com HQ: San Diego, CA, United StatesReported employees: 201-500 | San Diego, CA, United States | 201-500 | Exact match | 491 contacts → |
“Our mission is to identify, develop and deliver life-changing therapies to people living with rare disease.#InRareForLife Community Guidelines…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | Upsher-Smithupsher-smith.com HQ: Maple Grove, MN, United StatesReported employees: 51-200 | Maple Grove, MN, United States | 51-200 | Exact match | 395 contacts → |
“Upsher‑Smith is the commercial arm of the Bora Group, focused on developing, marketing, and distributing pharmaceutical products that make a real difference for patients.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | Mirum Pharmaceuticals, INCmirumpharma.com HQ: Foster City, CA, United StatesReported employees: 201-500 | Foster City, CA, United States | 201-500 | Exact match | 374 contacts → |
“Mirum Pharmaceuticals is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | Summit Therapeutics, INCsmmttx.com HQ: Miami, FL, United StatesReported employees: 201-500 | Miami, FL, United States | 201-500 | Exact match | 259 contacts → |
“Summit Mission Statement: To build a viable, long-lasting health care organization that assumes full responsibility for designing, developing, trial execution and enrollment…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | Nordic Pharmanordicpharma.com HQ: Hoofddorp, NetherlandsReported employees: 201-500 | Hoofddorp, Netherlands | 201-500 | Exact match | 221 contacts → |
“Nordic Pharma is a privately owned, medium-size, fully integrated Pharma company with a history of internal product development and acquisitions.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | Laboratorio Varifarmavarifarma.com HQ: Beccar, ArgentinaReported employees: 201-500 | Beccar, Argentina | 201-500 | Exact match | 217 contacts → |
“Focused on complex therapies and orphan diseases, Varifarma is a company which is devoted to improving the patients' quality of life.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | CG Lifecglife.com HQ: Chicago, IL, United StatesReported employees: 51-200 | Chicago, IL, United States | 51-200 | Exact match | 189 contacts → |
“Founded in 2003, CG Life is a specialized pharma agency built for the unique challenges of bringing therapies for rare and hard-to-treat diseases to market, where traditional…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Catalyst Pharmaceuticals, INCcatalystpharma.com HQ: Coral Gables, FL, United StatesReported employees: 51-200 | Coral Gables, FL, United States | 51-200 | Exact match | 185 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 15 | Genesis Pharmagenesispharma.com HQ: Athens, GreeceReported employees: 51-200 | Athens, Greece | 51-200 | Exact match | 175 contacts → |
“GENESIS Pharma is a European biopharmaceutical company specializing in the commercialization of innovative pharmaceutical products for serious and rare diseases.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | Centessa Pharmaceuticalscentessa.com HQ: United StatesReported employees: 51-200 | United States | 51-200 | Exact match | 137 contacts → |
“Centessa Pharmaceuticals plc, a wholly owned subsidiary of Eli Lilly and Company, is developing a portfolio of orexin receptor 2 (OX2R) agonists for the potential treatment of…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Orphalanorphalan.com HQ: Paris, FranceReported employees: 51-200 | Paris, France | 51-200 | Exact match | 118 contacts → |
“Transforming Rare Disease Treatment At Orphalan we identify, develop, and provide access to innovative treatments for patients with rare diseases to make a positive and meaningful…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Pioneering Medicinesflagshippioneering.com HQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 116 contacts → |
“Pioneering Medicines, Flagship Pioneering’s in house drug discovery and development unit, is dedicated to conceiving and developing a broad portfolio of life-changing treatments…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Vivus LLCvivus.com HQ: Campbell, CA, United StatesReported employees: 11-50 | Campbell, CA, United States | 11-50 | Exact match | 114 contacts → |
“With demonstrated clinical development and commercialization expertise, VIVUS is dedicated to addressing the therapeutic needs of patients with serious medical conditions and…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | QOL Medical LLCqolmed.com HQ: Vero Beach, FL, United StatesReported employees: 51-200 | Vero Beach, FL, United States | 51-200 | Exact match | 98 contacts → |
“QOL Medical is a specialty biopharmaceutical company dedicated to improving the quality of life for patients with rare diseases, for their families, and for the physicians and…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | Fulcrum Therapeuticsfulcrumtx.com HQ: Cambridge, MA, United StatesReported employees: 51-200 | Cambridge, MA, United States | 51-200 | Exact match | 84 contacts → |
“We are a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | Adienne Pharma & Biotechadienne.com HQ: Lugano, SwitzerlandReported employees: 51-200 | Lugano, Switzerland | 51-200 | Exact match | 80 contacts → |
“ADIENNE is an integrated biopharmaceutical group of companies based in Lugano, Switzerland, with a proprietary portfolio of orphan-designated marketed medicinal product, clinical…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Zevra Therapeuticszevra.com HQ: Boston, MA, United StatesReported employees: 51-200 | Boston, MA, United States | 51-200 | Exact match | 78 contacts → |
“We are a purpose-driven, commercial-stage company with a late-stage pipeline committed to redefining what is possible in bringing life-changing therapies to people living with…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | Valentech Pharmavalentechforlife.com HQ: Bogotá, ColombiaReported employees: 51-200 | Bogotá, Colombia | 51-200 | Exact match | 74 contacts → |
“Valentech is a specialized Latin American healthcare company focused on enabling patient access to innovative therapies for high‑burden rare diseases across Central and South…” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Eusa Pharmaeusapharma.com HQ: Hemel Hempstead, United KingdomReported employees: 201-500 | Hemel Hempstead, United Kingdom | 201-500 | Exact match | 70 contacts → |
“We are committed to transforming the lives of people affected by cancer or rare disease, globally.” Recorded description excerpt · September 20, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 252 company matches
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Orphan drug developers: 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
251 records in the displayed employee bands.
1 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 252 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 developers by location
1 lists · largest firstEvery Orphan drug developers 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 developers?
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 developers did RevenueBase find worldwide?
The saved snapshot records 252 matching company records, 23,330 tracked contacts and 10,884 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 developers worldwide. Snapshot: September 20, 2026. https://revenuebase.ai/companies/browse/orphan-drug-developers.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗