Company lists · Data refreshed September 2026
Top 25 Rare disease drug manufacturers based in the United States
RevenueBase identified 410 company matches for rare disease drug manufacturers associated with the United States as of September 2026 and tracks 313,445 professional contacts across them, including 119,530 with verified work emails. Boston is the largest hub.
This list covers company matches for rare disease drug manufacturers associated with the United States, 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 17, 2026.
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 rare disease drug manufacturers based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 88,560 contacts → | ||||
Exact match “We’re celebrating over 175 years of daring scientific innovation-and we’re not done yet.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 2 | 49,409 contacts → | ||||
Exact match “We're a medicine company turning science into healing to make life better for people around the world.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 3 | 33,433 contacts → | ||||
Exact match “Amgen harnesses the best of biology and technology to fight the world’s toughest diseases, and make people’s lives easier, fuller and longer.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 4 | 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 17, 2026 · View company records in RevenueBase | |||||
| 5 | 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 17, 2026 · View company records in RevenueBase | |||||
| 6 | 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 17, 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 17, 2026 · View company records in RevenueBase | |||||
| 8 | 4,649 contacts → | ||||
Exact match “Lupin Pharmaceuticals, Inc.” Recorded in the RevenueBase company graph · Sep 17, 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 17, 2026 · View company records in RevenueBase | |||||
| 10 | 2,570 contacts → | ||||
Exact match “At Eisai Inc, human health care (hhc) is our goal.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 11 | 2,154 contacts → | ||||
Exact match “Legend Biotech is a global, commercial-stage biotechnology company developing and manufacturing novel therapies.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 12 | 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 17, 2026 · View company records in RevenueBase | |||||
| 13 | 1,556 contacts → | ||||
Exact match “Leading with purpose.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 14 | 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 17, 2026 · View company records in RevenueBase | |||||
| 15 | 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 17, 2026 · View company records in RevenueBase | |||||
| 16 | 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 17, 2026 · View company records in RevenueBase | |||||
| 17 | 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 17, 2026 · View company records in RevenueBase | |||||
| 18 | 837 contacts → | ||||
Exact match “We exist to bring transformative medicines to people living with genetic conditions.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 19 | 790 contacts → | ||||
Exact match “Azurity Pharmaceuticals is a privately held global pharmaceutical company dedicated to redefining medicine for the real world.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 20 | 771 contacts → | ||||
Exact match “At Cencora, we are investing and innovating alongside our pharma customers to unlock their product’s potential.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 21 | 727 contacts → | ||||
Exact match “Kyowa Kirin is a global specialty pharmaceutical company that applies state-of-the-art biotechnologies to discover and deliver novel medicines.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 22 | 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 17, 2026 · View company records in RevenueBase | |||||
| 23 | 625 contacts → | ||||
Exact match “ANI Pharmaceuticals, Inc.(Nasdaq: ANIP) is a diversified biopharmaceutical company committed to its mission of “Serving Patients, Improving Lives" by developing, manufacturing…” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 24 | 603 contacts → | ||||
Exact match “Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Recorded in the RevenueBase company graph · Sep 17, 2026 · View company records in RevenueBase | |||||
| 25 | 582 contacts → | ||||
Exact match “PANTHERx Rare makes rare disease care more hyper-personalized and less overwhelming by focusing relentlessly on each patient and each therapy.” Recorded in the RevenueBase company graph · Sep 17, 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 “rare disease drug manufacturers”. 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 410 rare disease drug manufacturers based in the United States
See the full list, filter by size, metro and specialty, and access 119,530 tracked professional contacts with verified work emails. Direct dials are available where recorded.
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Rare disease drug manufacturers based in the United States at a glance
As of Sep 17, 2026Companies by employee count
Email recency for this list is measured worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 410 companies.
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.
- What qualifies
- Company records associated with the United States whose business descriptions match “rare disease drug manufacturers” 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. Where headquarters information is recorded, preview rows are also filtered to this page’s location. 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 119,530 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 17, 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.
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 “rare disease drug manufacturers”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many rare disease drug manufacturers are there based in the United States?
RevenueBase identified 410 company matches for rare disease drug manufacturers associated with the United States 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?
Pfizer tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.
Which city has the most rare disease drug manufacturers?
Boston leads with 13 company records, followed by New York (12) and San Diego (10).
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 119,530 professional contacts with verified work emails tracked across these 410 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 17, 2026.
Ready to reach these companies? Verified emails and direct dials for all 410 rare disease drug manufacturers based in the United States. Start with a free account, no credit card.
Download this list →Related lists
Rare disease drug manufacturers by geography
- Rare disease drug manufacturers based in Ahmedabad, India
- Rare disease drug manufacturers based in Hyderabad, India
- Rare disease drug manufacturers based in Mumbai, India
- Rare disease drug manufacturers based in Argentina
- Rare disease drug manufacturers based in Australia
- Rare disease drug manufacturers based in Brazil
- Rare disease drug manufacturers based in Canada
- Rare disease drug manufacturers based in China
- Rare disease drug manufacturers based in France
- Rare disease drug manufacturers based in Germany
- Rare disease drug manufacturers based in India
- Rare disease drug manufacturers based in Italy
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
- All company lists
- Lists by location
- Lists A-Z
- New lists
- Rare disease drug manufacturers based in Ahmedabad, India
- Rare disease drug manufacturers based in Hyderabad, India
- Rare disease drug manufacturers based in Mumbai, India
- Rare disease drug manufacturers based in Argentina
- Rare disease drug manufacturers based in Australia
- Rare disease drug manufacturers based in Brazil
Company intelligence · public preview
Top 25 rare disease drug manufacturers based in the United States
Find companies in any niche.
Describe the businesses you want in your own words. RevenueBase SmartSearch matches company descriptions by meaning.
RevenueBase records 410 matching company records for rare disease drug manufacturers based in the United States, with 313,445 tracked contacts and 119,530 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 17, 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 rare disease drug manufacturers based in the United States
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 |  Pfizerpfizer.comHQ: New York, NY, United StatesReported employees: 10001+ | New York, NY, United States | 10001+ | Exact match | 88,560 contacts → |
“We’re celebrating over 175 years of daring scientific innovation-and we’re not done yet.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 |  ELI Lilly And Companylilly.comHQ: Indianapolis, IN, United StatesReported employees: 10001+ | Indianapolis, IN, United States | 10001+ | Exact match | 49,409 contacts → |
“We're a medicine company turning science into healing to make life better for people around the world.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 |  AMGENamgen.comHQ: Thousand Oaks, CA, United StatesReported employees: 10001+ | Thousand Oaks, CA, United States | 10001+ | Exact match | 33,433 contacts → |
“Amgen harnesses the best of biology and technology to fight the world’s toughest diseases, and make people’s lives easier, fuller and longer.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 |  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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 |  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 17, 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 |  Lupin Pharmaceuticalslupin.comHQ: Somerset, NJ, United StatesReported employees: Not recorded | Somerset, NJ, United States | Not recorded | Exact match | 4,649 contacts → |
“Lupin Pharmaceuticals, Inc.” Recorded description excerpt · September 17, 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 |  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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 |  Legend Biotechlegendbiotech.comHQ: Somerset, NJ, United StatesReported employees: 1,001-5,000 | Somerset, NJ, United States | 1,001-5,000 | Exact match | 2,154 contacts → |
“Legend Biotech is a global, commercial-stage biotechnology company developing and manufacturing novel therapies.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 |  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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 |  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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 |  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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 |  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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 |  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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 |  Bridgebiobridgebio.comHQ: Palo Alto, CA, United StatesReported employees: 501-1,000 | Palo Alto, CA, United States | 501-1,000 | Exact match | 837 contacts → |
“We exist to bring transformative medicines to people living with genetic conditions.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 |  Azurity Pharmaceuticalsazurity.comHQ: Woburn, MA, United StatesReported employees: 501-1,000 | Woburn, MA, United States | 501-1,000 | Exact match | 790 contacts → |
“Azurity Pharmaceuticals is a privately held global pharmaceutical company dedicated to redefining medicine for the real world.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 |  Cencora Pharma Solutionscencora.comHQ: Conshohocken, PA, United StatesReported employees: 10001+ | Conshohocken, PA, United States | 10001+ | Exact match | 771 contacts → |
“At Cencora, we are investing and innovating alongside our pharma customers to unlock their product’s potential.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 |  Kyowa Kirin, Inc.- U.Skkna.kyowakirin.comHQ: Princeton, NJ, United StatesReported employees: 501-1,000 | Princeton, NJ, United States | 501-1,000 | Exact match | 727 contacts → |
“Kyowa Kirin is a global specialty pharmaceutical company that applies state-of-the-art biotechnologies to discover and deliver novel medicines.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 |  ANI Pharmaceuticals, INCanipharmaceuticals.comHQ: Princeton, NJ, United StatesReported employees: 501-1,000 | Princeton, NJ, United States | 501-1,000 | Exact match | 625 contacts → |
“ANI Pharmaceuticals, Inc.(Nasdaq: ANIP) is a diversified biopharmaceutical company committed to its mission of “Serving Patients, Improving Lives" by developing, manufacturing…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 |  Agios Pharmaceuticalsagios.comHQ: Cambridge, MA, United StatesReported employees: 501-1,000 | Cambridge, MA, United States | 501-1,000 | Exact match | 603 contacts → |
“Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 |  Pantherx Rare Pharmacypantherxrare.comHQ: Pittsburgh, PA, United StatesReported employees: 501-1,000 | Pittsburgh, PA, United States | 501-1,000 | Exact match | 582 contacts → |
“PANTHERx Rare makes rare disease care more hyper-personalized and less overwhelming by focusing relentlessly on each patient and each therapy.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 410 company matches
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Rare disease drug manufacturers: dataset insights
September 17, 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
409 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 worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 410 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 17, 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 ↗
Questions about this company list
What does Exact match mean for rare disease drug manufacturers?
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 rare disease drug manufacturers did RevenueBase find based in the United States?
The saved snapshot records 410 matching company records, 313,445 tracked contacts and 119,530 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 17, 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
Rare disease drug manufacturers by geography
- Rare disease drug manufacturers based in Ahmedabad, India
- Rare disease drug manufacturers based in Hyderabad, India
- Rare disease drug manufacturers based in Mumbai, India
- Rare disease drug manufacturers based in Argentina
- Rare disease drug manufacturers based in Australia
- Rare disease drug manufacturers based in Brazil
- Rare disease drug manufacturers based in Canada
- Rare disease drug manufacturers based in China
- Rare disease drug manufacturers based in France
- Rare disease drug manufacturers based in Germany
- Rare disease drug manufacturers based in India
- Rare disease drug manufacturers based in Italy
Cite this list
RevenueBase Data Team. Top 25 rare disease drug manufacturers based in the United States. Snapshot: September 17, 2026. https://revenuebase.ai/companies/rare-disease-drug-manufacturers/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
More company lists
- All company lists
- Lists by location
- Lists A-Z
- New lists
- Rare disease drug manufacturers based in Ahmedabad, India
- Rare disease drug manufacturers based in Hyderabad, India
- Rare disease drug manufacturers based in Mumbai, India
- Rare disease drug manufacturers based in Argentina
- Rare disease drug manufacturers based in Australia
- Rare disease drug manufacturers based in Brazil
Company intelligence · public preview
Top 25 rare disease drug manufacturers based in the United States
RevenueBase records 410 matching company records for rare disease drug manufacturers based in the United States, with 313,445 tracked contacts and 119,530 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 17, 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 rare disease drug manufacturers based in the United States
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 | Pfizerpfizer.com HQ: New York, NY, United StatesReported employees: 10001+ | New York, NY, United States | 10001+ | Exact match | 88,560 contacts → |
“We’re celebrating over 175 years of daring scientific innovation-and we’re not done yet.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | ELI Lilly And Companylilly.com HQ: Indianapolis, IN, United StatesReported employees: 10001+ | Indianapolis, IN, United States | 10001+ | Exact match | 49,409 contacts → |
“We're a medicine company turning science into healing to make life better for people around the world.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 | AMGENamgen.com HQ: Thousand Oaks, CA, United StatesReported employees: 10001+ | Thousand Oaks, CA, United States | 10001+ | Exact match | 33,433 contacts → |
“Amgen harnesses the best of biology and technology to fight the world’s toughest diseases, and make people’s lives easier, fuller and longer.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | 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 17, 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | Lupin Pharmaceuticalslupin.com HQ: Somerset, NJ, United StatesReported employees: Not recorded | Somerset, NJ, United States | Not recorded | Exact match | 4,649 contacts → |
“Lupin Pharmaceuticals, Inc.” Recorded description excerpt · September 17, 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | Legend Biotechlegendbiotech.com HQ: Somerset, NJ, United StatesReported employees: 1,001-5,000 | Somerset, NJ, United States | 1,001-5,000 | Exact match | 2,154 contacts → |
“Legend Biotech is a global, commercial-stage biotechnology company developing and manufacturing novel therapies.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 16 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | Bridgebiobridgebio.com HQ: Palo Alto, CA, United StatesReported employees: 501-1,000 | Palo Alto, CA, United States | 501-1,000 | Exact match | 837 contacts → |
“We exist to bring transformative medicines to people living with genetic conditions.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Azurity Pharmaceuticalsazurity.com HQ: Woburn, MA, United StatesReported employees: 501-1,000 | Woburn, MA, United States | 501-1,000 | Exact match | 790 contacts → |
“Azurity Pharmaceuticals is a privately held global pharmaceutical company dedicated to redefining medicine for the real world.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | Cencora Pharma Solutionscencora.com HQ: Conshohocken, PA, United StatesReported employees: 10001+ | Conshohocken, PA, United States | 10001+ | Exact match | 771 contacts → |
“At Cencora, we are investing and innovating alongside our pharma customers to unlock their product’s potential.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | Kyowa Kirin, Inc.- U.Skkna.kyowakirin.com HQ: Princeton, NJ, United StatesReported employees: 501-1,000 | Princeton, NJ, United States | 501-1,000 | Exact match | 727 contacts → |
“Kyowa Kirin is a global specialty pharmaceutical company that applies state-of-the-art biotechnologies to discover and deliver novel medicines.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | 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 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | ANI Pharmaceuticals, INCanipharmaceuticals.com HQ: Princeton, NJ, United StatesReported employees: 501-1,000 | Princeton, NJ, United States | 501-1,000 | Exact match | 625 contacts → |
“ANI Pharmaceuticals, Inc.(Nasdaq: ANIP) is a diversified biopharmaceutical company committed to its mission of “Serving Patients, Improving Lives" by developing, manufacturing…” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | Agios Pharmaceuticalsagios.com HQ: Cambridge, MA, United StatesReported employees: 501-1,000 | Cambridge, MA, United States | 501-1,000 | Exact match | 603 contacts → |
“Agios is a commercial-stage biopharmaceutical company dedicated to redefining the future of rare disease treatment.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Pantherx Rare Pharmacypantherxrare.com HQ: Pittsburgh, PA, United StatesReported employees: 501-1,000 | Pittsburgh, PA, United States | 501-1,000 | Exact match | 582 contacts → |
“PANTHERx Rare makes rare disease care more hyper-personalized and less overwhelming by focusing relentlessly on each patient and each therapy.” Recorded description excerpt · September 17, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 410 company matches
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Rare disease drug manufacturers: dataset insights
September 17, 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
409 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 worldwide rather than for United States, so it is reported there. Company counts and contact totals on this page are exact for United States, over 410 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 17, 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 ↗
Questions about this company list
What does Exact match mean for rare disease drug manufacturers?
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 rare disease drug manufacturers did RevenueBase find based in the United States?
The saved snapshot records 410 matching company records, 313,445 tracked contacts and 119,530 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 17, 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
Rare disease drug manufacturers by geography
- Rare disease drug manufacturers based in Ahmedabad, India
- Rare disease drug manufacturers based in Hyderabad, India
- Rare disease drug manufacturers based in Mumbai, India
- Rare disease drug manufacturers based in Argentina
- Rare disease drug manufacturers based in Australia
- Rare disease drug manufacturers based in Brazil
- Rare disease drug manufacturers based in Canada
- Rare disease drug manufacturers based in China
- Rare disease drug manufacturers based in France
- Rare disease drug manufacturers based in Germany
- Rare disease drug manufacturers based in India
- Rare disease drug manufacturers based in Italy
Cite this list
RevenueBase Data Team. Top 25 rare disease drug manufacturers based in the United States. Snapshot: September 17, 2026. https://revenuebase.ai/companies/rare-disease-drug-manufacturers/united-states.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
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- Rare disease drug manufacturers based in Ahmedabad, India
- Rare disease drug manufacturers based in Hyderabad, India
- Rare disease drug manufacturers based in Mumbai, India
- Rare disease drug manufacturers based in Argentina
- Rare disease drug manufacturers based in Australia
- Rare disease drug manufacturers based in Brazil