Company lists · Data refreshed September 2026
Top 25 Rare disease companies based in the United States
RevenueBase identified 270 company matches for rare disease companies associated with the United States as of September 2026 and tracks 91,999 professional contacts across them, including 33,184 with verified work emails. Boston is the largest hub.
This list covers company matches for rare disease companies 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 21, 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 companies based in the United States
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 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 21, 2026 · View company records in RevenueBase | |||||
| 2 | 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 21, 2026 · View company records in RevenueBase | |||||
| 3 | 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 21, 2026 · View company records in RevenueBase | |||||
| 4 | 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 21, 2026 · View company records in RevenueBase | |||||
| 5 | 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 21, 2026 · View company records in RevenueBase | |||||
| 6 | 1,556 contacts → | ||||
Exact match “Leading with purpose.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 7 | 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 21, 2026 · View company records in RevenueBase | |||||
| 8 | 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 21, 2026 · View company records in RevenueBase | |||||
| 9 | 837 contacts → | ||||
Exact match “We exist to bring transformative medicines to people living with genetic conditions.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 10 | 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 21, 2026 · View company records in RevenueBase | |||||
| 11 | 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 21, 2026 · View company records in RevenueBase | |||||
| 12 | 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 21, 2026 · View company records in RevenueBase | |||||
| 13 | 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 21, 2026 · View company records in RevenueBase | |||||
| 14 | 575 contacts → | ||||
Exact match “Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 15 | 561 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 16 | 548 contacts → | ||||
Exact match “At AMAG, aiming higher is in our DNA.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 17 | 513 contacts → | ||||
Exact match “Chiesi USA, Inc, headquartered in Cary, N.C, is a specialty pharmaceutical company focused on commercialization of products for the hospital, rare disease and target office-based…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 18 | 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 21, 2026 · View company records in RevenueBase | |||||
| 19 | 411 contacts → | ||||
Exact match “Rhythm Pharmaceuticals is a global, commercial-stage biopharmaceutical company focused on transforming the lives of patients with rare neuroendocrine diseases by rapidly advancing…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 20 | 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 21, 2026 · View company records in RevenueBase | |||||
| 21 | 392 contacts → | ||||
Exact match “SpringWorks Therapeutics, a healthcare company of Merck KGaA, Darmstadt, Germany, is a commercial-stage biopharmaceutical company dedicated to improving the lives of patients with…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 22 | 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 21, 2026 · View company records in RevenueBase | |||||
| 23 | 365 contacts → | ||||
Exact match “Kiniksa is a biopharmaceutical company dedicated to improving the lives of patients suffering from debilitating diseases by discovering, acquiring, developing, and commercializing…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 24 | 326 contacts → | ||||
Exact match “Sobi is an international specialty healthcare company dedicated to developing treatments and extensive patient support networks for people with rare and difficult to treat…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 25 | 313 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 21, 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 companies”. Each Exact match badge shows membership in that saved search; it is not an individual similarity score or a service-quality rating. Recorded descriptions are shown where available.
Showing 25 of 270 rare disease companies based in the United States
See the full list, filter by size, metro and specialty, and access 33,184 tracked professional contacts with verified work emails. Direct dials are available where recorded.
Free signup · no credit card
Rare disease companies based in the United States at a glance
As of Sep 21, 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 270 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 companies” by meaning at the Exact setting. Descriptions are compiled in the RevenueBase company graph from public websites, profiles and company information where available; they are not guaranteed quotations from the company's website.
- RevenueBase SmartSearch
- Describe a niche company in your own words to find businesses by meaning. Exact, Similar, Related and Broad control the breadth of RevenueBase SmartSearch. This saved list uses Exact. Each row's Exact match badge shows inclusion in that search, not an individual score or a service-quality judgment.
- Public preview
- The public preview omits records without a verified work email. 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 33,184 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 21, 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 companies”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many rare disease companies are there based in the United States?
RevenueBase identified 270 company matches for rare disease companies 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?
Johnson & Johnson Innovative Medicine tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.
Which city has the most rare disease companies?
Boston leads with 16 company records, followed by New York (14) and San Diego (11).
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 33,184 professional contacts with verified work emails tracked across these 270 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 21, 2026.
Ready to reach these companies? Verified emails and direct dials for all 270 rare disease companies based in the United States. Start with a free account, no credit card.
Download this list →Related lists
Rare disease companies by geography
Browse the directory
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 companies based in France
- Rare disease companies based in the United Kingdom
- AI companies based in the United States
- Cybersecurity companies based in the United States
- Manufacturing companies based in the United States
- Media companies based in the United States
Company intelligence · public preview
Top 25 rare disease companies 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 270 matching company records for rare disease companies based in the United States, with 91,999 tracked contacts and 33,184 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 21, 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 companies 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 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Vanda Pharmaceuticalsvandapharma.com HQ: Washington, DC, United StatesReported employees: 201-500 | Washington, DC, United States | 201-500 | Exact match | 575 contacts → |
“Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 |  Takeda Pharmaceuticals America, INCtakeda.comHQ: Lincolnshire, IL, United StatesReported employees: 10001+ | Lincolnshire, IL, United States | 10001+ | Exact match | 561 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 16 |  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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 |  Chiesi Usa, INCchiesiusa.comHQ: Cary, NC, United StatesReported employees: 201-500 | Cary, NC, United States | 201-500 | Exact match | 513 contacts → |
“Chiesi USA, Inc, headquartered in Cary, N.C, is a specialty pharmaceutical company focused on commercialization of products for the hospital, rare disease and target office-based…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Rhythm Pharmaceuticals INCrhythmtx.com HQ: Boston, MA, United StatesReported employees: 201-500 | Boston, MA, United States | 201-500 | Exact match | 411 contacts → |
“Rhythm Pharmaceuticals is a global, commercial-stage biopharmaceutical company focused on transforming the lives of patients with rare neuroendocrine diseases by rapidly advancing…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | Springworks Therapeuticsspringworkstx.com HQ: Stamford, CT, United StatesReported employees: 201-500 | Stamford, CT, United States | 201-500 | Exact match | 392 contacts → |
“SpringWorks Therapeutics, a healthcare company of Merck KGaA, Darmstadt, Germany, is a commercial-stage biopharmaceutical company dedicated to improving the lives of patients with…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 |  Kiniksa Pharmaceuticalskiniksa.comHQ: Lexington, MA, United StatesReported employees: 201-500 | Lexington, MA, United States | 201-500 | Exact match | 365 contacts → |
“Kiniksa is a biopharmaceutical company dedicated to improving the lives of patients suffering from debilitating diseases by discovering, acquiring, developing, and commercializing…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 |  SOBIsobi-northamerica.comHQ: Waltham, MA, United StatesReported employees: 201-500 | Waltham, MA, United States | 201-500 | Exact match | 326 contacts → |
“Sobi is an international specialty healthcare company dedicated to developing treatments and extensive patient support networks for people with rare and difficult to treat…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Esperionesperion.com HQ: Ann Arbor, MI, United StatesReported employees: 201-500 | Ann Arbor, MI, United States | 201-500 | Exact match | 313 contacts → |
No recorded description excerpt is available for this public record. | |||||
Showing 25 of 270 company matches
Build a targeted audience, find relevant contacts and export available records to your workflow.
Free signup · 500 credits · limited free results
Rare disease companies: dataset insights
September 21, 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
269 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 270 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 21, 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 companies?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many rare disease companies did RevenueBase find based in the United States?
The saved snapshot records 270 matching company records, 91,999 tracked contacts and 33,184 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 21, 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 companies by geography
Browse the directory
Cite this list
RevenueBase Data Team. Top 25 rare disease companies based in the United States. Snapshot: September 21, 2026. https://revenuebase.ai/companies/rare-disease-companies/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 companies based in France
- Rare disease companies based in the United Kingdom
- AI companies based in the United States
- Cybersecurity companies based in the United States
- Manufacturing companies based in the United States
- Media companies based in the United States
Company intelligence · public preview
Top 25 rare disease companies based in the United States
RevenueBase records 270 matching company records for rare disease companies based in the United States, with 91,999 tracked contacts and 33,184 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 21, 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 companies 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 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 2 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 3 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 4 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 6 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 7 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 8 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 9 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 11 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 13 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 14 | Vanda Pharmaceuticalsvandapharma.com HQ: Washington, DC, United StatesReported employees: 201-500 | Washington, DC, United States | 201-500 | Exact match | 575 contacts → |
“Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 15 | Takeda Pharmaceuticals America, INCtakeda.com HQ: Lincolnshire, IL, United StatesReported employees: 10001+ | Lincolnshire, IL, United States | 10001+ | Exact match | 561 contacts → |
No recorded description excerpt is available for this public record. | |||||
| 16 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 17 | Chiesi Usa, INCchiesiusa.com HQ: Cary, NC, United StatesReported employees: 201-500 | Cary, NC, United States | 201-500 | Exact match | 513 contacts → |
“Chiesi USA, Inc, headquartered in Cary, N.C, is a specialty pharmaceutical company focused on commercialization of products for the hospital, rare disease and target office-based…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 18 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 19 | Rhythm Pharmaceuticals INCrhythmtx.com HQ: Boston, MA, United StatesReported employees: 201-500 | Boston, MA, United States | 201-500 | Exact match | 411 contacts → |
“Rhythm Pharmaceuticals is a global, commercial-stage biopharmaceutical company focused on transforming the lives of patients with rare neuroendocrine diseases by rapidly advancing…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 20 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 21 | Springworks Therapeuticsspringworkstx.com HQ: Stamford, CT, United StatesReported employees: 201-500 | Stamford, CT, United States | 201-500 | Exact match | 392 contacts → |
“SpringWorks Therapeutics, a healthcare company of Merck KGaA, Darmstadt, Germany, is a commercial-stage biopharmaceutical company dedicated to improving the lives of patients with…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | 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 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 23 | Kiniksa Pharmaceuticalskiniksa.com HQ: Lexington, MA, United StatesReported employees: 201-500 | Lexington, MA, United States | 201-500 | Exact match | 365 contacts → |
“Kiniksa is a biopharmaceutical company dedicated to improving the lives of patients suffering from debilitating diseases by discovering, acquiring, developing, and commercializing…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 24 | SOBIsobi-northamerica.com HQ: Waltham, MA, United StatesReported employees: 201-500 | Waltham, MA, United States | 201-500 | Exact match | 326 contacts → |
“Sobi is an international specialty healthcare company dedicated to developing treatments and extensive patient support networks for people with rare and difficult to treat…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 25 | Esperionesperion.com HQ: Ann Arbor, MI, United StatesReported employees: 201-500 | Ann Arbor, MI, United States | 201-500 | Exact match | 313 contacts → |
No recorded description excerpt is available for this public record. | |||||
Showing 25 of 270 company matches
Build a targeted audience, find relevant contacts and export available records to your workflow.
Free signup · 500 credits · limited free results
Rare disease companies: dataset insights
September 21, 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
269 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 270 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 21, 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 companies?
The separate RevenueBase SmartSearch column identifies inclusion at the closest semantic matching preset. It is not a literal keyword match, a per-company confidence score or a quality endorsement. Changing the search form opens a new search in RevenueBase; it does not reclassify these saved rows.
How many rare disease companies did RevenueBase find based in the United States?
The saved snapshot records 270 matching company records, 91,999 tracked contacts and 33,184 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 21, 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 companies by geography
Browse the directory
Cite this list
RevenueBase Data Team. Top 25 rare disease companies based in the United States. Snapshot: September 21, 2026. https://revenuebase.ai/companies/rare-disease-companies/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 companies based in France
- Rare disease companies based in the United Kingdom
- AI companies based in the United States
- Cybersecurity companies based in the United States
- Manufacturing companies based in the United States
- Media companies based in the United States