Company lists · Data refreshed September 2026
Top 25 Rare disease treatment companies worldwide
RevenueBase identified 619 company matches for rare disease treatment companies worldwide as of September 2026 and tracks 59,496 professional contacts across them, including 28,369 with verified work emails. The United States is the largest market and Boston the largest hub.
This list covers company matches for rare disease treatment companies worldwide, found by RevenueBase SmartSearch using the meaning of business descriptions in its company graph at Exact. Ranked by professionals tracked, largest first. Rows show recorded descriptions where available, Exact search membership and the snapshot date. Data refreshed September 21, 2026. Country and city lists for rare disease treatment companies follow the table.
Published by RevenueBase, a B2B data infrastructure company. This list was built with RevenueBase SmartSearch, the natural-language company search RevenueBase customers use to build their own targeted lists and unlock verified emails and direct dials at the companies on them. How these lists are built and judged.
The 25 largest rare disease treatment companies worldwide
Ranked by team size| # | Company | Contacts | |||
|---|---|---|---|---|---|
| 1 | 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 | |||||
| 2 | 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 | |||||
| 3 | 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 | |||||
| 4 | 3,593 contacts → | ||||
Exact match “Jazz Pharmaceuticals plc (Nasdaq: JAZZ), is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 5 | 1,561 contacts → | ||||
Exact match “Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases.” Recorded in the RevenueBase company graph · Sep 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 | 976 contacts → | ||||
Exact match “Pharming Group is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 10 | 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 | |||||
| 11 | 709 contacts → | ||||
Exact match “Partner of choice in specialty, hospital, and rare disease medicines ADVANZ PHARMA is a global pharmaceutical company with the purpose to improve patients’ lives by providing the…” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 12 | 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 | |||||
| 13 | 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 | |||||
| 14 | 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 | |||||
| 15 | 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 | |||||
| 16 | 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 | |||||
| 17 | 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 | |||||
| 18 | 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 | |||||
| 19 | 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 | |||||
| 20 | 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 | |||||
| 21 | 372 contacts → | ||||
Exact match “At Takeda UK and Ireland, we are guided by an unwavering commitment to put the patient first in everything we do.” Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 22 | 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 | |||||
| 23 | 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 | |||||
| 24 | 313 contacts → | ||||
Exact match Recorded description not available. Recorded in the RevenueBase company graph · Sep 21, 2026 · View company records in RevenueBase | |||||
| 25 | 282 contacts → | ||||
Exact match “Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases.” 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 treatment 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 619 rare disease treatment companies worldwide
See the full list, filter by size, metro and specialty, and access 28,369 tracked professional contacts with verified work emails. Direct dials are available where recorded.
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Rare disease treatment companies worldwide at a glance
As of Sep 21, 2026Companies by employee count
Email recency is reported on each country list linked below.
How this list was built
The recorded descriptions help you assess these company matches. Check a company’s website for its current services and locations.
- Source
- The RevenueBase company graph: 60 million companies and 398.9 million professional contacts resolved from primary sources, queried with RevenueBase SmartSearch, the natural-language company search available to every RevenueBase user.
- Coverage
- Every country. Country and city lists for rare disease treatment companies are linked below.
- What qualifies
- Company records worldwide whose business descriptions match “rare disease treatment companies” by meaning at the Exact setting. Descriptions are compiled in the RevenueBase company graph from public websites, profiles and company information where available; they are not guaranteed quotations from the company's website.
- RevenueBase SmartSearch
- Describe a niche company in your own words to find businesses by meaning. Exact, Similar, Related and Broad control the breadth of RevenueBase SmartSearch. This saved list uses Exact. Each row's Exact match badge shows inclusion in that search, not an individual score or a service-quality judgment.
- Public preview
- The public preview omits records without a verified work email. Dataset totals and geographic summaries describe the full matched records before these preview filters.
- Match quality
- RevenueBase's August 25, 2026 study assessed 4,198 description/company pairs across matching settings. The publisher reports 86% good or better at Exact. This evaluates the method across queries; it does not audit this individual list. Recorded company descriptions, where available, help you judge each match yourself. Read the full methodology: how the lists are built and what the study measures.
- Ranking
- Team size, largest first: companies are ordered by the number of professionals RevenueBase tracks at each one. Employee ranges shown are reported bands, for context.
- Verification
- Every displayed company carries at least one verified work email. RevenueBase tracks 28,369 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.
Rare disease treatment companies by location
3 lists · largest firstEvery Rare disease treatment companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
Frequently asked
What is RevenueBase SmartSearch?
RevenueBase SmartSearch finds niche companies by the meaning of their business descriptions in the RevenueBase company graph. Exact, Similar, Related and Broad control how closely companies must fit your description. This list uses Exact for “rare disease treatment companies”; each Exact match badge shows search membership, not an individual similarity or quality score.
How many rare disease treatment companies are there worldwide?
RevenueBase identified 619 company matches for rare disease treatment companies worldwide as of September 2026, using the meaning of business descriptions at Exact. This is the recorded matched set in the RevenueBase company graph.
What is the largest company on this list?
Genentech tops the displayed preview by professional contacts tracked in RevenueBase. Recorded headquarters appear in the company rows.
Which country has the most rare disease treatment companies?
The United States leads with 244 companies, followed by France (28) and the United Kingdom (25). Every rare disease treatment companies list by country and city is linked on this page.
Which city has the most rare disease treatment companies?
Boston leads with 16 company records, followed by Cambridge (10) and New York (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 28,369 professional contacts with verified work emails tracked across these 619 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 619 rare disease treatment companies worldwide. Start with a free account, no credit card.
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About this data
RevenueBase is a B2B data infrastructure company. Its company graph covers 60 million companies and 398.9 million professional contacts, with emails verified on a rolling cadence rather than scraped once and left to decay. These company lists are generated directly from that graph with RevenueBase SmartSearch, the same natural-language company search every RevenueBase user has: describe a market in plain words, get the companies that match, and add verified emails and direct dials for the people at them. It is the same data that powers RevenueBase's B2B data products, and a free account includes 500 credits to start. How these lists are built and judged.
More company lists
Company intelligence · public preview
Top 25 rare disease treatment companies worldwide
Find companies in any niche.
Describe the businesses you want in your own words. RevenueBase SmartSearch matches company descriptions by meaning.
RevenueBase records 619 matching company records for rare disease treatment companies worldwide, with 59,496 tracked contacts and 28,369 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 treatment companies worldwide
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 |  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. | |||||
| 2 |  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. | |||||
| 3 |  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. | |||||
| 4 |  Jazz Pharmaceuticalsjazzpharma.comHQ: Dublin, GermanyReported employees: 1,001-5,000 | Dublin, Germany | 1,001-5,000 | Exact match | 3,593 contacts → |
“Jazz Pharmaceuticals plc (Nasdaq: JAZZ), is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 |  SOBIsobi.comHQ: Stockholm, SwedenReported employees: 1,001-5,000 | Stockholm, Sweden | 1,001-5,000 | Exact match | 1,561 contacts → |
“Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases.” Recorded description excerpt · September 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 | Pharming Grouppharming.com HQ: Leiden, NetherlandsReported employees: 201-500 | Leiden, Netherlands | 201-500 | Exact match | 976 contacts → |
“Pharming Group is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 |  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. | |||||
| 11 |  Advanz Pharmaadvanzpharma.comHQ: London, United KingdomReported employees: 501-1,000 | London, United Kingdom | 501-1,000 | Exact match | 709 contacts → |
“Partner of choice in specialty, hospital, and rare disease medicines ADVANZ PHARMA is a global pharmaceutical company with the purpose to improve patients’ lives by providing the…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | 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. | |||||
| 13 |  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. | |||||
| 14 |  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. | |||||
| 15 |  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. | |||||
| 16 | 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. | |||||
| 17 | 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. | |||||
| 18 | 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. | |||||
| 19 | 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. | |||||
| 20 | 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. | |||||
| 21 |  Takeda UK And Irelandtakeda.comHQ: United StatesReported employees: 501-1,000 | United States | 501-1,000 | Exact match | 372 contacts → |
“At Takeda UK and Ireland, we are guided by an unwavering commitment to put the patient first in everything we do.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 |  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. | |||||
| 23 |  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. | |||||
| 24 | 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. | |||||
| 25 |  Viridian Therapeutics, INCviridiantherapeutics.comHQ: Waltham, MA, United StatesReported employees: 201-500 | Waltham, MA, United States | 201-500 | Exact match | 282 contacts → |
“Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 619 company matches
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Rare disease treatment 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
617 records in the displayed employee bands.
2 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
Email recency for this list is measured across the whole country list rather than for Worldwide, so it is reported there. Company counts and contact totals on this page are exact for Worldwide, over 619 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 ↗
Rare disease treatment companies by location
3 lists · largest firstEvery Rare disease treatment companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
Questions about this company list
What does Exact match mean for rare disease treatment 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 treatment companies did RevenueBase find worldwide?
The saved snapshot records 619 matching company records, 59,496 tracked contacts and 28,369 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.
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Cite this list
RevenueBase Data Team. Top 25 rare disease treatment companies worldwide. Snapshot: September 21, 2026. https://revenuebase.ai/companies/browse/rare-disease-treatment-companies.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗
More company lists
Company intelligence · public preview
Top 25 rare disease treatment companies worldwide
RevenueBase records 619 matching company records for rare disease treatment companies worldwide, with 59,496 tracked contacts and 28,369 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 treatment companies worldwide
The public list shows selected company records with contact and verified-email coverage. Reported employee bands provide separate company-size context.
| # | Company | Headquarters on record | Reported employees | RevenueBase SmartSearch | Tracked contacts |
|---|---|---|---|---|---|
| 1 | 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. | |||||
| 2 | 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. | |||||
| 3 | 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. | |||||
| 4 | Jazz Pharmaceuticalsjazzpharma.com HQ: Dublin, GermanyReported employees: 1,001-5,000 | Dublin, Germany | 1,001-5,000 | Exact match | 3,593 contacts → |
“Jazz Pharmaceuticals plc (Nasdaq: JAZZ), is a global biopharmaceutical company whose purpose is to innovate to transform the lives of patients and their families.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 5 | SOBIsobi.com HQ: Stockholm, SwedenReported employees: 1,001-5,000 | Stockholm, Sweden | 1,001-5,000 | Exact match | 1,561 contacts → |
“Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases.” Recorded description excerpt · September 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 | Pharming Grouppharming.com HQ: Leiden, NetherlandsReported employees: 201-500 | Leiden, Netherlands | 201-500 | Exact match | 976 contacts → |
“Pharming Group is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 10 | 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. | |||||
| 11 | Advanz Pharmaadvanzpharma.com HQ: London, United KingdomReported employees: 501-1,000 | London, United Kingdom | 501-1,000 | Exact match | 709 contacts → |
“Partner of choice in specialty, hospital, and rare disease medicines ADVANZ PHARMA is a global pharmaceutical company with the purpose to improve patients’ lives by providing the…” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 12 | 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. | |||||
| 13 | 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. | |||||
| 14 | 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. | |||||
| 15 | 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. | |||||
| 16 | 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. | |||||
| 17 | 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. | |||||
| 18 | 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. | |||||
| 19 | 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. | |||||
| 20 | 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. | |||||
| 21 | Takeda UK And Irelandtakeda.com HQ: United StatesReported employees: 501-1,000 | United States | 501-1,000 | Exact match | 372 contacts → |
“At Takeda UK and Ireland, we are guided by an unwavering commitment to put the patient first in everything we do.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
| 22 | 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. | |||||
| 23 | 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. | |||||
| 24 | 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. | |||||
| 25 | Viridian Therapeutics, INCviridiantherapeutics.com HQ: Waltham, MA, United StatesReported employees: 201-500 | Waltham, MA, United States | 201-500 | Exact match | 282 contacts → |
“Viridian is a biopharmaceutical company focused on engineering and developing potential best-in-class medicines for patients with serious and rare diseases.” Recorded description excerpt · September 21, 2026Open company data in RevenueBase ↗Free signup required. | |||||
Showing 25 of 619 company matches
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Rare disease treatment 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
617 records in the displayed employee bands.
2 matching records are outside the displayed bands or do not have a displayed size. Bars show shares of the displayed bands.
Email recency for this list is measured across the whole country list rather than for Worldwide, so it is reported there. Company counts and contact totals on this page are exact for Worldwide, over 619 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 ↗
Rare disease treatment companies by location
3 lists · largest firstEvery Rare disease treatment companies list RevenueBase publishes, by country and city. Counts are matching companies in each list as of its own refresh date.
Countries
Questions about this company list
What does Exact match mean for rare disease treatment 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 treatment companies did RevenueBase find worldwide?
The saved snapshot records 619 matching company records, 59,496 tracked contacts and 28,369 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
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Cite this list
RevenueBase Data Team. Top 25 rare disease treatment companies worldwide. Snapshot: September 21, 2026. https://revenuebase.ai/companies/browse/rare-disease-treatment-companies.
This is a selected public preview of saved company records, rather than an independently verified census. Open the source page ↗